Trial Outcomes & Findings for Safety and Efficacy of BL-8040 for the Mobilization of Donor Hematopoietic Stem Cells and Allogeneic Transplantation in Patients With Advanced Hematological Malignancies (NCT NCT02639559)

NCT ID: NCT02639559

Last Updated: 2023-11-07

Results Overview

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

50 participants

Primary outcome timeframe

Up to Day 2

Results posted on

2023-11-07

Participant Flow

Participant milestones

Participant milestones
Measure	Arm 1: Donors -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10\^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10\^6 CD34+ cells/kg and at least ≥ 2 x 10\^6 CD34+ cells/kg from the combined collections.	Arm 2: Recipients -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0
Overall Study STARTED	25	25
Overall Study COMPLETED	24	22
Overall Study NOT COMPLETED	1	3

Reasons for withdrawal

Reasons for withdrawal
Measure	Arm 1: Donors -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10\^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10\^6 CD34+ cells/kg and at least ≥ 2 x 10\^6 CD34+ cells/kg from the combined collections.	Arm 2: Recipients -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0
Overall Study Mobilization failure	0	3
Overall Study Issues with vascular acess	1	0

Baseline Characteristics

Safety and Efficacy of BL-8040 for the Mobilization of Donor Hematopoietic Stem Cells and Allogeneic Transplantation in Patients With Advanced Hematological Malignancies

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Arm 1: Donors n=25 Participants -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10\^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10\^6 CD34+ cells/kg and at least ≥ 2 x 10\^6 CD34+ cells/kg from the combined collections.	Arm 2: Recipients n=25 Participants -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0	Total n=50 Participants Total of all reporting groups
Age, Continuous	55 years n=5 Participants	58 years n=7 Participants	55 years n=5 Participants
Sex: Female, Male Female	7 Participants n=5 Participants	10 Participants n=7 Participants	17 Participants n=5 Participants
Sex: Female, Male Male	18 Participants n=5 Participants	15 Participants n=7 Participants	33 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	2 Participants n=5 Participants	2 Participants n=7 Participants	4 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	21 Participants n=5 Participants	23 Participants n=7 Participants	44 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	2 Participants n=5 Participants	0 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants	1 Participants n=7 Participants	2 Participants n=5 Participants
Race (NIH/OMB) White	20 Participants n=5 Participants	22 Participants n=7 Participants	42 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants	0 Participants n=7 Participants	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	4 Participants n=5 Participants	2 Participants n=7 Participants	6 Participants n=5 Participants
Region of Enrollment United States	25 participants n=5 Participants	25 participants n=7 Participants	50 participants n=5 Participants

PRIMARY outcome

Timeframe: Up to Day 2

Population: This outcome measure is for Arm 1 - Donors only.

Outcome measures

Outcome measures
Measure	Arm 1: Donors n=24 Participants -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10\^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10\^6 CD34+ cells/kg and at least ≥ 2 x 10\^6 CD34+ cells/kg from the combined collections.	Arm 2: Recipients -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0
Number of Donors That Mobilize ≥ 2 x 10^6 CD34+ Cells/kg of Recipients Weight After a Single Injection of BL-8040 After no More Than Two Leukapheresis Collections (Arm 1 - Donors Only)	22 Participants	—

SECONDARY outcome

Timeframe: Up to 5 years

Population: This outcome measure is for Donor Arm only.

-Adverse events will be graded according to the NCI CTCAE version 4.03

Outcome measures

Outcome measures
Measure	Arm 1: Donors n=25 Participants -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10\^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10\^6 CD34+ cells/kg and at least ≥ 2 x 10\^6 CD34+ cells/kg from the combined collections.	Arm 2: Recipients -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 paresthesia	3 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 insomnia	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 anemia	17 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 sinus bradycardia	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 sinus tachycardia	2 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 eye pain	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 watering eyes	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 nausea	5 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 oral dysesthesia	6 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 vomiting	2 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 chills	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 fatigue	3 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 fever	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 infusion related reaction	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 injection site reaction	20 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 localized edema	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 pain	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 vibration sensation	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 allergic reaction	20 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 papulopustular rash	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 bruising	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 hemoglobin increased	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 lymphocyte count decreased	6 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 3 lymphocyte count decreased	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 lymphocyte count increased	22 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 platelet count decreased	22 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 weight loss	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 white blood cell decreased	2 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 A1c increased	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 hyperglycemia	2 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 hyperkalemia	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 hypocalcemia	10 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 hypomagnesemia	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 arthralgia	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 bone pain	2 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 foot pain	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 numbness in arms	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 pain in extremity	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 dysesthesia	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 dizziness	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 dysgeusia	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 headache	3 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 dyspnea	1 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 hypertension	7 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 3 hypertension	4 Participants	—
Safety and Tolerability of BL-8040 in Healthy Donors as Measured by Number and Grade of Adverse Events (Arm 1 Donors Only) Grade 1/2 hypotension	2 Participants	—

SECONDARY outcome

Timeframe: Up to Day 28

Population: -Donors are not evaluable for this outcome measure.

-Time to neutrophil engraftment is measured by determining the first of 3 consecutive measurements of neutrophil count ≥ 500/μL following conditioning regimen induced nadir.

Outcome measures

Outcome measures
Measure	Arm 1: Donors -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10\^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10\^6 CD34+ cells/kg and at least ≥ 2 x 10\^6 CD34+ cells/kg from the combined collections.	Arm 2: Recipients n=22 Participants -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0
Time to Neutrophil Engraftment Post-transplant in Patients Undergoing Allogeneic Stem Cell Transplant (Arm 2 Recipients Only)	—	13 days Interval 11.0 to 26.0

SECONDARY outcome

Timeframe: Through 90 days

Population: -Donors are not evaluable for this outcome measure. 1 recipient was not evaluable due to no platelet nadir and 1 recipient was not evaluable due to no engraftment.

-Time to platelet engraftment is measured by determining the first of 3 consecutive measurements of platelet count ≥ 20,000/μL without platelet transfusion support for 7 days.

Outcome measures

Outcome measures
Measure	Arm 1: Donors -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10\^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10\^6 CD34+ cells/kg and at least ≥ 2 x 10\^6 CD34+ cells/kg from the combined collections.	Arm 2: Recipients n=20 Participants -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0
Time to Platelet Engraftment Post-transplant in Patients Undergoing Allogeneic Stem Cell Transplant (Arm 2 Recipients Only)	—	20 days Interval 15.0 to 41.0

SECONDARY outcome

Timeframe: Up to 1 year after transplantation

Population: -Donors are not evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Arm 1: Donors -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10\^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10\^6 CD34+ cells/kg and at least ≥ 2 x 10\^6 CD34+ cells/kg from the combined collections.	Arm 2: Recipients n=22 Participants -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0
Number of Recipients With Primary Graft Failure After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only)	—	0 Participants

SECONDARY outcome

Timeframe: Up to 1 year after transplantation

Population: -Donors are not evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Arm 1: Donors -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10\^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10\^6 CD34+ cells/kg and at least ≥ 2 x 10\^6 CD34+ cells/kg from the combined collections.	Arm 2: Recipients n=22 Participants -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0
Incidence of Secondary Graft Failure After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only)	—	0 Participants

SECONDARY outcome

Timeframe: Day 100

Population: -Donors are not evaluable for this outcome measure.

* Acute GVHD rate and worst severity is noted * 4 organ categories (skin, liver, lower GI, and upper GI) * Skin: Grade I: 1-2 , Grade II: 3, Grade III: N/A, Grade IV: 4 * Liver: Grade I: 0, Grade II: 1, Grade III: 2-4, Grade IV: N/A * Lower GI: Grade I: 0, Grade II: 1: Grade II: 2-3: Grade IV: 4 * Upper GI: Grade I: 0, Grade II: 1, Grade III: N/A, Grade IV: N/A * The cumulative incidence of grade 2-4 acute GVHD was determined using competing risk analysis. Competing risks for acute GVHD were death, relapse, and graft failure.

Outcome measures

Outcome measures
Measure	Arm 1: Donors -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10\^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10\^6 CD34+ cells/kg and at least ≥ 2 x 10\^6 CD34+ cells/kg from the combined collections.	Arm 2: Recipients n=22 Participants -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0
Cumulative Incidence of Grade 2-4 Acute Graft Versus Host Disease (GvHD) as Measured by Minnesota Acute GVHD Criteria (Arm 2 Recipients Only)	—	0.3636 proportion of participants Interval 0.169 to 0.5623

SECONDARY outcome

Timeframe: From Day 100 through 1 year after transplantation

Population: -Donors are not evaluable for this outcome measure.

* Chronic GVHD rate and severity for the first 365 days after PBSC infusion will be assessed based on the NIH criteria * The cumulative incidence of chronic GVHD was determined using competing risk analysis. Competing risks for acute GVHD were death, relapse, and graft failure.

Outcome measures

Outcome measures
Measure	Arm 1: Donors -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10\^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10\^6 CD34+ cells/kg and at least ≥ 2 x 10\^6 CD34+ cells/kg from the combined collections.	Arm 2: Recipients n=22 Participants -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0
Cumulative Incidence of Chronic GvHD in Patients Who Have Undergone Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only)	—	0.5000 proportion of participants Interval 0.2676 to 0.6946

SECONDARY outcome

Timeframe: Up to Day 2

Population: -Recipients are not evaluable for this outcome measure. -Only donors who received 1.25 mg/kg of BL-8040 were evaluable for this outcome measure (the 1st 10 donors received 1.00 mg/kg of BL-8040)

Outcome measures

Outcome measures
Measure	Arm 1: Donors n=9 Participants -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10\^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10\^6 CD34+ cells/kg and at least ≥ 2 x 10\^6 CD34+ cells/kg from the combined collections.	Arm 2: Recipients -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0
Number of Participants Who Collect 5 x 106 CD34+ Cells/kg of Recipient Weight in a Single Leukapheresis and in 2 Leukapheresis Sessions (Arm 1 Donors Only)	9 Participants	—

SECONDARY outcome

Timeframe: Up to 1 year after transplantation

Population: -Donors are not evaluable for this outcome measure.

-CMV reactivation will be defined as a positive test for CMV viremia as determined by an antigenemia assay or quantitative PCR that results in the administration of antiviral treatment directed against CMV

Outcome measures

Outcome measures
Measure	Arm 1: Donors -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10\^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10\^6 CD34+ cells/kg and at least ≥ 2 x 10\^6 CD34+ cells/kg from the combined collections.	Arm 2: Recipients n=22 Participants -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0
Incidence of CMV Reactivation After Transplantation of Hematopoietic Cells Mobilized With BL-8040 in CMV Seropositive Recipients	—	8 Participants

SECONDARY outcome

Timeframe: Up to 1 year after transplantation

Population: -Donors are not evaluable for this outcome measure.

-Death that results from a transplant procedure related complication (e.g. infection, organ failure, hemorrhage, GVHD) rather than from relapse of the underlying disease or an unrelated cause

Outcome measures

Outcome measures
Measure	Arm 1: Donors -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10\^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10\^6 CD34+ cells/kg and at least ≥ 2 x 10\^6 CD34+ cells/kg from the combined collections.	Arm 2: Recipients n=22 Participants -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0
Cumulative Incidence of Treatment-related Mortality After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only)	—	0.2273 proportion of participants Interval 0.0792 to 0.4208

SECONDARY outcome

Timeframe: At 1 year post-tranplantation

Population: Donors are not evaluable for this outcome measure.

-Disease relapse occurs in recipients who entered transplant in CR. Progression occurs in recipients with existent disease at transplant who meet criteria for progressive disease post-transplant. A recipient will be considered relapsed when there is a recurrence of the original malignant disease after transplantation. Date of relapse/progression is defined as the date at which the first observation of hematologic, radiographic, or cytogenetic changes which signify progression/relapse is made

Outcome measures

Outcome measures
Measure	Arm 1: Donors -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10\^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10\^6 CD34+ cells/kg and at least ≥ 2 x 10\^6 CD34+ cells/kg from the combined collections.	Arm 2: Recipients n=22 Participants -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0
Cumulative Incidence of Disease Relapse/Progression After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only)	—	0.227 proportion of participants Interval 0.079 to 0.42

SECONDARY outcome

Timeframe: At 2 years post-tranplantation

Population: Donors are not evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Arm 1: Donors -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10\^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10\^6 CD34+ cells/kg and at least ≥ 2 x 10\^6 CD34+ cells/kg from the combined collections.	Arm 2: Recipients n=22 Participants -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0
Cumulative Incidence of Disease Relapse/Progression After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only)	—	0.273 proportion of participants Interval 0.107 to 0.47

SECONDARY outcome

Timeframe: At 2 years post-transplantation

Population: Donors are not evaluable for this outcome measure.

-An event is defined as either graft failure, disease relapse as evidenced by hematologic, radiographic, or cytogenetic changes, or death. The event free survival is the time from Day 0 to occurrence of the first event.

Outcome measures

Outcome measures
Measure	Arm 1: Donors -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10\^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10\^6 CD34+ cells/kg and at least ≥ 2 x 10\^6 CD34+ cells/kg from the combined collections.	Arm 2: Recipients n=22 Participants -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0
Kaplan-Meier Estimate of Event Free Survival After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only)	—	50.0 percentage of participants

SECONDARY outcome

Timeframe: At 3 years post-transplantation

Population: Donors are not evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Arm 1: Donors -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10\^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10\^6 CD34+ cells/kg and at least ≥ 2 x 10\^6 CD34+ cells/kg from the combined collections.	Arm 2: Recipients n=22 Participants -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0
Kaplan-Meier Estimate of Event Free Survival After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only)	—	34.1 percentage of participants

SECONDARY outcome

Timeframe: At 2 years post-transplantation

Population: Donors are not evaluable for this outcome measure.

-The time from Day 0 to death

Outcome measures

Outcome measures
Measure	Arm 1: Donors -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10\^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10\^6 CD34+ cells/kg and at least ≥ 2 x 10\^6 CD34+ cells/kg from the combined collections.	Arm 2: Recipients n=22 Participants -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0
Kaplan-Meier Estimate of Overall Survival After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only)	—	63.6 percentage of participants

SECONDARY outcome

Timeframe: At 3 years post-transplantation

Population: Donors are not evaluable for this outcome measure.

-The time from Day 0 to death

Outcome measures

Outcome measures
Measure	Arm 1: Donors -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10\^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10\^6 CD34+ cells/kg and at least ≥ 2 x 10\^6 CD34+ cells/kg from the combined collections.	Arm 2: Recipients n=22 Participants -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0
Kaplan-Meier Estimate of Overall Survival After Transplantation of Hematopoietic Cells Mobilized With BL-8040 (Arm 2 Recipients Only)	—	49.0 percentage of participants

SECONDARY outcome

Timeframe: At 3-4 hours after BL-8040

Population: Recipients are not evaluable for this outcome measure.

Outcome measures

Outcome measures
Measure	Arm 1: Donors n=25 Participants -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10\^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10\^6 CD34+ cells/kg and at least ≥ 2 x 10\^6 CD34+ cells/kg from the combined collections.	Arm 2: Recipients -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0
Median Peripheral Blood CD34+ Cell Count (Arm 1 Donor Only)	15 CD34/microliters Interval 1.0 to 46.0	—

Adverse Events

Arm 1: Donors

Serious events: 0 serious events

Other events: 25 other events

Deaths: 0 deaths

Arm 2: Recipients

Serious events: 12 serious events

Other events: 22 other events

Deaths: 13 deaths

Serious adverse events

Other adverse events

Additional Information

Geoffrey Uy, M.D.

Washington University School of Medicine

Phone: 314-454-8304

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Measure	Arm 1: Donors n=25 participants at risk -Donors will receive subcutaneous (SC) BL-8040 in the morning (Day 1) followed by leukapheresis approximately 180 minutes (up to 270 minutes) after the injection per institutional protocol. If the donor does not reach the collection goal for mobilization (≥ 5.0 x 10\^6 CD34+ cells/kg), a second leukapheresis will be performed on Day 2 (24 hours ± 2 hours from the BL-8040 injection) in an effort to reach a total of ≥ 5 x 10\^6 CD34+ cells/kg and at least ≥ 2 x 10\^6 CD34+ cells/kg from the combined collections.	Arm 2: Recipients n=22 participants at risk -All or part of the leukapheresis product will be infused into the recipient per institutional guidelines. The day of the infusion will be considered Day 0; if the infusion occurs over multiple days, the final day of infusion will be considered Day 0
Blood and lymphatic system disorders Febrile neutropenia	0.00% 0/25 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.	9.1% 2/22 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.
Gastrointestinal disorders Diarrhea	0.00% 0/25 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.	13.6% 3/22 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.
Gastrointestinal disorders Mucositis oral	0.00% 0/25 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.	4.5% 1/22 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.
Gastrointestinal disorders Nausea	0.00% 0/25 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.	4.5% 1/22 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.
Gastrointestinal disorders Vomiting	0.00% 0/25 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.	4.5% 1/22 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.
General disorders Fever	0.00% 0/25 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.	4.5% 1/22 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.
General disorders Generalized weakness	0.00% 0/25 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.	4.5% 1/22 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.
Infections and infestations Sepsis	0.00% 0/25 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.	9.1% 2/22 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.
Infections and infestations Upper respiratory infection	0.00% 0/25 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.	4.5% 1/22 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.
Injury, poisoning and procedural complications Fall	0.00% 0/25 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.	4.5% 1/22 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.
Nervous system disorders Headache	0.00% 0/25 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.	4.5% 1/22 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.
Psychiatric disorders Delirium	0.00% 0/25 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.	4.5% 1/22 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.
Renal and urinary disorders Acute kidney injury	0.00% 0/25 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.	13.6% 3/22 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.
Reproductive system and breast disorders Genital edema	0.00% 0/25 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.	4.5% 1/22 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.
Reproductive system and breast disorders Testicular pain	0.00% 0/25 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.	4.5% 1/22 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.
Respiratory, thoracic and mediastinal disorders Respiratory failure	0.00% 0/25 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.	4.5% 1/22 • -Adverse events for donors were collected from the date the participant signed the consent form through 30 days after the last dose of BL-8040. In addition, any adverse events at least possibly related to BL-8040 that occurs up to 5 years after the BL-8040 dose should be recorded. -Adverse events for recipients were collected from the start of the conditioning regimen until Day +100 post transplant. Only grade 3-5 non-hematologic adverse events will be collected.