RecruitingNCT05190471Updated Mar 10, 2025

A Clinical Trial of BP1002 in Patients With Refractory/Relapsed Acute Myeloid Leukemia (AML)

A Phase 1 interventional study of BP1002; Liposomal Bcl-2 Antisense Oligodeoxynucleotide and Decitabine (in combination with BP1002) in Acute Myeloid Leukemia, in Relapse and Acute Myeloid Leukemia Refractory, sponsored by Bio-Path Holdings, Inc.. Recruiting at 4 sites in United States. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2025-03-10.

Sponsored by Bio-Path Holdings, Inc. · Phase 1, Interventional, and Treatment

From the registry’s dates

Started Aug 2022; still recruiting 4 years 1 month later.

Phase: Phase 1
Study type: Interventional
Enrollment: 48
Allocation: Non-randomized
Ages: 18 Years and older
Sex: All

Study 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).

Conditions studied

Acute Myeloid Leukemia, in Relapse
Acute Myeloid Leukemia Refractory

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In context

Leukemia

5,441 studies on the registry are indexed under Leukemia; 636 are open to participants now.

This study's planned enrollment of 48 is above the median of 38 across 4,247 interventional studies indexed under Leukemia.

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Lead sponsor

Bio-Path Holdings, Inc. is the lead sponsor of 6 studies on the registry; 3 are open to participants now.

Counted across the registry records on this site, refreshed daily.

Who can participate

Ages eligible: 18 Years and older
Sexes eligible: All
Accepts healthy volunteers: No

Inclusion criteria

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:
1. Aspartate transaminase (AST) and alanine transaminase (ALT) ≤2.5 times the upper limit of normal (ULN); and
2. 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;
3. 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

Exclusion criteria

Exclusion Criteria:

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)
Unable or unwilling to communicate or cooperate with the Investigator or follow the protocol for any reason

Study design

Phase: Phase 1
Primary purpose: Treatment
Allocation: Non-randomized
Intervention model: Sequential assignment
Masking: None (open label)
Enrollment: 48 participants (estimated)

Study arms

Experimental
Relapsed/Refractory AML - BP1002 monotherapy
BP1002 monotherapy dose escalation
Drug: BP1002; Liposomal Bcl-2 Antisense Oligodeoxynucleotide
Experimental
Relapsed/Refractory AML - BP1002 in combination with decitabine
BP1002 single dose in combination with decitabine
Drug: BP1002; Liposomal Bcl-2 Antisense Oligodeoxynucleotide · Drug: Decitabine (in combination with BP1002)

Interventions

DrugBP1002; Liposomal Bcl-2 Antisense Oligodeoxynucleotide
Dose escalation of BP1002 monotherapy
Also known as: Liposomal Bcl-2; L-Bcl-2
DrugDecitabine (in combination with BP1002)
Dose expansion of BP1002 in combination with decitabine
Also known as: Decitabine

What researchers measure

Primary outcomes

Identify Dose Limiting Toxicity (DLT) of BP1002
Identify DLT of BP1002 using non-hematologic and hematologic measures per NCI CTCAE criteria
Time frame: 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
Time frame: 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
Time frame: 30 days
Recommended Phase 2 (RP2D) of BP1002
Determine RP2D by evaluating Maximally Tolerated Dose (MTD) data
Time frame: 210 days
Determine plasma pharmacokinetics (PK) of BP1002 using maximum plasma drug concentration
Evaluate plasma PK of BP1002 using maximum plasma drug concentration (Cmax)
Time frame: 30 days
Determine plasma pharmacokinetics (PK) of BP1002 using volume of distribution
Evaluate in vivo PK of BP1002 using volume of distribution (Vd)
Time frame: 30 days
Determine plasma pharmacokinetics (PK) of BP1002 using elimination rate constant
Evaluate in vivo PK of BP1002 using elimination rate constant
Time frame: 30 days
Determine half-life plasma pharmacokinetics (PK) of BP1002
Evaluate in vivo PK of BP1002 half-life (t1/2)
Time frame: 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)
Time frame: 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
Time frame: 30 days
Determine anti-drug antibody (ADA) levels of BP1002
Evaluate ADA via peripheral blood
Time frame: 30 days

Secondary outcomes

Determine evidence of response by bone marrow aspirate
Assess complete remission (CR), CR with incomplete hematologic recovery (CRi), and CR with partial hematologic recovery (CRh) per Döhner 2017
Time frame: 180 days
Determine evidence of response by complete blood counts using peripheral blood
Assess complete remission (CR), CR with incomplete hematologic recovery (CRi), and CR with partial hematologic recovery (CRh) per Döhner 2017
Time frame: 180 days
Assessment of morphologic leukemia free state (MLFS) and partial remissions by bone marrow aspirate and complete blood counts
To assess percentage of participants with MLFS and partial remissions per Döhner 2017
Time frame: 180 days
Assessment of morphologic leukemia free state (MLFS) and partial remissions by bone marrow aspirate
To assess percentage of participants with MLFS and partial remissions per Döhner 2017
Time frame: 180 days
Assessment of blast count reductions by complete blood counts using peripheral blood
To assess blast count reductions per Williams 2016
Time frame: 180 days
To determine progression-free survival (PFS), overall survival (OS), and duration of response
To assess progression-free survival (PFS), overall survival (OS), and duration of response from date of study entry to study closure or death
Time frame: 180 days

Other outcomes

Exploratory objective to correlate treatment response with cytogenetic characteristics
Flow cytometry assays to determine the effects of BP1002 on Bcl-2 protein expression
Time frame: 30 days
Exploratory objective to correlate treatment response with molecular characteristics
Flow cytometry assays to determine the effects of BP1002 on Bcl-2 protein expression
Time frame: 30 days

Study locations

4 of 4 sites recruiting

Scripps Green Hospital
La Jolla, California 92037, United States
- Kiley Borchard · Contact · borchard.kiley@scrippshealth.org · 858-554-9253
- David Hermel, MD · Principal investigator
Recruiting
UCLA Medical Center
Los Angeles, California 90024, United States
- Gary Schiller, MD · Contact
- Gary Schiller, MD · Principal investigator
Recruiting
Weill Cornell Medical College - NewYork-Presbyterian Hospital
New York, New York 10021, United States
- Dunay Bach · Contact · dub4001@med.cornell.edu · 212-746-4447
- Gail Roboz, MD · Contact · gar2001@med.cornell.edu · 646-962-2700
- Gail J Roboz, MD · Principal investigator
Recruiting
MD Anderson Cancer Center
Houston, Texas 77030, United States
- Miranda Lim, BSN, RN · Contact · MGLim@mdanderson.org · 713-794-1722
- Maro Ohanian, D.O. · Contact · mohanian@mdanderson.org · 713-792-2631
- Maro Ohanian, D.O. · Principal investigator
Recruiting

Updates

Tracking since Sep 25, 2026

No changes since tracking began. The registry record was last updated on Mar 10, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗

Registry details

Key details

Study ID: NCT05190471
Lead sponsor: Bio-Path Holdings, Inc.
Responsible party: Sponsor
First posted: Jan 13, 2022
Start date: Aug 16, 2022
Primary completion: Mar 2027 (estimated)
Completion: Sep 2027 (estimated)
Last update: Mar 10, 2025

Study contacts

Michael Hickey

Contact

mhickey@biopathholdings.com

832-742-1361

Gail J Roboz, MD

principal investigator · Weill Cornell Medical College - New York-Presbyterian Hospital

Oversight

Data monitoring committee: Yes
FDA-regulated drug: Yes
FDA-regulated device: No

View the source record on ClinicalTrials.gov ↗

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