Trial Outcomes & Findings for BL-8040 and Nelarabine for Relapsed or Refractory T-Acute Lymphoblastic Leukemia/ Lymphoblastic Lymphoma (NCT NCT02763384)

Last Updated: 2023-10-31

Results Overview

The descriptions and grading scales found in the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 will be utilized for all adverse event reporting.

Recruitment status

TERMINATED

Study phase

PHASE2

Target enrollment

12 participants

Primary outcome timeframe

Up to 30 days after completion of treatment (median follow-up of 51.5 days, full range 24-120 days)

Results posted on

2023-10-31

Participant Flow

Participant milestones

Participant milestones
Measure	Arm 1: BL-8040 and Nelarabine * Cycle 1: BL-8040 subcutaneous daily from Day 1 to Day 6 and nelarabine intravenously over 2 hours on Days 2, 4, and 6 * Cycles 2-4: BL-8040 subcutaneous daily from Day 1 to Day 5 and nelarabine intravenously over 2 hours on Days 1, 3, and 5 * Treatment may be repeated every 21 days for up to 4 cycles
Overall Study STARTED	12
Overall Study COMPLETED	10
Overall Study NOT COMPLETED	2

Reasons for withdrawal

Reasons for withdrawal
Measure	Arm 1: BL-8040 and Nelarabine * Cycle 1: BL-8040 subcutaneous daily from Day 1 to Day 6 and nelarabine intravenously over 2 hours on Days 2, 4, and 6 * Cycles 2-4: BL-8040 subcutaneous daily from Day 1 to Day 5 and nelarabine intravenously over 2 hours on Days 1, 3, and 5 * Treatment may be repeated every 21 days for up to 4 cycles
Overall Study Physician Decision	1
Overall Study Rapid decline - went on hospice	1

Baseline Characteristics

BL-8040 and Nelarabine for Relapsed or Refractory T-Acute Lymphoblastic Leukemia/ Lymphoblastic Lymphoma

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Arm 1: BL-8040 and Nelarabine n=12 Participants * Cycle 1: BL-8040 subcutaneous daily from Day 1 to Day 6 and nelarabine intravenously over 2 hours on Days 2, 4, and 6 * Cycles 2-4: BL-8040 subcutaneous daily from Day 1 to Day 5 and nelarabine intravenously over 2 hours on Days 1, 3, and 5 * Treatment may be repeated every 21 days for up to 4 cycles
Age, Continuous	30 years n=5 Participants
Sex: Female, Male Female	3 Participants n=5 Participants
Sex: Female, Male Male	9 Participants n=5 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	0 Participants n=5 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	11 Participants n=5 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	1 Participants n=5 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=5 Participants
Race (NIH/OMB) Asian	0 Participants n=5 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=5 Participants
Race (NIH/OMB) Black or African American	1 Participants n=5 Participants
Race (NIH/OMB) White	11 Participants n=5 Participants
Race (NIH/OMB) More than one race	0 Participants n=5 Participants
Race (NIH/OMB) Unknown or Not Reported	0 Participants n=5 Participants
Region of Enrollment United States	12 participants n=5 Participants

PRIMARY outcome

Timeframe: Up to 30 days after completion of treatment (median follow-up of 51.5 days, full range 24-120 days)

The descriptions and grading scales found in the revised NCI Common Terminology Criteria for Adverse Events (CTCAE) version 4.0 will be utilized for all adverse event reporting.

Outcome measures

Outcome measures
Measure	Arm 1: BL-8040 and Nelarabine n=12 Participants * Cycle 1: BL-8040 subcutaneous daily from Day 1 to Day 6 and nelarabine intravenously over 2 hours on Days 2, 4, and 6 * Cycles 2-4: BL-8040 subcutaneous daily from Day 1 to Day 5 and nelarabine intravenously over 2 hours on Days 1, 3, and 5 * Treatment may be repeated every 21 days for up to 4 cycles
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 white blood cell increased post BL-8040 injection	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 lymphadenopathy worsening	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 leukocytosis	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 infarct	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 chest pain - cardiac	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 pericardial effusion	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 sinus bradycardia	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 sinus tachycardia	3 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 tinnitus	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 abdominal pain at study drug injection site	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 constipation	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 diarrhea	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 diarrhea	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 gastrointestinal hemorrhage	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 mucositis oral	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 nausea	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 oral hemorrhage	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 vomiting	4 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 "feeling funny" post transfusion	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 biopsy pain	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 catheter pain from replacement	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 disease progression	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 pain at CVC site	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 pain at right jugular Hohn catheter	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 injection site reaction (rash)	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 redness at Hohn catheter insertion site	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 right knee pain	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 diffuse pain	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 pain in leg	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 pain, skin nodule at biopsy site	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 transfusion reaction to packed red blood cells	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 chills	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 edema limbs	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 fatigue	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 fever	3 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 gait disturbance	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 injection site reaction	8 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 localized edema	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 malaise	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 non-cardiac chest pain	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 non-cardiac chest pain	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 allergic reaction	3 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 lung infection	3 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 sepsis	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 sinusitis	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 upper respiratory infection	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 urinary tract infection	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 urinary tract infection	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 bruising	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 fall	3 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 hip fracture	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 activated partial thromboplastin time prolonged	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 activated partial thromboplastin time prolonged	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 alanine aminotransferase increased	3 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 alanine aminotransferase increased	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 alkaline phosphatase	5 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 aspartate aminotransferase increased	3 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 aspartate aminotransferase increased	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 cardiac troponin T increased	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 creatinine increased	4 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 electrocardiogram QT corrected interval prolonged	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 electrocardiogram QT corrected interval prolonged	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 INR increased	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 serum amylase increased	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 weight gain	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 weight loss	3 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 anorexia	6 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 anorexia	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 dehydration	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 hyperglycemia	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 hyperuricemia	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 hyperuricemia	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 hypoalbuminemia	5 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 hypokalemia	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grades 3-5 hypomagmesemia	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 hypophosphatemia	4 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 tumor lysis syndrome	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 muscle weakness lower limb	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 neck pain	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 osteoporosis	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 pain in extremity	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 altered mental status	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 altered mental status	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 dizziness	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 headache	3 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 paresthesia	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 peripheral motor neuropathy	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 peripheral sensory neuropathy	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 somnolence	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 syncope	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 tremor	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 anxiety	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 confusion	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 depression	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 insomnia	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 hematuria	3 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 proteinuria	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 urinary incontinence	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 urinary retention	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 urinary tract pain	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 irregular menstruation	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 hemoptysis	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 atelectasis	3 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 dyspnea	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 dyspnea	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 hypoxia	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 hypoxia	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 nasal congestion	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 pleural effusion	3 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 productive cough	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 pulmonary edema	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 cellulitis	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 dry skin	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 erythema multiforme	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 pruritus	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 purpura	3 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 rash maculo-papular	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 scalp pain	1 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 hypertension	4 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 hypertension	3 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 1-2 hypotension	2 Participants
Safety and Tolerability of Regimen as Measured by Number of Participants With Adverse Events Grade 3-5 hypotension	1 Participants

SECONDARY outcome

Timeframe: Completion of treatment (approximately 12 weeks)

Population: 2 participants were not evaluable for this outcome measure because they stopped treatment prior to disease response assessment.

Complete remission (CR) = an absolute neutrophil count (segs and bands) \> 1,000/mcL, no circulating lymphoblasts, platelets ≥ 100,000/mcL; and \< 5% marrow leukemia blast cells with complete disappearance of all measurable disease as confirmed by physical examination, CT scan and/or FDG-PET (Deauville criteria score of 1, 2, or 3) -Morphologic complete remission with incomplete blood count recovery (CRi)=Defined as CR with the exception of neutropenia \< 1,000/mcL or thrombocytopenia \<100,000/mcL

Outcome measures

Outcome measures
Measure	Arm 1: BL-8040 and Nelarabine n=10 Participants * Cycle 1: BL-8040 subcutaneous daily from Day 1 to Day 6 and nelarabine intravenously over 2 hours on Days 2, 4, and 6 * Cycles 2-4: BL-8040 subcutaneous daily from Day 1 to Day 5 and nelarabine intravenously over 2 hours on Days 1, 3, and 5 * Treatment may be repeated every 21 days for up to 4 cycles
Composite Complete Remission Rate (CRc=CR+CRi)	4 Participants

SECONDARY outcome

Timeframe: Through completion of treatment (median treatment length of 37.5 days, full range of 13-90 days)

Population: 2 participants were not evaluable for this outcome measure because they stopped treatment prior to disease response assessment.

* Complete remission (CR) = an absolute neutrophil count (segs and bands) \> 1,000/mcL, no circulating lymphoblasts, platelets ≥ 100,000/mcL; and \< 5% marrow leukemia blast cells with complete disappearance of all measurable disease as confirmed by physical examination, CT scan and/or FDG-PET (Deauville criteria score of 1, 2, or 3) * Morphologic complete remission with incomplete blood count recovery (CRi)=Defined as CR with the exception of neutropenia \< 1,000/mcL or thrombocytopenia \<100,000/mcL * Partial remission (PR)=A PR requires all of the CR criteria except that marrow may still contain 5-25% leukemia blast cells. An absolute neutrophil count (segs and bands) ≥ 1000/mcL, no circulating blasts, and platelets \> 100,000/mcL are requires as for a CR. For patients with measurable disease, a reduction of 50% or more in the sum of the products of the perpendicular diameters of all measurable lesions compared with pretreatment measurements and with no new or enlarging lesions

Outcome measures

Outcome measures
Measure	Arm 1: BL-8040 and Nelarabine n=10 Participants * Cycle 1: BL-8040 subcutaneous daily from Day 1 to Day 6 and nelarabine intravenously over 2 hours on Days 2, 4, and 6 * Cycles 2-4: BL-8040 subcutaneous daily from Day 1 to Day 5 and nelarabine intravenously over 2 hours on Days 1, 3, and 5 * Treatment may be repeated every 21 days for up to 4 cycles
Overall Response Rate (CR, CRi + PR)	5 Participants

SECONDARY outcome

Timeframe: Through completion of treatment (median treatment length of 37.5 days, full range of 13-90 days)

* Response is CR or CRi * Complete remission (CR) = an absolute neutrophil count (segs and bands) \> 1,000/mcL, no circulating lymphoblasts, platelets ≥ 100,000/mcL; and \< 5% marrow leukemia blast cells with complete disappearance of all measurable disease as confirmed by physical examination, CT scan and/or FDG-PET (Deauville criteria score of 1, 2, or 3) * Morphologic complete remission with incomplete blood count recovery (CRi)=Defined as CR with the exception of neutropenia \< 1,000/mcL or thrombocytopenia \<100,000/mcL

Outcome measures

Outcome measures
Measure	Arm 1: BL-8040 and Nelarabine n=4 Participants * Cycle 1: BL-8040 subcutaneous daily from Day 1 to Day 6 and nelarabine intravenously over 2 hours on Days 2, 4, and 6 * Cycles 2-4: BL-8040 subcutaneous daily from Day 1 to Day 5 and nelarabine intravenously over 2 hours on Days 1, 3, and 5 * Treatment may be repeated every 21 days for up to 4 cycles
Time to Response	20 days Interval 20.0 to 39.0

SECONDARY outcome

Timeframe: Through date of recurrence or completion of follow-up (maximum of 2 years after completion of treatment)

Defined as the interval from the date CR/CRi is documented to the date of recurrence or completion of follow-up if recurrence has not occurred.

Outcome measures

Outcome measures
Measure	Arm 1: BL-8040 and Nelarabine n=4 Participants * Cycle 1: BL-8040 subcutaneous daily from Day 1 to Day 6 and nelarabine intravenously over 2 hours on Days 2, 4, and 6 * Cycles 2-4: BL-8040 subcutaneous daily from Day 1 to Day 5 and nelarabine intravenously over 2 hours on Days 1, 3, and 5 * Treatment may be repeated every 21 days for up to 4 cycles
Duration of Response	280 days Interval 54.0 to 471.0

SECONDARY outcome

Timeframe: Through completion of follow-up (maximum of 2 years after completion of treatment)

EFS is defined as the interval from the date of first dose of study drug to date of treatment failure, recurrence, of death due to any cause.

Outcome measures

Outcome measures
Measure	Arm 1: BL-8040 and Nelarabine n=12 Participants * Cycle 1: BL-8040 subcutaneous daily from Day 1 to Day 6 and nelarabine intravenously over 2 hours on Days 2, 4, and 6 * Cycles 2-4: BL-8040 subcutaneous daily from Day 1 to Day 5 and nelarabine intravenously over 2 hours on Days 1, 3, and 5 * Treatment may be repeated every 21 days for up to 4 cycles
Event-free Survival (EFS)	23 days Interval 13.0 to 314.0

SECONDARY outcome

Timeframe: Through completion of follow-up (maximum of 2 years after completion of treatment)

Defined as the date of first dose of study drug to the date of death from any cause.

Outcome measures

Outcome measures
Measure	Arm 1: BL-8040 and Nelarabine n=12 Participants * Cycle 1: BL-8040 subcutaneous daily from Day 1 to Day 6 and nelarabine intravenously over 2 hours on Days 2, 4, and 6 * Cycles 2-4: BL-8040 subcutaneous daily from Day 1 to Day 5 and nelarabine intravenously over 2 hours on Days 1, 3, and 5 * Treatment may be repeated every 21 days for up to 4 cycles
Overall Survival	95 days Interval 24.0 to 440.0

SECONDARY outcome

Timeframe: Through completion of treatment (median treatment length of 37.5 days, full range of 13-90 days)

Estimate rate of patients who proceed to alloHCT after treatment

Outcome measures

Outcome measures
Measure	Arm 1: BL-8040 and Nelarabine n=12 Participants * Cycle 1: BL-8040 subcutaneous daily from Day 1 to Day 6 and nelarabine intravenously over 2 hours on Days 2, 4, and 6 * Cycles 2-4: BL-8040 subcutaneous daily from Day 1 to Day 5 and nelarabine intravenously over 2 hours on Days 1, 3, and 5 * Treatment may be repeated every 21 days for up to 4 cycles
Rate of Patients Who Proceed to alloHCT After Treatment	3 Participants

Adverse Events

Arm 1: BL-8040 and Nelarabine

Serious events: 8 serious events

Other events: 12 other events

Deaths: 9 deaths

Serious adverse events

Serious adverse events
Measure	Arm 1: BL-8040 and Nelarabine n=12 participants at risk * Cycle 1: BL-8040 subcutaneous daily from Day 1 to Day 6 and nelarabine intravenously over 2 hours on Days 2, 4, and 6 * Cycles 2-4: BL-8040 subcutaneous daily from Day 1 to Day 5 and nelarabine intravenously over 2 hours on Days 1, 3, and 5 * Treatment may be repeated every 21 days for up to 4 cycles
Blood and lymphatic system disorders Leukocytosis	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Cardiac disorders Chest pain - cardiac	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
General disorders Fatigue	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
General disorders Non-cardiac chest pain	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Infections and infestations Lung infection	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Infections and infestations Sepsis	16.7% 2/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Injury, poisoning and procedural complications Hip fracture	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Injury, poisoning and procedural complications Pain at BL-8040 injection site	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Neoplasms benign, malignant and unspecified (incl cysts and polyps) Disease progression	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Nervous system disorders Altered mental status	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Nervous system disorders Peripheral motor neuropathy	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Vascular disorders Hypotension	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).

Other adverse events

Other adverse events
Measure	Arm 1: BL-8040 and Nelarabine n=12 participants at risk * Cycle 1: BL-8040 subcutaneous daily from Day 1 to Day 6 and nelarabine intravenously over 2 hours on Days 2, 4, and 6 * Cycles 2-4: BL-8040 subcutaneous daily from Day 1 to Day 5 and nelarabine intravenously over 2 hours on Days 1, 3, and 5 * Treatment may be repeated every 21 days for up to 4 cycles
Blood and lymphatic system disorders Lymphadenopathy worsening	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Cardiac disorders Chest pain - cardiac	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Cardiac disorders Infarct	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Cardiac disorders Pericardial effusion	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Cardiac disorders Sinus bradycardia	16.7% 2/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Cardiac disorders Sinus tachycardia	25.0% 3/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Ear and labyrinth disorders Tinnitus	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Gastrointestinal disorders Constipation	16.7% 2/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Gastrointestinal disorders Diarrhea	25.0% 3/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Gastrointestinal disorders Lower gastrointestinal hemorrhage	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Gastrointestinal disorders Mucositis oral	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Gastrointestinal disorders Nausea	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Gastrointestinal disorders Oral hemorrhage	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Gastrointestinal disorders Vomiting	33.3% 4/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
General disorders Chills	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
General disorders Diffuse pain	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
General disorders Edema limbs	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
General disorders Fever	25.0% 3/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
General disorders Gait disturbance	16.7% 2/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
General disorders Injection site reaction	66.7% 8/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
General disorders Injection site reaction (rash)	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
General disorders Localized edema	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
General disorders Malaise	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
General disorders Non-cardiac chest pain	16.7% 2/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
General disorders Pain at CVC site	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
General disorders Pain in leg	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
General disorders Transfusion reaction to pRBC	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
General disorders "Feeling funny" post transfusion	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Immune system disorders Allergic reaction	25.0% 3/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Infections and infestations Lung infection	16.7% 2/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Infections and infestations Sinusitis	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Infections and infestations Upper respiratory infection	16.7% 2/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Infections and infestations Urinary tract infection	16.7% 2/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Injury, poisoning and procedural complications Biopsy pain	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Injury, poisoning and procedural complications Bruising	16.7% 2/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Injury, poisoning and procedural complications Catheter pain, replacement	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Injury, poisoning and procedural complications Fall	25.0% 3/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Injury, poisoning and procedural complications Pain at right internal jugular Hohn catheter	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Injury, poisoning and procedural complications Pain, skin nodule at biopsy site	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Injury, poisoning and procedural complications Redness at Hohn catheter insertion site	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Investigations Activated partial thromboplastin time prolonged	25.0% 3/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Investigations Alanine aminotransferase increased	33.3% 4/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Investigations Alkaline phosphatase increased	41.7% 5/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Investigations Aspartate aminotransferase increased	41.7% 5/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Investigations Cardiac troponin T increased	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Investigations Creatinine increased	33.3% 4/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Investigations Electrocardiogram QT corrected interval prolonged	25.0% 3/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Investigations INR increased	16.7% 2/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Investigations Serum amylase increased	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Investigations Weight gain	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Investigations Weight loss	25.0% 3/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Investigations White blood cell count increased post BL-8040 injection	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Metabolism and nutrition disorders Anorexia	66.7% 8/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Metabolism and nutrition disorders Dehydration	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Metabolism and nutrition disorders Hyperglycemia	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Metabolism and nutrition disorders Hyperuricemia	25.0% 3/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Metabolism and nutrition disorders Hypoalbuminemia	41.7% 5/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Metabolism and nutrition disorders Hypokalemia	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Metabolism and nutrition disorders Hypomagnesemia	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Metabolism and nutrition disorders Hypophosphatemia	33.3% 4/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Metabolism and nutrition disorders Tumor lysis syndrome	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Musculoskeletal and connective tissue disorders Muscle weakness lower limb	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Musculoskeletal and connective tissue disorders Neck pain	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Musculoskeletal and connective tissue disorders Osteoporosis	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Musculoskeletal and connective tissue disorders Pain in extremity	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Musculoskeletal and connective tissue disorders Right knee pain	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Nervous system disorders Altered mental status	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Nervous system disorders Dizziness	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Nervous system disorders Headache	25.0% 3/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Nervous system disorders Paresthesia	16.7% 2/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Nervous system disorders Peripheral sensory neuropathy	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Nervous system disorders Somnolence	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Nervous system disorders Syncope	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Nervous system disorders Tremor	16.7% 2/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Psychiatric disorders Anxiety	16.7% 2/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Psychiatric disorders Confusion	16.7% 2/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Psychiatric disorders Depression	16.7% 2/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Psychiatric disorders Insomnia	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Renal and urinary disorders Hematuria	25.0% 3/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Renal and urinary disorders Proteinuria	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Renal and urinary disorders Urinary incontinence	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Renal and urinary disorders Urinary retention	16.7% 2/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Renal and urinary disorders Urinary tract pain	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Renal and urinary disorders Irregular menstruation	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Respiratory, thoracic and mediastinal disorders Atelectasis	25.0% 3/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Respiratory, thoracic and mediastinal disorders Dyspnea	25.0% 3/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Respiratory, thoracic and mediastinal disorders Hemoptysis	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Respiratory, thoracic and mediastinal disorders Hypoxia	25.0% 3/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Respiratory, thoracic and mediastinal disorders Nasal congestion	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Respiratory, thoracic and mediastinal disorders Pleural effusion	25.0% 3/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Respiratory, thoracic and mediastinal disorders Productive cough	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Respiratory, thoracic and mediastinal disorders Pulmonary edema	16.7% 2/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Infections and infestations Cellulitis on hand	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Skin and subcutaneous tissue disorders Dry skin	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Skin and subcutaneous tissue disorders Erythema multiforme	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Skin and subcutaneous tissue disorders Pruritus	16.7% 2/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Skin and subcutaneous tissue disorders Purpura	25.0% 3/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Skin and subcutaneous tissue disorders Rash maculo-papular	16.7% 2/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Skin and subcutaneous tissue disorders Scalp pain	8.3% 1/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Vascular disorders Hypertension	58.3% 7/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).
Vascular disorders Hypotension	16.7% 2/12 • Adverse events were collected from first dose of study treatment through 30 days after the last study treatment (maximum of 4 cycles of treatment - each cycle is 21 days). All-cause mortality was collected from first dose of study treatment through completion of follow-up (maximum of 2 years following completion of treatment).

Additional Information

Geoffrey L. Uy, M.D.

Washington University School of Medicine

Phone: 314-747-8439

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place