A Phase III, Safety, Tolerability and Efficacy of Combination Treatment of BL-8040 and G-GSF as Compared to Placebo and G-CSF for the Mobilization of Hematopoietic Stem Cells for Autologous TransplantatIon in Subjects With MM (GENESIS)

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ClinicalTrials.gov Identifier: NCT03246529

Recruitment Status : Active, not recruiting

First Posted : August 11, 2017

Results First Posted : November 7, 2023

Last Update Posted : February 8, 2024

View this study on the modernized ClinicalTrials.gov

Sponsor:

Information provided by (Responsible Party):

BioLineRx, Ltd.

Study Type	Interventional
Study Design	Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Triple (Participant, Care Provider, Investigator); Primary Purpose: Treatment
Condition	Multiple Myeloma
Interventions	Drug: BL-8040 1.25 mg/kg + G-CSF Drug: Placebo +G-CSF
Enrollment	180

Participant Flow

Recruitment Details	A total of 180 subjects signed the Informed Consent Form (ICF) and screened to the study. A total of 136 subjects received at least one dose of G-CSF. A total of 134 subjects were treated with G-CSF and with BL-8040 or Placebo.
Pre-assignment Details


Arm/Group Title	BL-8040 + G-CSF Part 1	BL-8040 + G-CSF Part 2	Placebo + G-CSF Part 2
Arm/Group Description	Part 1 (lead-in) period. G-CSF give...	Part 2: randomized, double-blinded,...	Part 2: randomized, double-blinded,...
Arm/Group Description	Part 1 (lead-in) period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Part 2: randomized, double-blinded, placebo controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Part 2: randomized, double-blinded, placebo controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. Placebo s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.
Period Title: Core Study (Until 100 d Post-transplant)
Started	12	80	42
Post Transplantation Analysis Set	12	77	37
Completed	12	74	34
Not Completed	0	6	8
Reason Not Completed
Adverse Event	0	2	0
Lost to Follow-up	0	1	1
Other Reasons	0	2	2
Physician Decision	0	0	1
Withdrawal by Subject	0	1	4
Period Title: Follow-up Period at 1 Year FU Completion
Started	12	73 ^[1]	34
Completed ^[2]	9	66	32
Not Completed	3	7	2
Reason Not Completed
Disease progression	2	1	1
Lost to Follow-up	1	3	0
Withdrawal by Subject	0	3	0
Death	0	0	1
[1] One (1) subject early terminated the study following the core study period and before the follow up (FU) [2] Long Term FU is ongoing. Numbers are of the subject ongoing in the study.

Baseline Characteristics

Arm/Group Title		BL-8040 + G-CSF Part 1	BL-8040 + G-CSF Part 2	Placebo + G-CSF Part 2	Total
Arm/Group Description		Part 1 (lead-in) period. G-CSF give...	Part 2: randomized, double-blinded,...	Part 2: randomized, double-blinded,...	Total of all reporting groups
Arm/Group Description		Part 1 (lead-in) period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Part 2: randomized, double-blinded, placebo controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Part 2: randomized, double-blinded, placebo controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. Placebo s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Total of all reporting groups
Overall Number of Baseline Participants		12	80	42	134
Baseline Analysis Population Description		...
Baseline Analysis Population Description		Part 1: Full analysis set - all enrolled subjects. Part 2: ITT analysis set: all randomized subjects. According to the treatment group to which they were originally randomized.
Age, Continuous Mean (Standard Deviation) Unit of measure: Years
	Number Analyzed	12 participants	80 participants	42 participants	134 participants
		63.3 (4.8)	60.4 (9.4)	59.2 (9.6)	60.5 (9.1)
Sex: Female, Male Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	12 participants	80 participants	42 participants	134 participants
	Female	5 41.7%	25 31.3%	18 42.9%	48 35.8%
	Male	7 58.3%	55 68.8%	24 57.1%	86 64.2%
Race/Ethnicity, Customized Measure Type: Count of Participants Unit of measure: Participants	Number Analyzed	12 participants	80 participants	42 participants	134 participants
African American		2 16.7%	8 10.0%	2 4.8%	12 9.0%
Caucasian		9 75.0%	65 81.3%	40 95.2%	114 85.1%
Not reported		1 8.3%	0 0.0%	0 0.0%	1 0.7%
African		0 0.0%	1 1.3%	0 0.0%	1 0.7%
Asian		0 0.0%	2 2.5%	0 0.0%	2 1.5%
Hispanic origin		0 0.0%	1 1.3%	0 0.0%	1 0.7%
Unknown		0 0.0%	2 2.5%	0 0.0%	2 1.5%
Not Specified		0 0.0%	1 1.3%	0 0.0%	1 0.7%
Region of Enrollment Measure Type: Count of Participants Unit of measure: Participants	Number Analyzed	12 participants	80 participants	42 participants	134 participants
Hungary		0 0.0%	10 12.5%	6 14.3%	16 11.9%
United States		12 100.0%	52 65.0%	26 61.9%	90 67.2%
Italy		0 0.0%	9 11.3%	8 19.0%	17 12.7%
Germany		0 0.0%	3 3.8%	0 0.0%	3 2.2%
Spain		0 0.0%	6 7.5%	2 4.8%	8 6.0%
Treatment Indicated for Multiple Myeloma Before Study Initiation ^[1] Measure Type: Count of Participants Unit of measure: Participants	Number Analyzed	12 participants	80 participants	42 participants	134 participants
Bortezomib		7 58.3%	54 67.5%	28 66.7%	89 66.4%
Cisplatin		0 0.0%	0 0.0%	1 2.4%	1 0.7%
Cyclophosphamide		0 0.0%	5 6.3%	4 9.5%	9 6.7%
Daratumumab 0 - 1 2.4%		0 0.0%	0 0.0%	1 2.4%	1 0.7%
Doxorubicin		0 0.0%	0 0.0%	1 2.4%	1 0.7%
Etoposide		0 0.0%	0 0.0%	1 2.4%	1 0.7%
Ixazomib		0 0.0%	1 1.3%	0 0.0%	1 0.7%
Kyprolis		6 50.0%	2 2.5%	1 2.4%	9 6.7%
Lenalidomide		12 100.0%	57 71.3%	28 66.7%	97 72.4%
Melphalan		0 0.0%	0 0.0%	1 2.4%	1 0.7%
Pomalidomide		1 8.3%	0 0.0%	1 2.4%	2 1.5%
Thalidomide		0 0.0%	18 22.5%	12 28.6%	30 22.4%
		[1] Measure Description: Treatment Indicated for Multiple Myeloma Before Study Initiation
IMWG Classification at Screening ^[1] Measure Type: Count of Participants Unit of measure: Participants
	Number Analyzed	12 participants	80 participants	42 participants	134 participants
	PR (Partial Response)	0 0.0%	31 38.8%	10 23.8%	41 30.6%
	VGPR (Very Good Partial Response)	9 75.0%	33 41.3%	23 54.8%	65 48.5%
	CR (Complete Response)	2 16.7%	12 15.0%	7 16.7%	21 15.7%
	sCR (Stringent Complete Response)	1 8.3%	4 5.0%	2 4.8%	7 5.2%
		[1] Measure Description: Response criteria for the purposes of the study was defined in accordance with the International Myeloma Working Group (IMWG) Uniform Response Criteria.
Months from MM Diagnosis to Study Consent Mean (Standard Deviation) Unit of measure: Months
	Number Analyzed	12 participants	80 participants	42 participants	134 participants
		4.6 (0.9)	6.5 (11.1)	6.4 (6.9)	6.3 (9.4)

Outcome Measures

1.Primary Outcome


Title	Percentage of Subjects Mobilizing ≥6 × 10^6 CD34+ Cells/kg With up to 2 Apheresis Sessions
Description	Percentage of subjects mobilizing ≥6 × 10^6 CD34+ cells/kg ...
Description	Percentage of subjects mobilizing ≥6 × 10^6 CD34+ cells/kg with up to 2 apheresis sessions in preparation for autologous hematopoetic cell transplantation (auto-HCT) after treatment with G-CSF + single administration of BL-8040/placebo. Based on central laboratory data.
Time Frame	From first day of study treatment (G-CSF) until day of second apheresis which was planned to occur on Day 6

Outcome Measure Data

Analysis Population Description

FAS for Part 1 (Part 1 of the study assessed the mobilization using local lab only), ITT for Part 2


Arm/Group Title		BL-8040 + G-CSF Part 1	BL-8040 + G-CSF Part 2	Placebo + G-CSF Part 2
Arm/Group Description:		Part 1 (lead-in) period. G-CSF give...	Part 2: randomized, double-blinded,...	Part 2: randomized, double-blinded,...
Arm/Group Description:		Part 1 (lead-in) period. G-CSF given one time daily s.c. for 5-8 days (Days 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. Placebo s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.
Overall Number of Participants Analyzed		12	80	42
Measure Type: Number Unit of Measure: Percentage of responders
Central Lab	Number Analyzed	0 participants	80 participants	42 participants
Central Lab			70.0	14.3
Local Lab	Number Analyzed	12 participants	80 participants	42 participants
Local Lab		91.7	92.5	26.2

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	BL-8040 + G-CSF Part 2, Placebo + G-CSF Part 2
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	<0.0001
	Comments	[Not Specified]
	Method	Cochran-Mantel-Haenszel
	Comments	Stratified CMH test (by response status and baseline platelet count),

2.Secondary Outcome


Title	Percentage of Subjects Mobilizing ≥2 × 10^6 CD34+ Cells/kg in 1 Apheresis Session
Description	Percentage of subjects mobilizing ≥2 × 10^6 CD34+ cells/kg ...
Description	Percentage of subjects mobilizing ≥2 × 10^6 CD34+ cells/kg in 1 apheresis session after treatment with G-CSF + single administration of BL-8040/ placebo.
Time Frame	From first day of study treatment (G-CSF) until day of first apheresis which was planned to occur on Day 5

Outcome Measure Data

Analysis Population Description

FAS for Part 1 (Part 1 of the study assessed the mobilization using local lab only), ITT for Part 2


Arm/Group Title		BL-8040 + G-CSF Part 1	BL-8040 + G-CSF Part 2	Placebo + G-CSF Part 2
Arm/Group Description:		Part 1 (lead-in) period. G-CSF give...	Part 2: randomized, double-blinded,...	Part 2: randomized, double-blinded,...
Arm/Group Description:		Part 1 (lead-in) period. G-CSF given one time daily s.c. for 5-8 days (Days 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed	Part 2: randomized, double-blinded, placebo controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Part 2: randomized, double-blinded, placebo controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. Placebo s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.
Overall Number of Participants Analyzed		12	80	42
Measure Type: Number Unit of Measure: Percentage of responders
Central Lab	Number Analyzed	0 participants	80 participants	42 participants
Central Lab			87.5	47.6
Local Lab	Number Analyzed	12 participants	80 participants	42 participants
Local Lab		100	96.3	64.3

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	BL-8040 + G-CSF Part 2, Placebo + G-CSF Part 2
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	<0.0001
	Comments	[Not Specified]
	Method	Cochran-Mantel-Haenszel
	Comments	[Not Specified]

3.Secondary Outcome


Title	Percentage of Subjects Mobilizing ≥6 × 10^6 CD34+ Cells/kg in 1 Apheresis Session
Description	Percentage of subjects mobilizing ≥6 × 10^6 CD34+ cells/kg ...
Description	Percentage of subjects mobilizing ≥6 × 10^6 CD34+ cells/kg in 1 apheresis session after treatment with G-CSF + single administration of BL-8040/ placebo.
Time Frame	From first day of study treatment (G-CSF) until day of first apheresis which was planned to occur on Day 5

Outcome Measure Data

Analysis Population Description

FAS for Part 1 (Part 1 of the study assessed the mobilization using local lab only), ITT for Part 2


Arm/Group Title		BL-8040 + G-CSF Part 1	BL-8040 + G-CSF Part 2	Placebo + G-CSF Part 2
Arm/Group Description:		Part 1 (lead-in) period. G-CSF give...	Part 2: randomized, double-blinded,...	Part 2: randomized, double-blinded,...
Arm/Group Description:		Part 1 (lead-in) period. G-CSF given one time daily s.c. for 5-8 days (Days 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4. First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. Placebo s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.
Overall Number of Participants Analyzed		12	80	42
Measure Type: Number Unit of Measure: Percentage of responders
Central Lab	Number Analyzed	0 participants	80 participants	42 participants
Central Lab			67.5	4.8
Local Lab	Number Analyzed	12 participants	80 participants	42 participants
Local Lab		75	88.8	9.5

Statistical Analysis 1

Statistical Analysis Overview	Comparison Group Selection	BL-8040 + G-CSF Part 2, Placebo + G-CSF Part 2
	Comments	[Not Specified]
	Type of Statistical Test	Superiority
	Comments	[Not Specified]

Statistical Test of Hypothesis	P-Value	<0.0001
	Comments	[Not Specified]
	Method	Cochran-Mantel-Haenszel
	Comments	[Not Specified]

4.Secondary Outcome


Title	Time to Neutrophil Engraftment, After Auto-HCT
Description	Time to neutrophil engraftment after auto-HCT, where engraftment was d...
Description	Time to neutrophil engraftment after auto-HCT, where engraftment was defined as absolute neutrophil count (ANC) ≥0.5 × 10^9/L for 3 days or ≥1.0 × 10^9/L for 1 day following the conditioning regimen associated nadir.
Time Frame	End of engraftment period, which was defined as 29 days post transplantation

Outcome Measure Data

Analysis Population Description

Post Transplantation analysis set (all subjects treated, with at least 1 apheresis session, and who underwent transplantation)


Arm/Group Title	BL-8040 + G-CSF Part 1	BL-8040 + G-CSF Part 2	Placebo + G-CSF Part 2
Arm/Group Description:	Part 1 (lead-in) period. G-CSF give...	Part 2: randomized, double-blinded,...	Part 2: randomized, double-blinded,...
Arm/Group Description:	Part 1 (lead-in) period. G-CSF given one time daily s.c. for 5-8 days (Days 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. Placebo s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.
Overall Number of Participants Analyzed	12	77	37
Median (95% Confidence Interval) Unit of Measure: Days
	12 (11 to 12)	12 (11 to 12)	12 (11 to 12)

5.Secondary Outcome


Title	Time to Platelet Engraftment, After Auto-HCT
Description	Time to platelet engraftment, after auto-HCT, where engraftment was de...
Description	Time to platelet engraftment, after auto-HCT, where engraftment was defined as the first of 3 consecutive measurements of platelet count ≥20 × 10^9/L without platelet transfusion support for 7 days following the conditioning regimen associated nadir.
Time Frame	End of engraftment period, which was defined as 29 days post transplantation

Outcome Measure Data

Analysis Population Description

Post Transplantation analysis set (all subjects treated, with at least 1 apheresis session, and who underwent transplantation)


Arm/Group Title	BL-8040 + G-CSF Part 1	BL-8040 + G-CSF Part 2	Placebo + G-CSF Part 2
Arm/Group Description:	Part 1 (lead-in) period. G-CSF give...	Part 2: randomized, double-blinded,...	Part 2: randomized, double-blinded,...
Arm/Group Description:	Part 1 (lead-in) period. G-CSF given one time daily s.c. for 5-8 days (Days 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed	Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. Placebo s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.
Overall Number of Participants Analyzed	12	77	37
Median (95% Confidence Interval) Unit of Measure: Point estimate (days)
	17 (16.5 to 19.5)	18 (17 to 19)	17 (17 to 18)

6.Secondary Outcome


Title	Subjects With Graft Durability at 100 Days Post Transplant/ Early Termination
Description	Subjects achieving graft durability were defined as meeting the follow...
Description	Subjects achieving graft durability were defined as meeting the following 2 criteria: Platelet count ≥50 × 10^9/L without transfusion for at least 2 weeks. Hemoglobin level ≥10 g/dL with no erythropoietin support or transfusions for at least 1 month. This analysis was performed in part 2 of the study only.
Time Frame	Day 100 Post-Transplantation (± 7 days)

Outcome Measure Data

Analysis Population Description

Post Transplantation analysis set (all randomized subjects treated, with at least 1 apheresis session, and who underwent transplantation).

Graft durability was not assessed during Part 1 of the study.


Arm/Group Title	BL-8040 + G-CSF Part 2	Placebo + G-CSF Part 2
Arm/Group Description:	Part 2: randomized, double-blinded,...	Part 2: randomized, double-blinded,...
Arm/Group Description:	Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.	Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. Placebo s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.
Overall Number of Participants Analyzed	77	37
Measure Type: Number Unit of Measure: Percentage of responders
	92.2	91.9

Adverse Events

Time Frame	All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo).
Adverse Event Reporting Description	All AEs were collected after subjects signed the ICF and up to 30 days from the last dose of study therapy (G-CSF+BL-8040/Placebo). AE were followed by the investigators until their satisfactory resolution or stabilization. AE were recorded and graded according to the latest version of the NCI-CTCAE. All-cause mortality was reported across the entire study duration, including the 5 years follow-up phase.

Arm/Group Title	BL-8040 + G-CSF Part 1		BL-8040 + G-CSF (Part 2)		Placebo + G-CSF (Part 2)
Arm/Group Description	Part 1 (lead-in) period. G-CSF give...		Part 2: randomized, double-blinded,...		Part 2 (randomized, double-blinded,...
Arm/Group Description	Part 1 (lead-in) period. G-CSF given one time daily s.c. for 5-8 days (Days 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.		Part 2: randomized, double-blinded, placebo-controlled period. G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. BL-8040 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.		Part 2 (randomized, double-blinded, placebo-controlled period). G-CSF given one time daily s.c. for 5-8 days (Day 1 up to 8) at doses of approximately 10 µg/kg/day but not more than 15 µg/kg/day. Placebo 1.25 mg/kg s.c. on Day 4 (and Day 6, if needed). First apheresis on Day 5 with up to 3 additional procedures possible until Day 8, as needed.
All-Cause Mortality
	BL-8040 + G-CSF Part 1		BL-8040 + G-CSF (Part 2)		Placebo + G-CSF (Part 2)
	Affected / at Risk (%)		Affected / at Risk (%)		Affected / at Risk (%)
Total	0/12 (0.00%)		2/80 (2.50%)		1/42 (2.38%)
Serious Adverse Events Serious Adverse Events
	BL-8040 + G-CSF Part 1		BL-8040 + G-CSF (Part 2)		Placebo + G-CSF (Part 2)
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	1/12 (8.33%)		7/80 (8.75%)		0/42 (0.00%)
Gastrointestinal disorders
Hypokalaemia ^* 1	1/12 (8.33%)	1	0/80 (0.00%)	0	0/42 (0.00%)	0
Vomiting ^* 1	1/12 (8.33%)	1	0/80 (0.00%)	0	0/42 (0.00%)	0
General disorders
Injection site reaction ^* 1	0/12 (0.00%)	0	1/80 (1.25%)	1	0/42 (0.00%)	0
Immune system disorders
Hypersensitivity ^* 1	0/12 (0.00%)	0	1/80 (1.25%)	1	0/42 (0.00%)	0
Infections and infestations
Bacteremia ^* 1	0/12 (0.00%)	0	1/80 (1.25%)	1	0/42 (0.00%)	0
Injection site cellulitis ^* 1	0/12 (0.00%)	0	1/80 (1.25%)	1	0/42 (0.00%)	0
Investigations
Platelet count decreased ^* 1	0/12 (0.00%)	0	1/80 (1.25%)	1	0/42 (0.00%)	0
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Bladder neoplasm ^* 1	0/12 (0.00%)	0	1/80 (1.25%)	1	0/42 (0.00%)	0
Respiratory, thoracic and mediastinal disorders
Hypoxia ^* 1	0/12 (0.00%)	0	1/80 (1.25%)	1	0/42 (0.00%)	0
1 Term from vocabulary, MedDRA 20.1 * Indicates events were collected by non-systematic assessment
Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events	5%
	BL-8040 + G-CSF Part 1		BL-8040 + G-CSF (Part 2)		Placebo + G-CSF (Part 2)
	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events	Affected / at Risk (%)	# Events
Total	12/12 (100.00%)		77/80 (96.25%)		35/42 (83.33%)
Blood and lymphatic system disorders
Anaemia ^* 1	1/12 (8.33%)	1	0/80 (0.00%)	0	0/42 (0.00%)	0
Ear and labyrinth disorders
Ear discomfort ^* 1	1/12 (8.33%)	1	0/80 (0.00%)	0	0/42 (0.00%)	0
Auricular swelling ^* 1	1/12 (8.33%)	1	0/80 (0.00%)	0	0/42 (0.00%)	0
Gastrointestinal disorders
Constipation ^* 1	1/12 (8.33%)	1	0/80 (0.00%)	0	0/42 (0.00%)	0
Dyspepsia ^* 1	1/12 (8.33%)	1	0/80 (0.00%)	0	0/42 (0.00%)	0
Nausea ^* 1	2/12 (16.67%)	2	11/80 (13.75%)	11	5/42 (11.90%)	5
Vomiting ^* 1	1/12 (8.33%)	1	6/80 (7.50%)	6	2/42 (4.76%)	2
General disorders
Catheter site bruise ^* 1	1/12 (8.33%)	1	0/80 (0.00%)	0	0/42 (0.00%)	0
Catheter site pain ^* 1	1/12 (8.33%)	1	9/80 (11.25%)	9	6/42 (14.29%)	6
Chest pain ^* 1	1/12 (8.33%)	1	2/80 (2.50%)	3	4/42 (9.52%)	5
Injection site erythema ^* 1	3/12 (25.00%)	3	22/80 (27.50%)	22	0/42 (0.00%)	0
Injection site nodule ^* 1	1/12 (8.33%)	1	0/80 (0.00%)	0	0/42 (0.00%)	0
Injection site pain ^* 1	9/12 (75.00%)	14	40/80 (50.00%)	47	2/42 (4.76%)	3
Injection site pruritus ^* 1	5/12 (41.67%)	6	17/80 (21.25%)	17	0/42 (0.00%)	0
Injection site reaction ^* 1	0/12 (0.00%)	0	5/80 (6.25%)	5	0/42 (0.00%)	0
Injection site urticaria ^* 1	1/12 (8.33%)	1	0/80 (0.00%)	0	0/42 (0.00%)	0
Oedema peripheral ^* 1	1/12 (8.33%)	1	3/80 (3.75%)	3	3/42 (7.14%)	4
Pyrexia ^* 1	2/12 (16.67%)	2	0/80 (0.00%)	0	0/42 (0.00%)	0
Immune system disorders
Hypersensitivity ^* 1	0/12 (0.00%)	0	6/80 (7.50%)	6	0/42 (0.00%)	0
Investigations
Electrocardiogram QT prolonged ^* 1	1/12 (8.33%)	1	0/80 (0.00%)	0	0/42 (0.00%)	0
White blood cell count increased ^* 1	1/12 (8.33%)	1	0/80 (0.00%)	0	0/42 (0.00%)	0
Metabolism and nutrition disorders
Hypocalcaemia ^* 1	3/12 (25.00%)	3	0/80 (0.00%)	0	0/42 (0.00%)	0
Hypokalaemia ^* 1	2/12 (16.67%)	2	11/80 (13.75%)	11	5/42 (11.90%)	5
Hypomagnesaemia ^* 1	0/12 (0.00%)	0	1/80 (1.25%)	1	4/42 (9.52%)	5
Musculoskeletal and connective tissue disorders
Arthralgia ^* 1	1/12 (8.33%)	1	5/80 (6.25%)	6	4/42 (9.52%)	7
Back pain ^* 1	4/12 (33.33%)	4	14/80 (17.50%)	17	6/42 (14.29%)	10
Musculoskeletal chest pain ^* 1	0/12 (0.00%)	0	2/80 (2.50%)	2	3/42 (7.14%)	4
Bone pain ^* 1	2/12 (16.67%)	3	14/80 (17.50%)	15	13/42 (30.95%)	18
Spinal pain ^* 1	1/12 (8.33%)	1	0/80 (0.00%)	0	0/42 (0.00%)	0
Muscle spasms ^* 1	1/12 (8.33%)	1	0/80 (0.00%)	0	0/42 (0.00%)	0
Nervous system disorders
Paraesthesia ^* 1	1/12 (8.33%)	1	8/80 (10.00%)	11	4/42 (9.52%)	5
Headache ^* 1	0/12 (0.00%)	0	5/80 (6.25%)	5	4/42 (9.52%)	5
Hypoaesthesia ^* 1	0/12 (0.00%)	0	5/80 (6.25%)	6	3/42 (7.14%)	7
Respiratory, thoracic and mediastinal disorders
Dysphonia ^* 1	1/12 (8.33%)	1	0/80 (0.00%)	0	0/42 (0.00%)	0
Paranasal sinus discomfort ^* 1	1/12 (8.33%)	1	0/80 (0.00%)	0	0/42 (0.00%)	0
Dyspnoea ^* 1	1/12 (8.33%)	1	0/80 (0.00%)	0	0/42 (0.00%)	0
Skin and subcutaneous tissue disorders
Erythema ^* 1	2/12 (16.67%)	2	9/80 (11.25%)	15	0/42 (0.00%)	0
Pruritus ^* 1	8/12 (66.67%)	8	26/80 (32.50%)	28	0/42 (0.00%)	0
Rash ^* 1	2/12 (16.67%)	2	7/80 (8.75%)	7	0/42 (0.00%)	0
Rash maculo-papular ^* 1	1/12 (8.33%)	1	0/80 (0.00%)	0	0/42 (0.00%)	0
Urticaria ^* 1	3/12 (25.00%)	3	10/80 (12.50%)	10	0/42 (0.00%)	0
Dermatitis exfoliative ^* 1	0/12 (0.00%)	0	6/80 (7.50%)	6	0/42 (0.00%)	0
Vascular disorders
Flushing ^* 1	4/12 (33.33%)	4	0/80 (0.00%)	0	0/42 (0.00%)	0
Hypertension ^* 1	1/12 (8.33%)	1	0/80 (0.00%)	0	0/42 (0.00%)	0
1 Term from vocabulary, MedDRA 20.1 * Indicates events were collected by non-systematic assessment

Limitations and Caveats

[Not Specified]

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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.

The restrictions are as per the signed agreement with each PI and institution.

Results Point of Contact

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Name/Title:	Dr. Tami Rachmilewitz/ Chief Medical Officer
Organization:	Biolinerx
Phone:	+972-8-6429100 ext 170
EMail:	tamir@biolinerx.com

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

Crees ZD, Stockerl-Goldstein K, Vainstein A, Chen H, DiPersio JF. GENESIS: Phase III trial evaluating BL-8040 + G-CSF to mobilize hematopoietic cells for autologous transplant in myeloma. Future Oncol. 2019 Nov;15(31):3555-3563. doi: 10.2217/fon-2019-0380. Epub 2019 Sep 9.

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Responsible Party:	BioLineRx, Ltd.
ClinicalTrials.gov Identifier:	NCT03246529
Other Study ID Numbers:	BL-8040.SCM.301
First Submitted:	August 2, 2017
First Posted:	August 11, 2017
Results First Submitted:	April 27, 2023
Results First Posted:	November 7, 2023
Last Update Posted:	February 8, 2024

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