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(VOYAGER) Study of Avapritinib vs Regorafenib in Patients With Locally Advanced Unresectable or Metastatic GIST

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ClinicalTrials.gov Identifier: NCT03465722
Recruitment Status : Completed
First Posted : March 14, 2018
Results First Posted : May 14, 2021
Last Update Posted : October 6, 2022
Sponsor:
Information provided by (Responsible Party):
Blueprint Medicines Corporation

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition GIST
Interventions Drug: avapritinib
Drug: regorafenib
Enrollment 476
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Avapritinib Regorafinib
Hide Arm/Group Description 300 mg PO QD avapritinib: Avapritinib tablets for oral administration. Avapritinib will be dosed at 300 mg once daily, continuously. 160 mg PO QD regorafenib: Regorafenib tablets for oral administration. Regorafenib will be dosed at 160 mg once daily for 3 weeks out of every 4 weeks (ie. 3 weeks on/1 week off).
Period Title: Overall Study
Started 240 236
Completed 0 0
Not Completed 240 236
Reason Not Completed
Withdrawal by Subject             21             14
Death             89             87
Lost to Follow-up             5             3
Administrative/Other             33             32
Not treated             1             2
Sponsor decision             91             98
Arm/Group Title Avapritinib Regorafinib Total
Hide Arm/Group Description 300 mg PO QD avapritinib: Avapritinib tablets for oral administration. Avapritinib will be dosed at 300 mg once daily, continuously. 160 mg PO QD regorafenib: Regorafenib tablets for oral administration. Regorafenib will be dosed at 160 mg once daily for 3 weeks out of every 4 weeks (ie. 3 weeks on/1 week off). Total of all reporting groups
Overall Number of Baseline Participants 240 236 476
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 240 participants 236 participants 476 participants
61.1  (10.96) 61.0  (10.74) 61.1  (10.84)
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 240 participants 236 participants 476 participants
Female
78
  32.5%
80
  33.9%
158
  33.2%
Male
162
  67.5%
156
  66.1%
318
  66.8%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 240 participants 236 participants 476 participants
American Indian or Alaska Native
0
   0.0%
0
   0.0%
0
   0.0%
Asian
64
  26.7%
64
  27.1%
128
  26.9%
Native Hawaiian or Other Pacific Islander
1
   0.4%
1
   0.4%
2
   0.4%
Black or African American
9
   3.8%
5
   2.1%
14
   2.9%
White
139
  57.9%
143
  60.6%
282
  59.2%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
27
  11.3%
23
   9.7%
50
  10.5%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Singapore Number Analyzed 240 participants 236 participants 476 participants
2 2 4
Hungary Number Analyzed 240 participants 236 participants 476 participants
2 1 3
United States Number Analyzed 240 participants 236 participants 476 participants
71 62 133
Czechia Number Analyzed 240 participants 236 participants 476 participants
3 1 4
United Kingdom Number Analyzed 240 participants 236 participants 476 participants
12 12 24
Spain Number Analyzed 240 participants 236 participants 476 participants
14 11 25
Canada Number Analyzed 240 participants 236 participants 476 participants
2 6 8
Austria Number Analyzed 240 participants 236 participants 476 participants
0 1 1
Netherlands Number Analyzed 240 participants 236 participants 476 participants
2 6 8
South Korea Number Analyzed 240 participants 236 participants 476 participants
23 20 43
Sweden Number Analyzed 240 participants 236 participants 476 participants
6 7 13
Belgium Number Analyzed 240 participants 236 participants 476 participants
1 1 2
China Number Analyzed 240 participants 236 participants 476 participants
35 39 74
Poland Number Analyzed 240 participants 236 participants 476 participants
10 9 19
Italy Number Analyzed 240 participants 236 participants 476 participants
13 15 28
Australia Number Analyzed 240 participants 236 participants 476 participants
5 5 10
France Number Analyzed 240 participants 236 participants 476 participants
22 20 42
Germany Number Analyzed 240 participants 236 participants 476 participants
17 18 35
Body Mass Index (BMI)   [1] [2] 
Mean (Standard Deviation)
Unit of measure:  Kilogram per meter square
Number Analyzed 230 participants 225 participants 455 participants
25.50  (5.563) 24.69  (5.163) 25.10  (5.378)
[1]
Measure Description: Both height and weight measurements are needed for body mass index calculation. Some patients had missing height and/or weight measurements. Only patients with both a height and a weight measurement are included in the body mass index calculation
[2]
Measure Analysis Population Description: Both height and weight measurements are needed for body mass index calculation. Some patients had missing height and/or weight measurements. Only patients with both a height and a weight measurement are included in the body mass index calculation
1.Primary Outcome
Title Efficacy of Avapritinib Based on Progression-free Survival (PFS) Determined by Central Radiological Assessment Per Modified Response Evaluation Criteria in Solid Tumors (mRECIST), Version 1.1
Hide Description To demonstrate the efficacy of avapritinib based on progression-free survival (PFS) determined by central radiological assessment per modified Response Evaluation Criteria in Solid Tumors (mRECIST), version 1.1 in patients with advanced GIST following 2 or 3 regimens of prior treatment with a tyrosine kinase inhibitor (TKI), including imatinib, compared to patients treated with regorafenib. 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 24 Months
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat Population that included all patients randomized to study.
Arm/Group Title Avapritinib Regorafinib
Hide Arm/Group Description:
300 mg PO QD avapritinib: Avapritinib tablets for oral administration. Avapritinib will be dosed at 300 mg once daily, continuously.
160 mg PO QD regorafenib: Regorafenib tablets for oral administration. Regorafenib will be dosed at 160 mg once daily for 3 weeks out of every 4 weeks (ie. 3 weeks on/1 week off).
Overall Number of Participants Analyzed 240 236
Median (95% Confidence Interval)
Unit of Measure: months
4.2
(3.7 to 5.6)
5.6
(3.8 to 7.2)
2.Secondary Outcome
Title Objective Response Rate (ORR) Determined by Central Radiology Assessment Per mRECIST, Version 1.1
Hide 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 compared to patients treated with regorafenib. A complete response (CR) per modified Response Evaluation Criteria In Solid Tumors Criteria (RECIST v1.1) is defined as complete disappearance of all target lesions. A partial response (PR) 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 24 Months
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat Population that included all patients randomized to study.
Arm/Group Title Avapritinib Regorafinib
Hide Arm/Group Description:
300 mg PO QD avapritinib: Avapritinib tablets for oral administration. Avapritinib will be dosed at 300 mg once daily, continuously.
160 mg PO QD regorafenib: Regorafenib tablets for oral administration. Regorafenib will be dosed at 160 mg once daily for 3 weeks out of every 4 weeks (ie. 3 weeks on/1 week off).
Overall Number of Participants Analyzed 240 236
Measure Type: Count of Participants
Unit of Measure: Participants
Responder
41
  17.1%
17
   7.2%
Non-Responder
199
  82.9%
219
  92.8%
3.Secondary Outcome
Title Overall Survival (OS) in Patients With Advanced GIST Treated With Avapritinib Compared to Patients Treated With Regorafenib
Hide Description To evaluate overall survival (OS) in patients with advanced GIST treated with avapritinib compared to patients treated with regorafenib
Time Frame 24 Months
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Avapritinib Regorafinib
Hide Arm/Group Description:
300 mg PO QD avapritinib: Avapritinib tablets for oral administration. Avapritinib will be dosed at 300 mg once daily, continuously.
160 mg PO QD regorafenib: Regorafenib tablets for oral administration. Regorafenib will be dosed at 160 mg once daily for 3 weeks out of every 4 weeks (ie. 3 weeks on/1 week off).
Overall Number of Participants Analyzed 240 236
Median (95% Confidence Interval)
Unit of Measure: months
19.2 [1] 
(19.2 to NA)
17.4 [1] 
(15.8 to NA)
[1]
Since the primary endpoint of statistically significant improvement in PFS was not met patients are not being followed for OS. The upper bound of the confidence interval is not estimable by the Kaplan-Meier analysis due to the short follow-up time.
4.Secondary Outcome
Title European Organisation for Research and Treatment of Cancer Quality of Life (EORTC-QLQ-30). Change in Individual Scores in Patients With Advanced GIST Treated With Avapritinib Compared to Patients Treated With Regorafenib
Hide Description The Global Health Status Score is derived from question 29 and 30 on the EORTC-QLQ-C30 tool. The change in score was assessed between baseline and week 12 in patients treated with advanced GIST treated with avapritinib compared to patients treated with regorafenib. The Global Health Status Score score range is 0 to 100 with a higher score indicating better global health status. A positive change indicates improvement in global health status.
Time Frame Difference between baseline and week 12 of treatment
Hide Outcome Measure Data
Hide Analysis Population Description
Intent-to-Treat Population with both a baseline and a Week 12 measurements.
Arm/Group Title Avapritinib Regorafinib
Hide Arm/Group Description:
300 mg PO QD avapritinib: Avapritinib tablets for oral administration. Avapritinib will be dosed at 300 mg once daily, continuously.
160 mg PO QD regorafenib: Regorafenib tablets for oral administration. Regorafenib will be dosed at 160 mg once daily for 3 weeks out of every 4 weeks (ie. 3 weeks on/1 week off).
Overall Number of Participants Analyzed 123 138
Mean (Standard Deviation)
Unit of Measure: scores on a scale
-5.7  (24.29) -4.4  (20.74)
Time Frame From the date of first study drug administration to 30 days after last study drug administration, approximately 6 months
Adverse Event Reporting Description The total number of at risk patients includes patients that were randomized to treatment and received at least one dose of treatment. Patients that were randomized by did not receive treatment were excluded from the safety analysis.
 
Arm/Group Title Avapritinib Regorafinib
Hide Arm/Group Description 300 mg PO QD avapritinib: Avapritinib tablets for oral administration. Avapritinib will be dosed at 300 mg once daily, continuously. 160 mg PO QD regorafenib: Regorafenib tablets for oral administration. Regorafenib will be dosed at 160 mg once daily for 3 weeks out of every 4 weeks (ie. 3 weeks on/1 week off).
All-Cause Mortality
Avapritinib Regorafinib
Affected / at Risk (%) Affected / at Risk (%)
Total   89/239 (37.24%)   87/234 (37.18%) 
Hide Serious Adverse Events
Avapritinib Regorafinib
Affected / at Risk (%) Affected / at Risk (%)
Total   106/239 (44.35%)   91/234 (38.89%) 
Blood and lymphatic system disorders     
Anaemia † 1  27/239 (11.30%)  7/234 (2.99%) 
Febrile neutropenia † 1  1/239 (0.42%)  0/234 (0.00%) 
Haemolytic uraemic syndrome † 1  1/239 (0.42%)  0/234 (0.00%) 
Thrombocytopenia † 1  0/239 (0.00%)  1/234 (0.43%) 
Cardiac disorders     
Cardiac failure congestive † 1  2/239 (0.84%)  0/234 (0.00%) 
Myocarditis † 1  1/239 (0.42%)  0/234 (0.00%) 
Pericardial effusion † 1  1/239 (0.42%)  0/234 (0.00%) 
Acute coronary syndrome † 1  0/239 (0.00%)  1/234 (0.43%) 
Atrial fibrillation † 1  0/239 (0.00%)  1/234 (0.43%) 
Cardiac failure † 1  0/239 (0.00%)  1/234 (0.43%) 
Myocardial infarction † 1  0/239 (0.00%)  1/234 (0.43%) 
Ventricular arrhythmia † 1  0/239 (0.00%)  1/234 (0.43%) 
Acute myocardial infarction † 1  0/239 (0.00%)  1/234 (0.43%) 
Eye disorders     
Retinal vein occlusion † 1  1/239 (0.42%)  0/234 (0.00%) 
Gastrointestinal disorders     
Abdominal pain † 1  13/239 (5.44%)  11/234 (4.70%) 
Ascites † 1  5/239 (2.09%)  2/234 (0.85%) 
Gastrointestinal haemorrhage † 1  6/239 (2.51%)  5/234 (2.14%) 
Vomiting † 1  5/239 (2.09%)  2/234 (0.85%) 
Diarrhoea † 1  4/239 (1.67%)  5/234 (2.14%) 
Gastric haemorrhage † 1  4/239 (1.67%)  1/234 (0.43%) 
Abdominal distension † 1  2/239 (0.84%)  0/234 (0.00%) 
Intra-abdominal haemorrhage † 1  3/239 (1.26%)  0/234 (0.00%) 
Abdominal pain upper † 1  2/239 (0.84%)  3/234 (1.28%) 
Lower gastrointestinal haemorrhage † 1  2/239 (0.84%)  0/234 (0.00%) 
Nausea † 1  2/239 (0.84%)  0/234 (0.00%) 
Rectal haemorrhage † 1  2/239 (0.84%)  0/234 (0.00%) 
Constipation † 1  1/239 (0.42%)  1/234 (0.43%) 
Diverticular perforation † 1  1/239 (0.42%)  0/234 (0.00%) 
Duodenitis † 1  1/239 (0.42%)  0/234 (0.00%) 
Gastritis † 1  1/239 (0.42%)  2/234 (0.85%) 
Gastrointestinal fistula † 1  2/239 (0.84%)  0/234 (0.00%) 
Intestinal obstruction † 1  2/239 (0.84%)  3/234 (1.28%) 
Intestinal perforation † 1  2/239 (0.84%)  0/234 (0.00%) 
Oesophagitis † 1  1/239 (0.42%)  0/234 (0.00%) 
Upper gastrointestinal haemorrhage † 1  1/239 (0.42%)  0/234 (0.00%) 
Anal fistula † 1  0/239 (0.00%)  1/234 (0.43%) 
Colitis † 1  0/239 (0.00%)  1/234 (0.43%) 
Gastric ulcer † 1  0/239 (0.00%)  1/234 (0.43%) 
Gastrointestinal toxicity † 1  0/239 (0.00%)  1/234 (0.43%) 
Inguinal hernia strangulated † 1  0/239 (0.00%)  1/234 (0.43%) 
Large intestinal obstruction † 1  0/239 (0.00%)  1/234 (0.43%) 
Peritoneal haemorrhage † 1  0/239 (0.00%)  1/234 (0.43%) 
Small intestinal obstruction † 1  0/239 (0.00%)  2/234 (0.85%) 
Small intestine ulcer † 1  0/239 (0.00%)  1/234 (0.43%) 
Subileus † 1  0/239 (0.00%)  1/234 (0.43%) 
Gastric mucosal lesion † 1  0/239 (0.00%)  1/234 (0.43%) 
Small intestinal haemorrhage † 1  0/239 (0.00%)  1/234 (0.43%) 
General disorders     
Disease progression † 1  3/239 (1.26%)  1/234 (0.43%) 
Face oedema † 1  2/239 (0.84%)  0/234 (0.00%) 
Generalised oedema † 1  2/239 (0.84%)  0/234 (0.00%) 
Multi-organ failure † 1  3/239 (1.26%)  1/234 (0.43%) 
Pyrexia † 1  4/239 (1.67%)  7/234 (2.99%) 
Chest pain † 1  1/239 (0.42%)  1/234 (0.43%) 
Fatigue † 1  2/239 (0.84%)  2/234 (0.85%) 
General physical health deterioration † 1  3/239 (1.26%)  0/234 (0.00%) 
Hernia perforation † 1  1/239 (0.42%)  0/234 (0.00%) 
Hyperthermia † 1  1/239 (0.42%)  0/234 (0.00%) 
Inflammation † 1  1/239 (0.42%)  0/234 (0.00%) 
Asthenia † 1  0/239 (0.00%)  3/234 (1.28%) 
Condition aggravated † 1  1/239 (0.42%)  1/234 (0.43%) 
Malaise † 1  0/239 (0.00%)  2/234 (0.85%) 
Non-cardiac chest pain † 1  0/239 (0.00%)  1/234 (0.43%) 
Hepatobiliary disorders     
Hepatic haemorrhage † 1  3/239 (1.26%)  0/234 (0.00%) 
Jaundice † 1  2/239 (0.84%)  0/234 (0.00%) 
Portal hypertension † 1  2/239 (0.84%)  0/234 (0.00%) 
Cholangitis † 1  1/239 (0.42%)  0/234 (0.00%) 
Bile duct stenosis † 1  0/239 (0.00%)  1/234 (0.43%) 
Drug-induced liver injury † 1  0/239 (0.00%)  1/234 (0.43%) 
Hepatic failure † 1  0/239 (0.00%)  1/234 (0.43%) 
Hepatic function abnormal † 1  0/239 (0.00%)  1/234 (0.43%) 
Hepatic pain † 1  0/239 (0.00%)  1/234 (0.43%) 
Hepatotoxicity † 1  0/239 (0.00%)  1/234 (0.43%) 
Hyperbilirubinaemia † 1  0/239 (0.00%)  1/234 (0.43%) 
Immune system disorders     
Anaphylactic reaction † 1  1/239 (0.42%)  0/234 (0.00%) 
Drug hypersensitivity † 1  1/239 (0.42%)  0/234 (0.00%) 
Infections and infestations     
Sepsis † 1  5/239 (2.09%)  0/234 (0.00%) 
Bacteraemia † 1  2/239 (0.84%)  0/234 (0.00%) 
Pneumonia † 1  2/239 (0.84%)  2/234 (0.85%) 
Lung infection † 1  1/239 (0.42%)  1/234 (0.43%) 
Peritonitis † 1  1/239 (0.42%)  0/234 (0.00%) 
Rhinovirus infection † 1  1/239 (0.42%)  0/234 (0.00%) 
Urinary tract infection † 1  1/239 (0.42%)  3/234 (1.28%) 
Clostridium difficile colitis † 1  0/239 (0.00%)  1/234 (0.43%) 
Cystitis † 1  0/239 (0.00%)  1/234 (0.43%) 
Diverticulitis † 1  0/239 (0.00%)  1/234 (0.43%) 
Infection † 1  0/239 (0.00%)  1/234 (0.43%) 
Bronchitis † 1  0/239 (0.00%)  1/234 (0.43%) 
Enteritis infectious † 1  0/239 (0.00%)  1/234 (0.43%) 
Pneumonia bacterial † 1  0/239 (0.00%)  1/234 (0.43%) 
Injury, poisoning and procedural complications     
Spinal fracture † 1  1/239 (0.42%)  0/234 (0.00%) 
Subdural haematoma † 1  1/239 (0.42%)  0/234 (0.00%) 
Accidental overdose † 1  0/239 (0.00%)  1/234 (0.43%) 
Anastomotic stenosis † 1  0/239 (0.00%)  1/234 (0.43%) 
Hip fracture † 1  0/239 (0.00%)  1/234 (0.43%) 
Wound dehiscence † 1  0/239 (0.00%)  1/234 (0.43%) 
Investigations     
Amylase increased † 1  1/239 (0.42%)  0/234 (0.00%) 
Blood bilirubin increased † 1  1/239 (0.42%)  1/234 (0.43%) 
Neutrophil count decreased † 1  1/239 (0.42%)  0/234 (0.00%) 
Platelet count decreased † 1  1/239 (0.42%)  0/234 (0.00%) 
Metabolism and nutrition disorders     
Hypoglycaemia † 1  2/239 (0.84%)  0/234 (0.00%) 
Hypokalaemia † 1  2/239 (0.84%)  1/234 (0.43%) 
Hypophosphataemia † 1  2/239 (0.84%)  1/234 (0.43%) 
Decreased appetite † 1  1/239 (0.42%)  2/234 (0.85%) 
Dehydration † 1  1/239 (0.42%)  0/234 (0.00%) 
Hyponatraemia † 1  1/239 (0.42%)  1/234 (0.43%) 
Diabetes mellitus † 1  0/239 (0.00%)  2/234 (0.85%) 
Failure to thrive † 1  0/239 (0.00%)  1/234 (0.43%) 
Musculoskeletal and connective tissue disorders     
Back pain † 1  3/239 (1.26%)  1/234 (0.43%) 
Pain in extremity † 1  1/239 (0.42%)  0/234 (0.00%) 
Spinal pain † 1  1/239 (0.42%)  0/234 (0.00%) 
Arthritis † 1  0/239 (0.00%)  1/234 (0.43%) 
Musculoskeletal pain † 1  0/239 (0.00%)  1/234 (0.43%) 
Neoplasms benign, malignant and unspecified (incl cysts and polyps)     
Neoplasm progression † 1  1/239 (0.42%)  0/234 (0.00%) 
Tumour haemorrhage † 1  3/239 (1.26%)  1/234 (0.43%) 
Adenocarcinoma of colon † 1  1/239 (0.42%)  0/234 (0.00%) 
Breast cancer † 1  1/239 (0.42%)  0/234 (0.00%) 
Malignant ascites † 1  1/239 (0.42%)  0/234 (0.00%) 
Malignant neoplasm progression † 1  1/239 (0.42%)  1/234 (0.43%) 
Metastases to liver † 1  1/239 (0.42%)  0/234 (0.00%) 
Tumour necrosis † 1  1/239 (0.42%)  0/234 (0.00%) 
Basal cell carcinoma † 1  0/239 (0.00%)  1/234 (0.43%) 
Gastrointestinal stromal tumour † 1  0/239 (0.00%)  1/234 (0.43%) 
Liver carcinoma ruptured † 1  0/239 (0.00%)  1/234 (0.43%) 
Tumour fistulisation † 1  0/239 (0.00%)  1/234 (0.43%) 
Tumour pain † 1  0/239 (0.00%)  2/234 (0.85%) 
Peritoneal sarcoma † 1  0/239 (0.00%)  1/234 (0.43%) 
Tumour compression † 1  1/239 (0.42%)  0/234 (0.00%) 
Nervous system disorders     
Cognitive disorder † 1  3/239 (1.26%)  1/234 (0.43%) 
Haemorrhage intracranial † 1  2/239 (0.84%)  0/234 (0.00%) 
Transient ischaemic attack † 1  2/239 (0.84%)  0/234 (0.00%) 
Epilepsy † 1  1/239 (0.42%)  0/234 (0.00%) 
Seizure † 1  1/239 (0.42%)  0/234 (0.00%) 
Cerebrovascular accident † 1  0/239 (0.00%)  1/234 (0.43%) 
Coma † 1  1/239 (0.42%)  1/234 (0.43%) 
Facial paresis † 1  0/239 (0.00%)  1/234 (0.43%) 
VIIth nerve paralysis † 1  0/239 (0.00%)  1/234 (0.43%) 
Psychiatric disorders     
Confusional state † 1  0/239 (0.00%)  2/234 (0.85%) 
Insomnia † 1  0/239 (0.00%)  1/234 (0.43%) 
Major depression † 1  0/239 (0.00%)  1/234 (0.43%) 
Renal and urinary disorders     
Acute kidney injury † 1  4/239 (1.67%)  4/234 (1.71%) 
Autoimmune nephritis † 1  1/239 (0.42%)  0/234 (0.00%) 
Calculus urinary † 1  1/239 (0.42%)  0/234 (0.00%) 
Nephrolithiasis † 1  1/239 (0.42%)  0/234 (0.00%) 
Reproductive system and breast disorders     
Uterine haemorrhage † 1  1/239 (0.42%)  0/234 (0.00%) 
Benign prostatic hyperplasia † 1  0/239 (0.00%)  1/234 (0.43%) 
Respiratory, thoracic and mediastinal disorders     
Dyspnoea † 1  3/239 (1.26%)  5/234 (2.14%) 
Pleural effusion † 1  3/239 (1.26%)  1/234 (0.43%) 
Pulmonary oedema † 1  2/239 (0.84%)  0/234 (0.00%) 
Interstitial lung disease † 1  1/239 (0.42%)  0/234 (0.00%) 
Pneumonitis † 1  1/239 (0.42%)  0/234 (0.00%) 
Respiratory distress † 1  1/239 (0.42%)  0/234 (0.00%) 
Respiratory failure † 1  1/239 (0.42%)  2/234 (0.85%) 
Bronchitis chronic † 1  0/239 (0.00%)  1/234 (0.43%) 
Epistaxis † 1  0/239 (0.00%)  1/234 (0.43%) 
Pneumothorax † 1  0/239 (0.00%)  1/234 (0.43%) 
Pulmonary embolism † 1  0/239 (0.00%)  2/234 (0.85%) 
Skin and subcutaneous tissue disorders     
Purpura † 1  1/239 (0.42%)  0/234 (0.00%) 
Rash † 1  0/239 (0.00%)  1/234 (0.43%) 
Rash morbilliform † 1  0/239 (0.00%)  1/234 (0.43%) 
Skin toxicity † 1  0/239 (0.00%)  1/234 (0.43%) 
Vascular disorders     
Hypotension † 1  1/239 (0.42%)  1/234 (0.43%) 
Deep vein thrombosis † 1  0/239 (0.00%)  1/234 (0.43%) 
Hypertension † 1  0/239 (0.00%)  3/234 (1.28%) 
1
Term from vocabulary, MedDRA (18.1)
†
Indicates events were collected by systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Avapritinib Regorafinib
Affected / at Risk (%) Affected / at Risk (%)
Total   218/239 (91.21%)   224/234 (95.73%) 
Eye disorders     
Periorbital oedema † 1  68/239 (28.45%)  0/234 (0.00%) 
Lacrimation increased † 1  45/239 (18.83%)  0/234 (0.00%) 
Eyelid oedema † 1  35/239 (14.64%)  0/234 (0.00%) 
Gastrointestinal disorders     
Dyspepsia † 1  21/239 (8.79%)  16/234 (6.84%) 
Gastrooesophageal reflux disease † 1  16/239 (6.69%)  7/234 (2.99%) 
Dry mouth † 1  7/239 (2.93%)  15/234 (6.41%) 
Stomatitis † 1  7/239 (2.93%)  40/234 (17.09%) 
General disorders     
Oedema peripheral † 1  55/239 (23.01%)  13/234 (5.56%) 
Mucosal inflammation † 1  3/239 (1.26%)  28/234 (11.97%) 
Infections and infestations     
Conjunctivitis † 1  12/239 (5.02%)  0/234 (0.00%) 
Investigations     
White blood cell count decreased † 1  42/239 (17.57%)  7/234 (2.99%) 
Aspartate aminotransferase increased † 1  33/239 (13.81%)  33/234 (14.10%) 
Weight decreased † 1  24/239 (10.04%)  53/234 (22.65%) 
Blood creatinine increased † 1  21/239 (8.79%)  12/234 (5.13%) 
Weight increased † 1  18/239 (7.53%)  4/234 (1.71%) 
Alanine aminotransferase increased † 1  15/239 (6.28%)  29/234 (12.39%) 
Blood alkaline phosphatase increased † 1  6/239 (2.51%)  13/234 (5.56%) 
Metabolism and nutrition disorders     
Hypocalcaemia † 1  15/239 (6.28%)  11/234 (4.70%) 
Hypoalbuminaemia † 1  12/239 (5.02%)  16/234 (6.84%) 
Hypomagnesaemia † 1  11/239 (4.60%)  12/234 (5.13%) 
Musculoskeletal and connective tissue disorders     
Arthralgia † 1  9/239 (3.77%)  16/234 (6.84%) 
Muscle spasms † 1  6/239 (2.51%)  25/234 (10.68%) 
Myalgia † 1  7/239 (2.93%)  22/234 (9.40%) 
Nervous system disorders     
Dizziness † 1  32/239 (13.39%)  19/234 (8.12%) 
Memory impairment † 1  31/239 (12.97%)  5/234 (2.14%) 
Headache † 1  29/239 (12.13%)  43/234 (18.38%) 
Dysgeusia † 1  20/239 (8.37%)  11/234 (4.70%) 
Psychiatric disorders     
Anxiety † 1  12/239 (5.02%)  3/234 (1.28%) 
Respiratory, thoracic and mediastinal disorders     
Dysphonia † 1  8/239 (3.35%)  71/234 (30.34%) 
Cough † 1  17/239 (7.11%)  13/234 (5.56%) 
Skin and subcutaneous tissue disorders     
Hair colour changes † 1  33/239 (13.81%)  2/234 (0.85%) 
Alopecia † 1  24/239 (10.04%)  38/234 (16.24%) 
Pruritus † 1  11/239 (4.60%)  15/234 (6.41%) 
Dry skin † 1  10/239 (4.18%)  18/234 (7.69%) 
Rash maculo-papular † 1  7/239 (2.93%)  14/234 (5.98%) 
Palmar-plantar erythrodysaesthesia syndrome † 1  4/239 (1.67%)  143/234 (61.11%) 
1
Term from vocabulary, MedDRA (18.1)
†
Indicates events were collected by systematic assessment
[Not Specified]
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
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Name/Title: Medical Information Call Center
Organization: Blueprint Medicines
Phone: 1-888-258-7768
EMail: medinfo@blueprintmedicines.com
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Blueprint Medicines Corporation
ClinicalTrials.gov Identifier: NCT03465722    
Other Study ID Numbers: BLU-285-1303
First Submitted: March 7, 2018
First Posted: March 14, 2018
Results First Submitted: March 5, 2021
Results First Posted: May 14, 2021
Last Update Posted: October 6, 2022