A Clinical Trial of BP1002 in Patients With Refractory/Relapsed Acute Myeloid Leukemia (AML)
A Phase I/Ib Study of BP1002 (a Liposomal Bcl-2 Antisense Oligodeoxynucleotide) in Patients With Refractory/Relapsed Acute Myeloid Leukemia (AML)
1 other identifier
interventional
48
1 country
4
Brief Summary
This study evaluates the safety and tolerability of escalating doses of BP1002 (Liposomal Bcl-2 Antisense Oligodeoxynucleotide) in patients with refractory/relapsed AML. The study is designed to assess the safety profile, identify DLTs, biologically effective doses, PK, PD and potential anti-leukemic effects of BP1002 as single agent (dose escalation phase) followed by assessing BP1002 in combination with decitabine (dose expansion phase).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P50-P75 for phase_1
Started Aug 2022
Longer than P75 for phase_1
4 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
December 8, 2021
CompletedFirst Posted
Study publicly available on registry
January 13, 2022
CompletedStudy Start
First participant enrolled
August 16, 2022
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 1, 2027
ExpectedStudy Completion
Last participant's last visit for all outcomes
September 1, 2027
March 10, 2025
March 1, 2025
4.5 years
December 8, 2021
March 6, 2025
Conditions
Outcome Measures
Primary Outcomes (11)
Identify Dose Limiting Toxicity (DLT) of BP1002
Identify DLT of BP1002 using non-hematologic and hematologic measures per NCI CTCAE criteria
30 days
Identify and grade treatment-emergent adverse events (TEAE) of escalating doses of BP1002
Identify TEAE of BP1002 using non-hematologic and hematologic measures per NCI CTCAE criteria
30 days
Identify and grade treatment-emergent laboratory abnormalities of escalating doses of BP1002
Identify and grade treatment-emergent laboratory abnormalities of escalating doses of BP1002 using non-hematologic and hematologic measure per NCI CTCAE criteria
30 days
Recommended Phase 2 (RP2D) of BP1002
Determine RP2D by evaluating Maximally Tolerated Dose (MTD) data
210 days
Determine plasma pharmacokinetics (PK) of BP1002 using maximum plasma drug concentration
Evaluate plasma PK of BP1002 using maximum plasma drug concentration (Cmax)
30 days
Determine plasma pharmacokinetics (PK) of BP1002 using volume of distribution
Evaluate in vivo PK of BP1002 using volume of distribution (Vd)
30 days
Determine plasma pharmacokinetics (PK) of BP1002 using elimination rate constant
Evaluate in vivo PK of BP1002 using elimination rate constant
30 days
Determine half-life plasma pharmacokinetics (PK) of BP1002
Evaluate in vivo PK of BP1002 half-life (t1/2)
30 days
Identify conduction and rhythm changes (treatment emergent QTc elevations or other treatment emergent changes in ECG intervals) of escalating doses of BP1002
Collection of 12-lead ECGs at defined intervals to identify conduction and rhythm changes (treatment emergent QTc elevations or other treatment emergent changes in ECG intervals)
30 days
Determine pharmacodynamics (PD) of BP1002
Flow cytometry will be performed using peripheral blood to evaluate Bcl-2 target inhibition by BP1002 on pre and post treatment samples
30 days
Determine anti-drug antibody (ADA) levels of BP1002
Evaluate ADA via peripheral blood
30 days
Secondary Outcomes (6)
Determine evidence of response by bone marrow aspirate
180 days
Determine evidence of response by complete blood counts using peripheral blood
180 days
Assessment of morphologic leukemia free state (MLFS) and partial remissions by bone marrow aspirate and complete blood counts
180 days
Assessment of morphologic leukemia free state (MLFS) and partial remissions by bone marrow aspirate
180 days
Assessment of blast count reductions by complete blood counts using peripheral blood
180 days
- +1 more secondary outcomes
Other Outcomes (2)
Exploratory objective to correlate treatment response with cytogenetic characteristics
30 days
Exploratory objective to correlate treatment response with molecular characteristics
30 days
Study Arms (2)
Relapsed/Refractory AML - BP1002 monotherapy
EXPERIMENTALBP1002 monotherapy dose escalation
Relapsed/Refractory AML - BP1002 in combination with decitabine
EXPERIMENTALBP1002 single dose in combination with decitabine
Interventions
Dose escalation of BP1002 monotherapy
Dose expansion of BP1002 in combination with decitabine
Eligibility Criteria
You may qualify if:
- Adults ≥18 years of age, with histologic evidence of refractory/relapsed AML who have failed treatment with available therapies known to be active for refractory/relapsed AML
- Eastern Cooperative Oncology Group (ECOG) Performance Status Score of 0, 1 or 2
- For the dose expansion phase, participants with documented diagnosis of AML who are eligible for decitabine therapy
- Participants must have adequate hepatic and renal functions as defined by:
- Aspartate transaminase (AST) and alanine transaminase (ALT) ≤2.5 times the upper limit of normal (ULN); and
- Usually total bilirubin ≤ 1.5 ULN. In specific cases the PI may request a waiver of this requirement with medical justification and agreement with the medical monitor and Bio-Path Holdings. And;
- Estimated creatinine clearance of at least 60 mL/min. These estimations are calculated using the Cockcroft-Gault equation.
- Female participants of childbearing potential must agree to use an acceptable method of birth control (i.e. a hormonal contraceptive, intrauterine device, diaphragm with spermicide, condom with spermicide or abstinence) for the duration of the study and for at least 6 months after the last dose of study drug or decitabine
- Male participants must agree to use an acceptable method of contraception for the duration of the study
- Recovered from the effects of any prior surgery, radiotherapy, or antineoplastic treatment (with the exception of alopecia), based on Investigator assessment
- Participants must be willing and able to provide written informed consent
You may not qualify if:
- Active non-hematologic or lymphoid malignancy other than AML treated with immunotherapy, targeted therapy or chemotherapy within the previous 12 months
- Known, active leptomeningeal leukemia requiring intrathecal therapy. NOTE: Participants with a history of CNS disease may be allowed to participate based on at least 1 documented, negative spinal fluid assessment within 28 days prior to Screening
- Isolated potentially treatable extramedullary leukemia without also meeting bone marrow criteria for acute leukemia (for AML usually ≥ 5% blasts in BMA or biopsy). Participants may have leukemia with lower blast counts (Döhner 2017). Bio-Path Holdings and Investigator concurrence required.
- Acute promyelocytic leukemia (APL) with t(15;17)(q22;q12) PML-RARA
- Chronic myeloid leukemia in any phase
- Receipt of any anti-cancer therapy within 14 days prior to C1D1, with the exception of hydroxyurea or leukapheresis
- Participants may not be receiving any other investigational agents
- Female participants who are pregnant or breast-feeding
- Substance abuse, medical, psychological or social conditions that may interfere with the patient's participation in the study or evaluation of the study results
- Participants with human immunodeficiency virus (HIV) infection who have CD4+ T-cell counts \< 350 cells/mcL or with clinically active hepatitis B or C infection
- History of any hypersensitivity to hypomethylating agents, unless reaction is deemed irrelevant to the study by the Investigator and Medical Monitor
- Unresolved toxicity higher than CTCAE Grade 1 attributed to any prior therapy or procedure, excluding alopecia
- Presence of concurrent conditions that, in the opinion of the Investigator and/or Medical Monitor, may compromise the participant's ability to tolerate study treatment or interfere with any aspect of study conduct or interpretation of results. This includes, but is not limited to, unstable or uncontrolled angina, New York Heart Association (NYHA) class III or IV congestive heart failure, uncontrolled and sustained hypertension, clinically significant cardiac dysrhythmia or clinically significant baseline ECG abnormality (e.g., QTcF \>470 msec)
- Within the past 6 months, has had any of the following: myocardial infarction, unstable angina pectoris, coronary/peripheral artery bypass graft, cerebrovascular accident or transient ischemic attack
- Uncontrolled seizure disorder (i.e., seizures within the past 2 months)
- +1 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (4)
Scripps Green Hospital
La Jolla, California, 92037, United States
UCLA Medical Center
Los Angeles, California, 90024, United States
Weill Cornell Medical College - NewYork-Presbyterian Hospital
New York, New York, 10021, United States
MD Anderson Cancer Center
Houston, Texas, 77030, United States
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Gail J Roboz, MD
Weill Cornell Medical College - New York-Presbyterian Hospital
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 1
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
December 8, 2021
First Posted
January 13, 2022
Study Start
August 16, 2022
Primary Completion (Estimated)
March 1, 2027
Study Completion (Estimated)
September 1, 2027
Last Updated
March 10, 2025
Record last verified: 2025-03