(NAVIGATOR) Study of BLU-285 in Patients With Gastrointestinal Stromal Tumors (GIST) and Other Relapsed and Refractory Solid Tumors

• ClinicalTrials.gov Identifier: NCT02508532
• Recruitment Status: Completed
• First Posted: July 27, 2015
• Results First Posted: June 22, 2021
• Last Update Posted: June 21, 2022
• Sponsor: Blueprint Medicines Corporation
• Information provided by (Responsible Party): Blueprint Medicines Corporation

• Study Type: Interventional
• Study Design: Allocation: Non-Randomized; Intervention Model: Sequential Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Conditions: Gastrointestinal Stromal Tumors (GIST); Other Relapsed or Refractory Solid Tumors
• Intervention: Drug: Avapritinib
• Enrollment: 250

Participant Flow

Recruitment Details
Pre-assignment Details

Arm/Group Title	Experimental: Part 1 Avapritinib (Formerly BLU-285) 30 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 60 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 90 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 135 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 200 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 300 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 400 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 600 mg QD	Experimental: Part 1 and Part 2 Avapritinib (Formerly BLU-285) 300 or 400 mg QD
Arm/Group Description	Part 1: Patients received a starting dose of 30 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1: Patients received a starting dose of 60 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1: Patients received a starting dose of 90 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1: Patients received a starting dose of 135 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1: Patients received a starting dose of 200 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation. .	Part 1: Patients received a starting dose of 300 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1: Patients received a starting dose of 400 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1: Patients received a starting dose of 600 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1 and Part 2: Patients enrolled in Part 1 and Part 2 at a starting dose of 300 or 400 mg QD were included in the Part1/Part 2 safety and efficacy analysis. Patients received avapritinib in continuous 28 day cycles until discontinuation.
Period Title: Part 1 - Dose Determining Period
Started	6	6	6	6	6	6	7	3	0 [1]
Patients From Part 1 Included in the Part 2 Analyses	0	0	0	0	0	6	7	0	0
Completed	6	6	6	6	6	6	6	2	0
Not Completed	0	0	0	0	0	0	1	1	0
Reason Not Completed
did not complete >21 days of treatment	0	0	0	0	0	0	1	0	0
Not Evaluable	0	0	0	0	0	0	0	1	0
[1] This group includes patients enrolled in Part 1 and Part 2 at a starting dose of 300 or 400 mg QD. This group was not used to determine the MTD in Part 1
Period Title: Part 2 - Treatment
Started	0 [1]	0 [1]	0 [1]	0 [1]	0 [1]	0 [2]	0 [3]	0 [1]	217 [4]
Completed	0	0	0	0	0	0	0	0	0
Not Completed	0	0	0	0	0	0	0	0	217
Reason Not Completed
Disease Progression	0	0	0	0	0	0	0	0	118
Adverse Event	0	0	0	0	0	0	0	0	49
Physician Decision	0	0	0	0	0	0	0	0	12
Administrative	0	0	0	0	0	0	0	0	3
Sponsor Decision	0	0	0	0	0	0	0	0	28
Death	0	0	0	0	0	0	0	0	1
Withdrawal by Subject	0	0	0	0	0	0	0	0	6
[1] These patients are not included in Part 2 analyses; [2] Part 1 Only: 6 Patients from this group are included in the 300 or 400 mg QD group.; [3] Part 1 Only: 7 Patients from this group are included in the 300 or 400 mg QD group.; [4] Includes 13 patients enrolled in Part 1 at a starting dose of 300 mg (n = 6) or 400 mg (n = 7) QD.
Period Title: Part 1 and Part 2 End of Study
Started	6	6	6	6	6	0 [1]	0 [2]	3	217 [3]
Completed	0	0	0	0	0	0	0	0	0
Not Completed	6	6	6	6	6	0	0	3	217
Reason Not Completed
Disease Progression	2	0	0	0	0	0	0	1	6
Adverse Event	1	0	1	0	0	0	0	0	2
Death	0	4	2	4	3	0	0	1	111
Lost to Follow-up	1	1	1	0	0	0	0	0	6
Withdrawal by Subject	0	0	0	0	0	0	0	0	15
Physician Decision	0	0	1	0	1	0	0	0	8
Initiation of another therapy	0	0	0	0	0	0	0	0	1
Sponsor Decision	2	1	1	2	2	0	0	1	68
[1] These 6 patients are included in the 300 or 400 mg QD dose group; [2] These 7 patients are included in the 300 or 400 mg QD dose group; [3] Includes 13 patients enrolled in Part 1 at a starting dose of 300 mg (n = 6) or 400 mg (n = 7) QD.

Baseline Characteristics

Arm/Group Title		Experimental: Part 1 Avapritinib (Formerly BLU-285) 30 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 60 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 90 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 135 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 200 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 600 mg QD	Part 1 and Part 2 Avapritinib (Formerly BLU-285) 300 mg or 400 mg QD	Total
Arm/Group Description		Part 1: Patients received a starting dose of 30 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1: Patients received a starting dose of 60 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1: Patients received a starting dose of 90 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1: Patients received a starting dose of 135 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1: Patients received a starting dose of 200 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation. .	Part 1: Patients received a starting dose of 600 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1 and Part 2: Patients enrolled in Part 1 and Part 2 at a starting dose of 300 or 400 mg QD were included in the Part1/Part 2 safety and efficacy analysis. Patients received avapritinib in continuous 28 day cycles until discontinuation. Includes 13 patients from Part 1	Total of all reporting groups
Overall Number of Baseline Participants		6	6	6	6	6	3	217	250
Baseline Analysis Population Description		[Not Specified]
Age, Continuous; Mean (Standard Deviation); Unit of measure: Years
	Number Analyzed	6 participants	6 participants	6 participants	6 participants	6 participants	3 participants	217 participants	250 participants
		58.7 (12.21)	60.2 (6.77)	60.2 (11.84)	61.5 (10.05)	61.7 (8.31)	48.3 (20.82)	59.4 (11.00)	59.4 (10.97)
Sex: Female, Male; Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	6 participants	6 participants	6 participants	6 participants	6 participants	3 participants	217 participants	250 participants
	Female	4 66.7%	3 50.0%	1 16.7%	0 0.0%	3 50.0%	1 33.3%	84 38.7%	96 38.4%
	Male	2 33.3%	3 50.0%	5 83.3%	6 100.0%	3 50.0%	2 66.7%	133 61.3%	154 61.6%
Race (NIH/OMB); Measure Type: Count of Participants; Unit of measure: Participants
	Number Analyzed	6 participants	6 participants	6 participants	6 participants	6 participants	3 participants	217 participants	250 participants
	American Indian or Alaska Native	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%	2 0.9%	2 0.8%
	Asian	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%	22 10.1%	22 8.8%
	Native Hawaiian or Other Pacific Islander	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%	1 0.5%	1 0.4%
	Black or African American	1 16.7%	0 0.0%	0 0.0%	1 16.7%	0 0.0%	0 0.0%	10 4.6%	12 4.8%
	White	5 83.3%	6 100.0%	4 66.7%	5 83.3%	4 66.7%	3 100.0%	154 71.0%	181 72.4%
	More than one race	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%	0 0.0%
	Unknown or Not Reported	0 0.0%	0 0.0%	2 33.3%	0 0.0%	2 33.3%	0 0.0%	28 12.9%	32 12.8%
Region of Enrollment; Measure Type: Number; Unit of measure: Participants
Belgium	Number Analyzed	6 participants	6 participants	6 participants	6 participants	6 participants	3 participants	217 participants	250 participants
		0	0	1	1	1	0	13	16
France	Number Analyzed	6 participants	6 participants	6 participants	6 participants	6 participants	3 participants	217 participants	250 participants
		0	0	1	1	2	0	20	24
Germany	Number Analyzed	6 participants	6 participants	6 participants	6 participants	6 participants	3 participants	217 participants	250 participants
		1	2	0	1	0	0	14	18
Netherlands	Number Analyzed	6 participants	6 participants	6 participants	6 participants	6 participants	3 participants	217 participants	250 participants
		0	0	1	0	0	0	11	12
Poland	Number Analyzed	6 participants	6 participants	6 participants	6 participants	6 participants	3 participants	217 participants	250 participants
		0	0	0	0	0	0	8	8
South Korea	Number Analyzed	6 participants	6 participants	6 participants	6 participants	6 participants	3 participants	217 participants	250 participants
		0	0	0	0	0	0	17	17
Spain	Number Analyzed	6 participants	6 participants	6 participants	6 participants	6 participants	3 participants	217 participants	250 participants
		0	0	0	0	0	0	11	11
United Kingdom	Number Analyzed	6 participants	6 participants	6 participants	6 participants	6 participants	3 participants	217 participants	250 participants
		1	0	3	0	0	1	23	28
United States	Number Analyzed	6 participants	6 participants	6 participants	6 participants	6 participants	3 participants	217 participants	250 participants
		4	4	0	3	3	2	100	116
Body Mass Index (BMI) [1]; Mean (Standard Deviation); Unit of measure: Kg/m2
	Number Analyzed	3 participants	6 participants	6 participants	6 participants	6 participants	3 participants	198 participants	228 participants
		24.78 (4.22)	31.61 (8.63)	28.33 (3.76)	23.78 (2.71)	24.63 (5.16)	27.15 (4.54)	25.98 (6.22)	26.09 (6.15)
		[1] Measure Analysis Population Description: Body Mass Index was only calculated for patients with both a height and weight measurement at Baseline

Outcome Measures

1. Primary Outcome

Title	Part 1: Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D) of Avapritinib
Description	Patients with event(s) of dose-limiting toxicity
Time Frame	Cycle 1 (28 days) of treatment

Outcome Measure Data

Analysis Population Description

Patients that completed Part 1 or had a DLT

Arm/Group Title	Experimental: Part 1 Avapritinib (Formerly BLU-285) 30 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 60 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 90 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 135 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 200 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 300 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 400 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 600 mg QD
Arm/Group Description:	Part 1: Patients received a starting dose of 30 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1: Patients received a starting dose of 60 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1: Patients received a starting dose of 90 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1: Patients received a starting dose of 135 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1: Patients received a starting dose of 200 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation. .	Part 1: Patients received a starting dose of 300 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation. Patients that received at least one dose of avapritinib were included in the Part 2 analysis.	Part 1: Patients received a starting dose of 400 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation. Patients that received at least one dose of avapritinib were included in the Part 2 analysis.	Part 1: Patients received a starting dose of 600 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.
Overall Number of Participants Analyzed	6	6	6	6	6	6	6	2
Measure Type: Number; Unit of Measure: patients with event(s) of dose-limiting
	0	0	0	0	0	0	0	2

2. Primary Outcome

Title	Parts 1 and 2: Number of Patients With Adverse Events (AE) and Serious Adverse Events (SAE)
Description	The overall safety profile of the drug was assessed by reviewing the number of patients with AEs, SAEs and other events. There was no formal statistical analysis. Safety assessments continued for the duration of treatment.
Time Frame	AEs were collected from the start of study drug until 30 days after the last dose, SAEs were collected from the date of the informed consent signature until 30 days after the last dose of study drug, up to 5 years

Outcome Measure Data

Analysis Population Description

Safety Population - all patients that received at least one dose of avapritinib

Arm/Group Title	Experimental: Part 1 Avapritinib (Formerly BLU-285) 30 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 60 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 90 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 135 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 200 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 600 mg QD	Experimental: Part 1 and Part 2 Avapritinib (Formerly BLU-285) 300 mg or 400 mg QD
Arm/Group Description:	Part 1: Patients received a starting dose of 30 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1: Patients received a starting dose of 60 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1: Patients received a starting dose of 90 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1: Patients received a starting dose of 135 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1: Patients received a starting dose of 200 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation. .	Part 1: Patients received a starting dose of 600 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1 and Part 2: Patients enrolled in Part 1 and Part 2 at a starting dose of 300 or 400 mg QD were included in the Part1/Part 2 safety and efficacy analysis. 13 patients in this group were enrolled in Part 1 Patients received avapritinib in continuous 28 day cycles until discontinuation.
Overall Number of Participants Analyzed	6	6	6	6	6	3	217
Measure Type: Number; Unit of Measure: participants
Participants with an Adverse Event	6	6	6	6	6	3	216
Participants with a Serious Adverse Event	3	4	5	5	5	3	140

3. Primary Outcome

Title	Part 2: Objective Response Rate (ORR) Determined by Central Radiology Assessment Per mRECIST, Version 1.1
Description	To evaluate objective response rate (ORR) determined by central radiology assessment per mRECIST, version 1.1 in patients with advanced GIST treated with avapritinib. A complete response per modified Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) is defined as complete disappearance of all target lesions. A partial response is defined as at least 30% decrease in the sum of diameters of target lesions taking as reference the baseline sum of diameters. Overall Response (OR) = CR + PR
Time Frame	Tumor assessments were performed at screening, Cycle 3 Day 1, then every 2 cycles through Cycle 13, then every 3 cycles thereafter up to approximately 4 years. Each cycle is 28 days.

Outcome Measure Data

Analysis Population Description

patients enrolled in Part 1 or Part 2 with a starting dose of 300 or 400 mg, including 13 patients enrolled in Part 1

Arm/Group Title	Patients With a PDGFRA D842V Mutation	2L Patients Without a PDGFRA D842V Mutation	3L Patients Without a PDGFRA D842V Mutation
Arm/Group Description:	Patients with a PDGFRA D842V Mutation, any line of treatment	Patients that do not have a PDGFRA D842V mutation and have received 1 prior line of therapy	Patients that do not have a PDGFRA D842V mutation and have had 2 or more prior lines of therapy
Overall Number of Participants Analyzed	38	50	128
Measure Type: Count of Participants; Unit of Measure: Participants
Responder	36 94.7%	10 20.0%	20 15.6%
Non-Responder	2 5.3%	40 80.0%	108 84.4%

4. Secondary Outcome

Title	Maximum Plasma Drug Concentration (Cmax)
Description	Maximum plasma drug concentration (Cmax) following a single dose of avapritinib
Time Frame	Cycle 1 Day 1

Outcome Measure Data

Analysis Population Description

Patients enrolled in Part 1 and Part 2 with a starting dose of 300 or 400 mg QD and available samples for PK

Arm/Group Title	Part 1 Avapritinib (Formerly BLU-285) 300 mg QD	Part 1 Avapritinib (Formerly BLU-285) 400 mg QD
Arm/Group Description:	Part 1 and Part 2: Patients received a starting dose of 300 mg QD. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1 and Part 2: Patients received a starting dose of 400 mg QD. Patients received avapritinib in continuous 28 day cycles until discontinuation.
Overall Number of Participants Analyzed	113	50
Median (Standard Deviation); Unit of Measure: ng/mL
	305 (153)	343 (181)

5. Secondary Outcome

Title	Time to Maximum Plasma Drug Concentration (Tmax)
Description	Cycle 1 Day 1 PK time to maximum plasma drug concentration (Tmax)
Time Frame	Cycle 1 Day 1

Outcome Measure Data

Analysis Population Description

Patients enrolled in Part 1 and Part 2 with available samples for PK

Arm/Group Title	Part 1 and Part 2: Avapritinib (Formerly BLU-285) 300 mg QD	Part 1 and Part 2: Avapritinib (Formerly BLU-285) 400 mg QD
Arm/Group Description:	Part 1 and Part 2: Patients received a starting dose of 300 mg QD. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1 and Part 2: Patients received a starting dose of 400 mg QD. Patients received avapritinib in continuous 28 day cycles until discontinuation.
Overall Number of Participants Analyzed	113	50
Median (Full Range); Unit of Measure: hours
	4.0; (0.98 to 23.5)	4.02; (1.97 to 24.0)

6. Secondary Outcome

Title	Plasma Drug Concentration at 24 Hours Postdose Prior to the Next Daily Dose (C24)
Description	Plasma drug concentration at 24 hours postdose prior to the next daily dose (C24) following a single dose of avapritinib
Time Frame	Cycle 1 Day 1

Outcome Measure Data

Analysis Population Description

Patients enrolled in Part 1 or Part 2 with a starting dose of 300 or 400 mg QD and available PK samples

Arm/Group Title	Part 1 and Part 2: Avapritinib (Formerly BLU-285) 300 mg QD	Part 1 and Part 2: Avapritinib (Formerly BLU-285) 400 mg QD
Arm/Group Description:	Part 1 and Part 2: Patients received a starting dose of 300 mg QD. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1 and Part 2: Patients received a starting dose of 400 mg QD. Patients received avapritinib in continuous 28 day cycles until discontinuation.
Overall Number of Participants Analyzed	36	19
Mean (Standard Deviation); Unit of Measure: ng/mL
	134 (49.4)	185 (80.2)

7. Secondary Outcome

Title	Area Under the Plasma Concentration-time Curve From Time 0 to 24 Hours (AUC 0-24)
Description	Area under the plasma concentration-time curve from time 0 to 24 hours (AUC 0-24) following a single dose of avapritinib
Time Frame	Cycle 1 Day 1

Outcome Measure Data

Analysis Population Description

Patients enrolled in Part 1 or Part 2 with a starting dose of 300 or 400 mg QD and available PK samples

Arm/Group Title	Part 1 and Part 2: Avapritinib (Formerly BLU-285) 300 mg QD	Part 1 and Part 2: Avapritinib (Formerly BLU-285) 400 mg QD
Arm/Group Description:	Part 1 and Part 2: Patients received a starting dose of 300 mg QD. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1 and Part 2: Patients received a starting dose of 400 mg QD. Patients received avapritinib in continuous 28 day cycles until discontinuation.
Overall Number of Participants Analyzed	36	19
Mean (Standard Deviation); Unit of Measure: h*ng/mL
	4510 (1760)	5310 (2080)

8. Secondary Outcome

Title	Apparent Oral Clearance Unadjusted for Bioavailability (CL/F)
Description	Apparent oral clearance unadjusted for bioavailability (CL/F) following a single dose of avapritinib
Time Frame	Cycle 1 Day 1

Outcome Measure Data

Analysis Population Description

Patients enrolled in Part 1 or Part 2 with a starting dose of 300 or 400 mg QD and available PK samples

Arm/Group Title	Part 1 and Part 2: Avapritinib (Formerly BLU-285) 300 mg QD	Part 1 and Part 2: Avapritinib (Formerly BLU-285) 400 mg QD
Arm/Group Description:	Part 1 and Part 2: Patients received a starting dose of 300 mg QD. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1 and Part 2: Patients received a starting dose of 400 mg QD. Patients received avapritinib in continuous 28 day cycles until discontinuation.
Overall Number of Participants Analyzed	23	8
Mean (Standard Deviation); Unit of Measure: L/h
	31.5 (15.2)	29.9 (20.1)

9. Secondary Outcome

Title	Apparent Volume of Distribution, Unadjusted for Bioavailability (Vz/F)
Description	Apparent volume of distribution, unadjusted for bioavailability (Vz/F) following a single dose of avapritinib
Time Frame	Cycle 1 Day 1

Outcome Measure Data

Analysis Population Description

Patients enrolled in Part 1 and Part 2 with a starting dose of 300 or 400 mg QD and available PK samples

Arm/Group Title	Part 1 and Part 2: Avapritinib (Formerly BLU-285) 300 mg QD	Part 1 and Part 2: Avapritinib (Formerly BLU-285) 400 mg QD
Arm/Group Description:	Part 1 and Part 2: Patients received a starting dose of 300 mg QD. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1 and Part 2: Patients received a starting dose of 400 mg QD. Patients received avapritinib in continuous 28 day cycles until discontinuation.
Overall Number of Participants Analyzed	23	8
Mean (Standard Error); Unit of Measure: L
	1310 (676)	1340 (597)

10. Secondary Outcome

Title	Terminal Elimination Half-life (t1/2)
Description	Terminal elimination half-life (t1/2) following a single dose of avapritinib
Time Frame	Cycle 1 Day 1

Outcome Measure Data

Analysis Population Description

Patients enrolled in Part 1 or Part 2 with a starting dose of 300 or 400 mg QD and available PK samples

Arm/Group Title	Part 1 and Part 2: Avapritinib (Formerly BLU-285) 300 mg QD	Part 1 and Part 2: Avapritinib (Formerly BLU-285) 400 mg QD
Arm/Group Description:	Part 1 and Part 2: Patients received a starting dose of 300 mg QD. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1 and Part 2: Patients received a starting dose of 400 mg QD. Patients received avapritinib in continuous 28 day cycles until discontinuation.
Overall Number of Participants Analyzed	23	8
Mean (Standard Deviation); Unit of Measure: h
	32.1 (15.6)	43.5 (28.4)

11. Secondary Outcome

Title	Maximum Plasma Drug Concentration (Cmax) at Steady State
Description	Maximum plasma drug concentration (Cmax) at steady state following 15 days of QD dosing
Time Frame	Cycle 1 Day 15

Outcome Measure Data

Analysis Population Description

Patients enrolled in Part 1 and Part 2 with a starting dose of 300 or 400 mg QD and available samples for PK

Arm/Group Title	Part 1 and Part 2: Avapritinib (Formerly BLU-285) 300 mg QD	Part 1 and Part 2: Avapritinib (Formerly BLU-285) 400 mg QD
Arm/Group Description:	Part 1 and Part 2: Patients received a starting dose of 300 mg QD. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1 and Part 2: Patients received a starting dose of 400 mg QD. Patients received avapritinib in continuous 28 day cycles until discontinuation.
Overall Number of Participants Analyzed	110	38
Mean (Standard Deviation); Unit of Measure: ng/mL
	905 (402)	1140 (469)

12. Secondary Outcome

Title	Time of Maximal Concentration (Tmax) at Steady State
Description	Time of maximal concentration (Tmax) at steady state following 15 days of QD dosing
Time Frame	Cycle 1 Day 15

Outcome Measure Data

Analysis Population Description

Patients enrolled in Part 1 and Part 2 with a starting dose of 300 or 400 mg QD and available samples for PK

Arm/Group Title	Part 1 and Part 2: Avapritinib (Formerly BLU-285) 300 mg QD	Part 1 and Part 2: Avapritinib (Formerly BLU-285) 400 mg QD
Arm/Group Description:	Part 1 and Part 2: Patients received a starting dose of 300 mg QD. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1 and Part 2: Patients received a starting dose of 400 mg QD. Patients received avapritinib in continuous 28 day cycles until discontinuation.
Overall Number of Participants Analyzed	110	38
Median (Full Range); Unit of Measure: h
	4.0; (0.0 to 8.17)	3.99; (0.5 to 8.0)

13. Secondary Outcome

Title	Plasma Drug Concentration at 24 Hours Postdose Prior to the Next Daily Dose at Steady State (C24,ss)
Description	Plasma Drug Concentration at 24 Hours Postdose Prior to the Next Daily Dose at steady state (C24,ss) following 15 days of QD dosing
Time Frame	Cycle 1 Day 15

Outcome Measure Data

Analysis Population Description

Patients enrolled in Part 1 and Part 2 with a starting dose of 300 or 400 mg QD and available samples for PK

Arm/Group Title	Part 1 and Part 2: Avapritinib (Formerly BLU-285) 300 mg QD	Part 1 and Part 2: Avapritinib (Formerly BLU-285) 400 mg QD
Arm/Group Description:	Part 1 and Part 2: Patients received a starting dose of 300 mg QD. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1 and Part 2: Patients received a starting dose of 400 mg QD. Patients received avapritinib in continuous 28 day cycles until discontinuation.
Overall Number of Participants Analyzed	110	38
Mean (Standard Deviation); Unit of Measure: ng/mL
	593 (263)	760 (343)

14. Secondary Outcome

Title	Area Under the Plasma Concentration-time Curve Over the Dosing Interval at Steady Sate (AUC0-τ,ss) (τ=24 h)
Description	Area under the plasma concentration-time curve over the dosing interval at steady sate (AUC0-τ,ss) (τ=24 h) following 15 days of QD dosing
Time Frame	Cycle 1 Day 15

Outcome Measure Data

Analysis Population Description

Patients enrolled in Part 1 and Part 2 with a starting dose of 300 or 400 mg QD and available samples for PK

Arm/Group Title	Part 1 and Part 2: Avapritinib (Formerly BLU-285) 300 mg QD	Part 1 and Part 2: Avapritinib (Formerly BLU-285) 400 mg QD
Arm/Group Description:	Part 1 and Part 2: Patients received a starting dose of 300 mg QD. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1 and Part 2: Patients received a starting dose of 400 mg QD. Patients received avapritinib in continuous 28 day cycles until discontinuation.
Overall Number of Participants Analyzed	110	38
Mean (Standard Deviation); Unit of Measure: h*ng/mL
	16900 (7230)	21300 (9250)

15. Secondary Outcome

Title	Progression-free Survival Per mRECIST Version 1.1
Description	Progression-free survival is defined as the time in months from the start of treatment to the date of first documented progression or death due to any cause. Progression-free survival determined by central radiological assessment per modified Response Evaluation Criteria in Solid Tumors (mRECIST), version 1.1 in patients with advanced GIST. A progressively growing tumor must meet the following criteria: a) the target lesions must be greater or equal to 2cm in size and be a new GIST active lesion or b) the target lesions must be expanding on at least 2 sequential imaging studies.
Time Frame	Tumor assessments were performed at screening, Cycle 3 Day 1, then every 2 cycles through Cycle 13, then every 3 cycles thereafter up to approximately 4 years. Each cycle is 28 days.

Outcome Measure Data

Analysis Population Description

Patients enrolled in Part 1 or Part 2 with a starting dose of 300 or 400 mg QD

Arm/Group Title	Patients With a PDGFRA D842V Mutation	2L Patients Without a PDGFRA D842V Mutation	3L Patients Without a PDGFRA D842V Mutation
Arm/Group Description:	Patients with a PDGFRA D842V mutation, any line of treatment	Patients that do not have a PDGFRA D842V mutation and have received 1 prior line of therapy	Patients that do not have a PDGFRA D842V mutation and have had 2 or more prior lines of therapy
Overall Number of Participants Analyzed	38	50	128
Median (95% Confidence Interval); Unit of Measure: months
	27.6 [1]; (19.5 to NA)	5.5; (2.0 to 9.4)	3.7; (3.6 to 5.6)

[1]

The upper bounds of the confidence interval could not be calculated

16. Secondary Outcome

Title	Apparent Oral Clearance at Steady State, Unadjusted for Bioavailability (CLss/F)
Description	Apparent oral clearance at steady state, unadjusted for bioavailability (CLss/F) following 15 days of QD dosing
Time Frame	Cycle 1 Day 15

Outcome Measure Data

Analysis Population Description

Patients enrolled in Part 1 and Part 2 with a starting dose of 300 or 400 mg QD and available samples for PK

Arm/Group Title	Part 1 and Part 2: Avapritinib (Formerly BLU-285) 300 mg QD	Part 1 and Part 2: Avapritinib (Formerly BLU-285) 400 mg QD
Arm/Group Description:	Part 1 and Part 2: Patients received a starting dose of 300 mg QD. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1 and Part 2: Patients received a starting dose of 400 mg QD. Patients received avapritinib in continuous 28 day cycles until discontinuation.
Overall Number of Participants Analyzed	110	38
Mean (Standard Deviation); Unit of Measure: L/h
	21.8 (12.0)	22.8 (11.7)

17. Secondary Outcome

Title	Clinical Benefit Rate Determined by Central Radiology Assessment Per mRECIST, Version 1.1
Description	Percent of patients with a complete response, partial response or stable disease lasting more than 16 weeks. A complete response per modified Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) is defined as complete disappearance of all target lesions. A partial response is defined as at least 30% decrease in the sum of diameters of target lesions taking as reference the baseline sum of diameters. Stable disease is defined as a tumor that does not meet the criteria for progression or for response. A progressively growing tumor must meet the following criteria: a) the target lesions must be greater or equal to 2cm in size and be a new GIST active lesion or b) the target lesions must be expanding on at least 2 sequential imaging studies.
Time Frame	Tumor assessments were performed at screening, Cycle 3 Day 1, then every 2 cycles through Cycle 13, then every 3 cycles thereafter up to approximately 4 years. Each cycle is 28 days.

Outcome Measure Data

Analysis Population Description

patients with a starting dose of 300 or 400 mg QD

Arm/Group Title	Patients With a PDGFRA D842V Mutation	2L Patients Without a PDGFRA D842V Mutation	3L Patients Without a PDGFRA D842V Mutation
Arm/Group Description:	Patients with a PDGFRA D842V mutation, any line of treatment	Patients that do not have a PDGFRA D842V mutation and have received 1 prior line of therapy	Patients that do not have a PDGFRA D842V mutation and have had 2 or more prior lines of therapy
Overall Number of Participants Analyzed	38	50	128
Measure Type: Count of Participants; Unit of Measure: Participants
Clinical Benefit	37 97.4%	26 52.0%	51 39.8%
No Clinical Benefit	1 2.6%	24 48.0%	77 60.2%

18. Secondary Outcome

Title	Response Rate Determined by Central Radiology Assessment Per Choi Criteria
Description	A complete response is defined as complete disappearance of all target lesions. A partial response is ≥10% decrease tumor size at computed tomography (CT) or ≥15% decrease in tumor attenuation at computed tomography (CT) and no new lesions. The response rate is defined as complete response plus partial response.
Time Frame	Tumor assessments were performed at screening, Cycle 3 Day 1, then every 2 cycles through Cycle 13, then every 3 cycles thereafter up to approximately 4 years. Each cycle is 28 days.

Outcome Measure Data

Analysis Population Description

Patients enrolled in Part 1 or Part 2 with a starting dose of 300 or 400 mg

Arm/Group Title	Patients With a PDGFRA D842V Mutation	2L Patients Without a PDGFRA D842V Mutation	3L Patients Without a PDGFRA D842V Mutation
Arm/Group Description:	Patients with a PDGFRA D842V mutation, any line of treatment	Patients that do not have a PDGFRA D842V mutation and have received 1 prior line of therapy	Patients that do not have a PDGFRA D842V mutation and have had 2 or more prior lines of therapy
Overall Number of Participants Analyzed	38	50	128
Measure Type: Count of Participants; Unit of Measure: Participants
Responder	37 97.4%	18 36.0%	45 35.2%
Non-responder	1 2.6%	32 64.0%	83 64.8%

19. Secondary Outcome

Title	Duration of Response Determined by Central Radiology Assessment Per mRECIST, Version 1.1
Description	Duration from time to first documented CR/PR to date of first documented disease progression or death. A complete response per modified Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) is defined as complete disappearance of all target lesions. A partial response is defined as at least 30% decrease in the sum of diameters of target lesions taking as reference the baseline sum of diameters. Overall Response (OR) = CR + PR
Time Frame	Tumor assessments were performed at screening, Cycle 3 Day 1, then every 2 cycles through Cycle 13, then every 3 cycles thereafter up to approximately 4 years. Each cycle is 28 days.

Outcome Measure Data

Analysis Population Description

Patients in Part 1 and Part 2 with a starting dose of 300 or 400 mg QD. Only patients that achieved a CR or PR are included in this analysis

Arm/Group Title	Patients With PDGFRA D842V Mutation	2L Patients Without a PDGFRA D842V Mutation	3L Patients Without a PDGFRA D842V Mutation
Arm/Group Description:	Patients with a PDGFRA D842V mutation, any line of treatment	Patients that do not have a PDGFRA D842V mutation and have received 1 prior line of therapy	Patients that do not have a PDGFRA D842V mutation and have had 2 or more prior lines of therapy
Overall Number of Participants Analyzed	36	10	20
Median (95% Confidence Interval); Unit of Measure: months
	22.1 [1]; (14.3 to NA)	19.2; (9.2 to 19.2)	10.2; (7.2 to 15.0)

[1]

The upper bounds of the confidence interval could not be calculated

20. Secondary Outcome

Title	Median PFS on Last Prior Anti-cancer Therapy
Description	Progression Free Survival (PFS) is defined as the time in months from the start of treatment to the date of first documented disease progression or death due to any cause, which ever occurs first. PFS on last prior anti-cancer therapy is defined as the time in months from the start of last prior anti-cancer therapy to progression on that therapy.
Time Frame	Historical data collected at enrollment, all available data on prior therapy was collected

Outcome Measure Data

Analysis Population Description

Patients enrolled on any dose (Part 1 and Part 2) with data on progression-free survival for the most recent prior therapy

Arm/Group Title	Patients With a PDGFRA D842V Mutation	2L Patients Without a PDGFRA D842V Mutation	3L Patients Without a PDGFRA D842V Mutation
Arm/Group Description:	Patients with a PDGFRA D842V mutation, any line of treatment	Patients that do not have a PDGFRA D842V mutation and have received 1 prior line of therapy	Patients that do not have a PDGFRA D842V mutation and have had 2 or more prior lines of therapy
Overall Number of Participants Analyzed	45	51	142
Median (95% Confidence Interval); Unit of Measure: months
	5.9; (4.2 to 8.3)	31.0; (26.4 to 48.0)	6.4; (4.3 to 8.3)

21. Secondary Outcome

Title	Change From Baseline in Levels of KIT and PDGFRα Mutant Allele Fractions in Peripheral Blood
Description	Change of mutant allele fraction (MAF) summarizes the largest fold change. Change from baseline only displayed for patients with pre and post treatment MAF measurements. A positive number represents an increase in MAF. Data is only provided for patients that had both a baseline measurement and an end of treatment measurement.
Time Frame	Baseline and End of treatment

Outcome Measure Data

Analysis Population Description

Patients enrolled in Part 1 or Part 2 with a starting dose of 300 or 400 mg QD

Arm/Group Title	Experimental: Part 1 and Part 2 Avapritinib (Formerly BLU-285) 300 mg or 400 mg QD
Arm/Group Description:	Part 1 and Part 2: Patients enrolled in Part 1 and Part 2 at a starting dose of 300 or 400 mg QD were included in the Part1/Part 2 safety and efficacy analysis. Patients received avapritinib in continuous 28 day cycles until discontinuation.
Overall Number of Participants Analyzed	1
Mean (Standard Deviation); Unit of Measure: fraction of total
Change in PDGFRA MAF	8.702 [1] (NA)
Change in KIT MAF	14.398 [1] (NA)

[1]

Only one patient had data for this outcome measure so the standard deviation cannot be calculated

22. Secondary Outcome

Title	KIT, PDGFRA, and Other Cancer-relevant Mutations Present in Tumor Tissue at Baseline and EOT
Description	Change in mutations in tumor tissue at baseline and end of treatment (EOT). EOT tumor biopsies were optional and there were no EOT samples collected.
Time Frame	Baseline and end of treatment

Outcome Measure Data

Analysis Population Description

Patients with pre-and post-treatment tumor biopsies. No patients provided a post-treatment biopsy.

Arm/Group Title	Patients With a PDGFRA D842V Mutation	2L Patients Without a PDGFRA D842V Mutation	3L Patients Without a PDGFRA D842V Mutation
Arm/Group Description:	Patients with a PDGFRA D842V Mutation, any line of treatment	Patients that do not have a PDGFRA D842V mutation and have received 1 prior line of therapy	Patients that do not have a PDGFRA D842V mutation and have had 2 or more prior lines of therapy
Overall Number of Participants Analyzed	0	0	0

No data displayed because Outcome Measure has zero total analyzed.

Adverse Events

Time Frame	For each patient adverse event data were collected starting at the time of the first dose of study drug until 30 days after the final dose of study drug. Serious adverse event data were collected from the time the informed consent was signed until 30 days after the final dose of study drug. On average 8 months, up to 5 years.
Adverse Event Reporting Description	[Not Specified]
Arm/Group Title	Experimental: Part 1 Avapritinib (Formerly BLU-285) 30 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 60 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 90 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 135 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 200 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 600 mg QD	Experimental: Part 1 and Part 2 Avapritinib (Formerly BLU-285) 300 or 400 mg QD
Arm/Group Description	Part 1: Patients received a starting dose of 30 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1: Patients received a starting dose of 60 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1: Patients received a starting dose of 90 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1: Patients received a starting dose of 135 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1: Patients received a starting dose of 200 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1: Patients received a starting dose of 600 mg QD for 28 days and they were assessed for dose limiting toxicities (DLT). If no DTLs were observed the dose escalation continued. Patients received avapritinib in continuous 28 day cycles until discontinuation.	Part 1 and Part 2: Patients enrolled in Part 1 and Part 2 at a starting dose of 300 or 400 mg QD were included in the Part1/Part 2 safety and efficacy analysis. Patients received avapritinib in continuous 28 day cycles until discontinuation.
All-Cause Mortality
	Experimental: Part 1 Avapritinib (Formerly BLU-285) 30 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 60 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 90 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 135 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 200 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 600 mg QD	Experimental: Part 1 and Part 2 Avapritinib (Formerly BLU-285) 300 or 400 mg QD
	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)
Total	0/6 (0.00%)	4/6 (66.67%)	2/6 (33.33%)	4/6 (66.67%)	3/6 (50.00%)	1/3 (33.33%)	111/217 (51.15%)
Serious Adverse Events
	Experimental: Part 1 Avapritinib (Formerly BLU-285) 30 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 60 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 90 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 135 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 200 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 600 mg QD	Experimental: Part 1 and Part 2 Avapritinib (Formerly BLU-285) 300 or 400 mg QD
	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)
Total	3/6 (50.00%)	4/6 (66.67%)	5/6 (83.33%)	5/6 (83.33%)	5/6 (83.33%)	3/3 (100.00%)	140/217 (64.52%)
Blood and lymphatic system disorders
Anemia † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	1/6 (16.67%)	1/3 (33.33%)	24/217 (11.06%)
Febrile neutropenia † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Cardiac disorders
Myocardial infarction † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/3 (0.00%)	1/217 (0.46%)
Angina pectoris † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Angina unstable † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Atrial fibrillation † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Cardiac disorder † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Cardiac failure † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Pericardial effusion † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/3 (0.00%)	0/217 (0.00%)
Ear and labyrinth disorders
Vertigo † 1	1/6 (16.67%)	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	0/217 (0.00%)
Endocrine disorders
Adrenal haemorrhage † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Eye disorders
Papilloedema † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Gastrointestinal disorders
Abdominal pain † 1	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/3 (0.00%)	8/217 (3.69%)
Upper gastrointestinal haemorrhage † 1	1/6 (16.67%)	1/6 (16.67%)	0/6 (0.00%)	1/6 (16.67%)	1/6 (16.67%)	0/3 (0.00%)	4/217 (1.84%)
Gastrointestinal haemorrhage † 1	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	1/6 (16.67%)	0/3 (0.00%)	3/217 (1.38%)
Vomiting † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/3 (0.00%)	4/217 (1.84%)
Diarrhoea † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/3 (0.00%)	3/217 (1.38%)
Ascites † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	3/217 (1.38%)
Melaena † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	3/217 (1.38%)
Nausea † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/3 (0.00%)	2/217 (0.92%)
Peritoneal haemorrhage † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	3/217 (1.38%)
Small intestinal haemorrhage † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	3/217 (1.38%)
Small intestinal obstruction † 1	1/6 (16.67%)	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Colitis † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	2/217 (0.92%)
Gastric haemorrhage † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	2/217 (0.92%)
Intestinal obstruction † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	2/217 (0.92%)
Intra-abdominal haemorrhage † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	2/217 (0.92%)
Lower gastrointestinal haemorrhage † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	2/217 (0.92%)
Rectal haemorrhage † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	2/217 (0.92%)
Subileus † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	2/217 (0.92%)
Enteritis † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Enterocolitis † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Faecaloma † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Gastroduodenal haemorrhage † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Gastrointestinal ulcer haemorrhage † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Ileus † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Intestinal fistula † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Large intestinal obstruction † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Oesophagitis † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Oesophagitis ulcerative † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
General disorders
Disease progression † 1	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	1/6 (16.67%)	0/3 (0.00%)	18/217 (8.29%)
General physical health deterioration † 1	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	6/217 (2.76%)
Fatigue † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	2/217 (0.92%)
Malaise † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	2/217 (0.92%)
Pyrexia † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	2/217 (0.92%)
Asthenia † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Cyst rupture † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Face oedema † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/3 (33.33%)	0/217 (0.00%)
Haemorrhagic cyst † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Hypothermia † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Oedema peripheral † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/3 (0.00%)	0/217 (0.00%)
Hepatobiliary disorders
Cholecystitis † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Cholecystitis acute † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Hepatic failure † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/3 (0.00%)	0/217 (0.00%)
Hepatocellular injury † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Hyperbilirubinaemia † 1	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	0/217 (0.00%)
Infections and infestations
Sepsis † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	8/217 (3.69%)
Pneumonia † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	7/217 (3.23%)
Gastroenteritis † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/3 (0.00%)	2/217 (0.92%)
Urinary tract infection † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	3/217 (1.38%)
Clostridium difficile infection † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	1/6 (16.67%)	0/3 (0.00%)	0/217 (0.00%)
Device related infection † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	2/217 (0.92%)
Upper respiratory tract infection † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	2/217 (0.92%)
Abdominal abscess † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Appendicitis † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Catheter site infection † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Cellulitis † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Clostridium difficile colitis † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Cystitis † 1	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	0/217 (0.00%)
Gastroenteritis viral † 1	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	0/217 (0.00%)
Herpes simplex encephalitis † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Herpes zoster † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Infected cyst † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/3 (0.00%)	0/217 (0.00%)
Peritonitis † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Pneumococcal sepsis † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Pneumonia escherichia † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Pseudomembranous colitis † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Pulmonary sepsis † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Skin infection † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/3 (0.00%)	0/217 (0.00%)
Spinal cord infection † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Staphylococcal bacteraemia † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Streptococcal infection † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Wound infection † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Injury, poisoning and procedural complications
Subdural haematoma † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	2/217 (0.92%)
Cervical vertebral fracture † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Femur fracture † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Forearm fracture † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Hip fracture † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/3 (0.00%)	0/217 (0.00%)
Wound dehiscence † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Investigations
Blood creatine phosphokinase increased † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Metabolism and nutrition disorders
Dehydration † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	3/217 (1.38%)
Hypoglycaemia † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	2/217 (0.92%)
Diabetic ketoacidosis † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Fluid overload † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Hypomagnesaemia † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Hyponatraemia † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Malnutrition † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Musculoskeletal and connective tissue disorders
Fistula discharge † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Muscular weakness † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Pain in extremity † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Tumour haemorrhage † 1	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	4/217 (1.84%)
Tumour rupture † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	2/217 (0.92%)
Benign gastric neoplasm † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Breast cancer † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Leiomyosarcoma † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Metastases to perineum † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Metastases to peritoneum † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Metastatic neoplasm † 1	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	0/217 (0.00%)
Oesophageal squamous cell carcinoma † 1	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	0/217 (0.00%)
Prostate cancer † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/3 (0.00%)	0/217 (0.00%)
Renal cell carcinoma † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Nervous system disorders
Cerebral haemorrhage † 1	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	2/217 (0.92%)
Cognitive disorder † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/3 (0.00%)	2/217 (0.92%)
Encephalopathy † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	3/217 (1.38%)
Transient ischaemic attack † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	3/217 (1.38%)
Dementia † 1	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Haemorrhage intracranial † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	2/217 (0.92%)
Central nervous system lesion † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Dementia with Lewy bodies † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Dyskinesia † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Epilepsy † 1	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	0/217 (0.00%)
Headache † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Hepatic encephalopathy † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Nervous system disorder † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Neurological decompensation † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Peroneal nerve palsy † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Somnolence † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Syncope † 1	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	0/217 (0.00%)
Psychiatric disorders
Confusional state † 1	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	3/217 (1.38%)
Delirium † 1	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Psychotic disorder † 1	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Agitation † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Major depression † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/3 (0.00%)	0/217 (0.00%)
Mental disorder † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Mental status changes † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Mood altered † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/3 (33.33%)	0/217 (0.00%)
Personality change † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Schizophrenia † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Renal and urinary disorders
Acute kidney injury † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/3 (0.00%)	4/217 (1.84%)
Hydronephrosis † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	2/217 (0.92%)
Nephrolithiasis † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Renal failure † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Urinary retention † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Reproductive system and breast disorders
Pelvic pain † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Prostatitis † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/3 (0.00%)	0/217 (0.00%)
Testicular pain † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Respiratory, thoracic and mediastinal disorders
Pleural effusion † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/3 (0.00%)	5/217 (2.30%)
Dyspnoea † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	2/217 (0.92%)
Hypoxia † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	1/3 (33.33%)	0/217 (0.00%)
Pneumonia aspiration † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	2/217 (0.92%)
Bronchospasm † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Chronic obstructive pulmonary disease † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Haemothorax † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Laryngeal oedema † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Lung disorder † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Pulmonary embolism † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Respiratory failure † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Restrictive pulmonary disease † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Skin and subcutaneous tissue disorders
Angioedema † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Circumoral oedema † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
Dermatitis acneiform † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/3 (33.33%)	0/217 (0.00%)
Vascular disorders
Hypertension † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/3 (33.33%)	0/217 (0.00%)
Peripheral arterial occlusive disease † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	1/217 (0.46%)
1 Term from vocabulary, MedRA; † Indicates events were collected by systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	Experimental: Part 1 Avapritinib (Formerly BLU-285) 30 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 60 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 90 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 135 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 200 mg QD	Experimental: Part 1 Avapritinib (Formerly BLU-285) 600 mg QD	Experimental: Part 1 and Part 2 Avapritinib (Formerly BLU-285) 300 or 400 mg QD
	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)	Affected / at Risk (%)
Total	6/6 (100.00%)	6/6 (100.00%)	5/6 (83.33%)	6/6 (100.00%)	6/6 (100.00%)	3/3 (100.00%)	214/217 (98.62%)
Blood and lymphatic system disorders
Neutropenia † 1	1/6 (16.67%)	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	1/3 (33.33%)	24/217 (11.06%)
Leukopenia † 1	1/6 (16.67%)	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/3 (33.33%)	16/217 (7.37%)
Lymphopenia † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/3 (0.00%)	14/217 (6.45%)
Eye disorders
Periorbital oedema † 1	4/6 (66.67%)	4/6 (66.67%)	1/6 (16.67%)	4/6 (66.67%)	2/6 (33.33%)	1/3 (33.33%)	95/217 (43.78%)
Lacrimation increased † 1	2/6 (33.33%)	2/6 (33.33%)	2/6 (33.33%)	4/6 (66.67%)	2/6 (33.33%)	1/3 (33.33%)	76/217 (35.02%)
Eyelid oedema † 1	0/6 (0.00%)	1/6 (16.67%)	1/6 (16.67%)	1/6 (16.67%)	0/6 (0.00%)	0/3 (0.00%)	18/217 (8.29%)
Vision blurred † 1	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	1/6 (16.67%)	1/6 (16.67%)	0/3 (0.00%)	14/217 (6.45%)
Gastrointestinal disorders
Constipation † 1	2/6 (33.33%)	3/6 (50.00%)	2/6 (33.33%)	1/6 (16.67%)	2/6 (33.33%)	1/3 (33.33%)	54/217 (24.88%)
Dyspepsia † 1	2/6 (33.33%)	2/6 (33.33%)	0/6 (0.00%)	4/6 (66.67%)	0/6 (0.00%)	1/3 (33.33%)	36/217 (16.59%)
Gastrooesophageal reflux disease † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	2/6 (33.33%)	0/6 (0.00%)	0/3 (0.00%)	22/217 (10.14%)
Abdominal distension † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/3 (0.00%)	22/217 (10.14%)
Abdominal pain upper † 1	2/6 (33.33%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/3 (0.00%)	18/217 (8.29%)
Dry mouth † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	2/6 (33.33%)	2/6 (33.33%)	1/3 (33.33%)	16/217 (7.37%)
Dysphagia † 1	2/6 (33.33%)	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	0/3 (0.00%)	11/217 (5.07%)
General disorders
Feeling cold † 1	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/3 (0.00%)	17/217 (7.83%)
Influenza like illness † 1	1/6 (16.67%)	0/6 (0.00%)	2/6 (33.33%)	1/6 (16.67%)	1/6 (16.67%)	1/3 (33.33%)	11/217 (5.07%)
Chills † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/3 (0.00%)	12/217 (5.53%)
Injury, poisoning and procedural complications
Contusion † 1	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	1/6 (16.67%)	1/3 (33.33%)	10/217 (4.61%)
Investigations
Blood bilirubin increased † 1	1/6 (16.67%)	1/6 (16.67%)	1/6 (16.67%)	1/6 (16.67%)	0/6 (0.00%)	2/3 (66.67%)	48/217 (22.12%)
Weight decreased † 1	1/6 (16.67%)	2/6 (33.33%)	0/6 (0.00%)	3/6 (50.00%)	1/6 (16.67%)	2/3 (66.67%)	38/217 (17.51%)
Aspartate aminotransferase increased † 1	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/3 (0.00%)	35/217 (16.13%)
Blood creatinine increased † 1	0/6 (0.00%)	1/6 (16.67%)	2/6 (33.33%)	1/6 (16.67%)	1/6 (16.67%)	0/3 (0.00%)	21/217 (9.68%)
Weight increased † 1	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/3 (0.00%)	15/217 (6.91%)
Alanine aminotransferase increased † 1	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/3 (0.00%)	13/217 (5.99%)
Neutrophil count decreased † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/3 (0.00%)	14/217 (6.45%)
White blood cell count decreased † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	0/3 (0.00%)	14/217 (6.45%)
Metabolism and nutrition disorders
Hypokalaemia † 1	2/6 (33.33%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	2/6 (33.33%)	1/3 (33.33%)	43/217 (19.82%)
Hypophosphataemia † 1	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	3/6 (50.00%)	2/6 (33.33%)	1/3 (33.33%)	34/217 (15.67%)
Decreased appetite † 1	1/6 (16.67%)	3/6 (50.00%)	2/6 (33.33%)	3/6 (50.00%)	2/6 (33.33%)	3/3 (100.00%)	88/217 (40.55%)
Musculoskeletal and connective tissue disorders
Back pain † 1	1/6 (16.67%)	0/6 (0.00%)	2/6 (33.33%)	1/6 (16.67%)	1/6 (16.67%)	1/3 (33.33%)	25/217 (11.52%)
Myalgia † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	1/6 (16.67%)	1/3 (33.33%)	15/217 (6.91%)
Arthralgia † 1	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	1/6 (16.67%)	1/6 (16.67%)	0/3 (0.00%)	14/217 (6.45%)
Nervous system disorders
Memory impairment † 1	2/6 (33.33%)	2/6 (33.33%)	0/6 (0.00%)	2/6 (33.33%)	2/6 (33.33%)	1/3 (33.33%)	73/217 (33.64%)
Dizziness † 1	2/6 (33.33%)	1/6 (16.67%)	0/6 (0.00%)	2/6 (33.33%)	2/6 (33.33%)	2/3 (66.67%)	51/217 (23.50%)
Dysgeusia † 1	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	3/6 (50.00%)	1/6 (16.67%)	2/3 (66.67%)	40/217 (18.43%)
Disturbance in attention † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	1/6 (16.67%)	0/3 (0.00%)	16/217 (7.37%)
Peripheral sensory neuropathy † 1	1/6 (16.67%)	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	2/6 (33.33%)	1/3 (33.33%)	13/217 (5.99%)
Paraesthesia † 1	0/6 (0.00%)	0/6 (0.00%)	2/6 (33.33%)	2/6 (33.33%)	0/6 (0.00%)	0/3 (0.00%)	11/217 (5.07%)
Psychiatric disorders
Insomnia † 1	2/6 (33.33%)	2/6 (33.33%)	0/6 (0.00%)	3/6 (50.00%)	1/6 (16.67%)	1/3 (33.33%)	32/217 (14.75%)
Anxiety † 1	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	1/6 (16.67%)	1/3 (33.33%)	20/217 (9.22%)
Depression † 1	0/6 (0.00%)	2/6 (33.33%)	0/6 (0.00%)	2/6 (33.33%)	2/6 (33.33%)	0/3 (0.00%)	11/217 (5.07%)
Respiratory, thoracic and mediastinal disorders
Cough † 1	2/6 (33.33%)	1/6 (16.67%)	4/6 (66.67%)	0/6 (0.00%)	4/6 (66.67%)	2/3 (66.67%)	27/217 (12.44%)
Skin and subcutaneous tissue disorders
Hair colour changes † 1	2/6 (33.33%)	2/6 (33.33%)	1/6 (16.67%)	4/6 (66.67%)	2/6 (33.33%)	1/3 (33.33%)	50/217 (23.04%)
Alopecia † 1	1/6 (16.67%)	2/6 (33.33%)	0/6 (0.00%)	1/6 (16.67%)	0/6 (0.00%)	1/3 (33.33%)	31/217 (14.29%)
Rash † 1	1/6 (16.67%)	0/6 (0.00%)	1/6 (16.67%)	1/6 (16.67%)	2/6 (33.33%)	1/3 (33.33%)	30/217 (13.82%)
Dry skin † 1	2/6 (33.33%)	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	0/6 (0.00%)	1/3 (33.33%)	21/217 (9.68%)
Pruritus † 1	3/6 (50.00%)	2/6 (33.33%)	0/6 (0.00%)	0/6 (0.00%)	2/6 (33.33%)	0/3 (0.00%)	18/217 (8.29%)
Rash maculo-papular † 1	1/6 (16.67%)	0/6 (0.00%)	0/6 (0.00%)	1/6 (16.67%)	1/6 (16.67%)	0/3 (0.00%)	13/217 (5.99%)
1 Term from vocabulary, MedRA; † Indicates events were collected by systematic assessment

Limitations and Caveats

[Not Specified]

More Information

Certain Agreements

Principal Investigators are NOT employed by the organization sponsoring the study.

There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.

Results Point of Contact

• Name/Title: Medical Information
• Organization: Blueprint Medicines
• Phone: 1-888-258-7768
• EMail: medinfo@blueprintmedicines.com

Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

Kang YK, George S, Jones RL, Rutkowski P, Shen L, Mir O, Patel S, Zhou Y, von Mehren M, Hohenberger P, Villalobos V, Brahmi M, Tap WD, Trent J, Pantaleo MA, Schoffski P, He K, Hew P, Newberry K, Roche M, Heinrich MC, Bauer S. Avapritinib Versus Regorafenib in Locally Advanced Unresectable or Metastatic GI Stromal Tumor: A Randomized, Open-Label Phase III Study. J Clin Oncol. 2021 Oct 1;39(28):3128-3139. doi: 10.1200/JCO.21.00217. Epub 2021 Aug 3.

von Mehren M, Heinrich MC, Shi H, Iannazzo S, Mankoski R, Dimitrijevic S, Hoehn G, Chiroli S, George S. Clinical efficacy comparison of avapritinib with other tyrosine kinase inhibitors in gastrointestinal stromal tumors with PDGFRA D842V mutation: a retrospective analysis of clinical trial and real-world data. BMC Cancer. 2021 Mar 19;21(1):291. doi: 10.1186/s12885-021-08013-1.

Jones RL, Serrano C, von Mehren M, George S, Heinrich MC, Kang YK, Schoffski P, Cassier PA, Mir O, Chawla SP, Eskens FALM, Rutkowski P, Tap WD, Zhou T, Roche M, Bauer S. Avapritinib in unresectable or metastatic PDGFRA D842V-mutant gastrointestinal stromal tumours: Long-term efficacy and safety data from the NAVIGATOR phase I trial. Eur J Cancer. 2021 Mar;145:132-142. doi: 10.1016/j.ejca.2020.12.008. Epub 2021 Jan 16.

Joseph CP, Abaricia SN, Angelis MA, Polson K, Jones RL, Kang YK, Riedel RF, Schoffski P, Serrano C, Trent J, Tetzlaff ED, Si TD, Zhou T, Doyle A, Bauer S, Roche M, Havnaer T. Optimal Avapritinib Treatment Strategies for Patients with Metastatic or Unresectable Gastrointestinal Stromal Tumors. Oncologist. 2021 Apr;26(4):e622-e631. doi: 10.1002/onco.13632. Epub 2021 Jan 5.

Heinrich MC, Jones RL, von Mehren M, Schoffski P, Serrano C, Kang YK, Cassier PA, Mir O, Eskens F, Tap WD, Rutkowski P, Chawla SP, Trent J, Tugnait M, Evans EK, Lauz T, Zhou T, Roche M, Wolf BB, Bauer S, George S. Avapritinib in advanced PDGFRA D842V-mutant gastrointestinal stromal tumour (NAVIGATOR): a multicentre, open-label, phase 1 trial. Lancet Oncol. 2020 Jul;21(7):935-946. doi: 10.1016/S1470-2045(20)30269-2. Erratum In: Lancet Oncol. 2020 Sep;21(9):e418.

Gebreyohannes YK, Wozniak A, Zhai ME, Wellens J, Cornillie J, Vanleeuw U, Evans E, Gardino AK, Lengauer C, Debiec-Rychter M, Sciot R, Schoffski P. Robust Activity of Avapritinib, Potent and Highly Selective Inhibitor of Mutated KIT, in Patient-derived Xenograft Models of Gastrointestinal Stromal Tumors. Clin Cancer Res. 2019 Jan 15;25(2):609-618. doi: 10.1158/1078-0432.CCR-18-1858. Epub 2018 Oct 1.

• Responsible Party: Blueprint Medicines Corporation
• ClinicalTrials.gov Identifier: NCT02508532
• Other Study ID Numbers: BLU-285-1101
• First Submitted: July 23, 2015
• First Posted: July 27, 2015
• Results First Submitted: March 5, 2021
• Results First Posted: June 22, 2021
• Last Update Posted: June 21, 2022