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

March 6, 2025 updated by: Bio-Path Holdings, Inc.

A Phase I/Ib Study of BP1002 (a Liposomal Bcl-2 Antisense Oligodeoxynucleotide) in Patients With Refractory/Relapsed Acute Myeloid Leukemia (AML)

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).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
48

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • United States
    • California
      • La Jolla, California, United States, 92037
        • Recruiting
        • Scripps Green Hospital
        • Contact:
        • Principal Investigator:
          • David Hermel, MD
      • Los Angeles, California, United States, 90024
        • Recruiting
        • UCLA Medical Center
        • Contact:
          • Gary Schiller, MD
        • Principal Investigator:
          • Gary Schiller, MD
    • New York
      • New York, New York, United States, 10021
        • Recruiting
        • Weill Cornell Medical College - NewYork-Presbyterian Hospital
        • Contact:
        • Contact:
        • Principal Investigator:
          • Gail J Roboz, MD
    • Texas
      • Houston, Texas, United States, 77030
        • Recruiting
        • MD Anderson Cancer Center
        • Contact:
        • Contact:
        • Principal Investigator:
          • Maro Ohanian, D.O.

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. 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
  2. Eastern Cooperative Oncology Group (ECOG) Performance Status Score of 0, 1 or 2
  3. For the dose expansion phase, participants with documented diagnosis of AML who are eligible for decitabine therapy

  4. 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.
  5. 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
  6. Male participants must agree to use an acceptable method of contraception for the duration of the study
  7. Recovered from the effects of any prior surgery, radiotherapy, or antineoplastic treatment (with the exception of alopecia), based on Investigator assessment
  8. Participants must be willing and able to provide written informed consent

Exclusion Criteria:

  1. Active non-hematologic or lymphoid malignancy other than AML treated with immunotherapy, targeted therapy or chemotherapy within the previous 12 months
  2. 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
  3. 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.
  4. Acute promyelocytic leukemia (APL) with t(15;17)(q22;q12) PML-RARA
  5. Chronic myeloid leukemia in any phase
  6. Receipt of any anti-cancer therapy within 14 days prior to C1D1, with the exception of hydroxyurea or leukapheresis
  7. Participants may not be receiving any other investigational agents
  8. Female participants who are pregnant or breast-feeding
  9. Substance abuse, medical, psychological or social conditions that may interfere with the patient's participation in the study or evaluation of the study results
  10. 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
  11. History of any hypersensitivity to hypomethylating agents, unless reaction is deemed irrelevant to the study by the Investigator and Medical Monitor
  12. Unresolved toxicity higher than CTCAE Grade 1 attributed to any prior therapy or procedure, excluding alopecia
  13. 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)
  14. 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
  15. Uncontrolled seizure disorder (i.e., seizures within the past 2 months)
  16. Unable or unwilling to communicate or cooperate with the Investigator or follow the protocol for any reason

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: Relapsed/Refractory AML - BP1002 monotherapy
BP1002 monotherapy dose escalation
Drug: BP1002; Liposomal Bcl-2 Antisense Oligodeoxynucleotide
Dose escalation of BP1002 monotherapy
Other Names:
  • Liposomal Bcl-2; L-Bcl-2
Experimental: Relapsed/Refractory AML - BP1002 in combination with decitabine
BP1002 single dose in combination with decitabine
Drug: BP1002; Liposomal Bcl-2 Antisense Oligodeoxynucleotide
Dose escalation of BP1002 monotherapy
Other Names:
  • Liposomal Bcl-2; L-Bcl-2
Drug: Decitabine (in combination with BP1002)
Dose expansion of BP1002 in combination with decitabine
Other Names:
  • Decitabine

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Identify Dose Limiting Toxicity (DLT) of BP1002
Time Frame: 30 days
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
Time Frame: 30 days
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
Time Frame: 30 days
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
Time Frame: 210 days
Determine RP2D by evaluating Maximally Tolerated Dose (MTD) data
210 days
Determine plasma pharmacokinetics (PK) of BP1002 using maximum plasma drug concentration
Time Frame: 30 days
Evaluate plasma PK of BP1002 using maximum plasma drug concentration (Cmax)
30 days
Determine plasma pharmacokinetics (PK) of BP1002 using volume of distribution
Time Frame: 30 days
Evaluate in vivo PK of BP1002 using volume of distribution (Vd)
30 days
Determine plasma pharmacokinetics (PK) of BP1002 using elimination rate constant
Time Frame: 30 days
Evaluate in vivo PK of BP1002 using elimination rate constant
30 days
Determine half-life plasma pharmacokinetics (PK) of BP1002
Time Frame: 30 days
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
Time Frame: 30 days
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
Time Frame: 30 days
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
Time Frame: 30 days
Evaluate ADA via peripheral blood
30 days

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Determine evidence of response by bone marrow aspirate
Time Frame: 180 days
Assess complete remission (CR), CR with incomplete hematologic recovery (CRi), and CR with partial hematologic recovery (CRh) per Döhner 2017
180 days
Determine evidence of response by complete blood counts using peripheral blood
Time Frame: 180 days
Assess complete remission (CR), CR with incomplete hematologic recovery (CRi), and CR with partial hematologic recovery (CRh) per Döhner 2017
180 days
Assessment of morphologic leukemia free state (MLFS) and partial remissions by bone marrow aspirate and complete blood counts
Time Frame: 180 days
To assess percentage of participants with MLFS and partial remissions per Döhner 2017
180 days
Assessment of morphologic leukemia free state (MLFS) and partial remissions by bone marrow aspirate
Time Frame: 180 days
To assess percentage of participants with MLFS and partial remissions per Döhner 2017
180 days
Assessment of blast count reductions by complete blood counts using peripheral blood
Time Frame: 180 days
To assess blast count reductions per Williams 2016
180 days
To determine progression-free survival (PFS), overall survival (OS), and duration of response
Time Frame: 180 days
To assess progression-free survival (PFS), overall survival (OS), and duration of response from date of study entry to study closure or death
180 days

Other Outcome Measures

Other Outcome Measures

For clinical trials, a planned measurement described in the protocol that is used to determine the effect of an intervention/treatment on participants. For observational studies, a measurement or observation that is used to describe patterns of diseases or traits, or associations with exposures, risk factors, or treatment. Types of outcome measures include primary outcome measure and secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Exploratory objective to correlate treatment response with cytogenetic characteristics
Time Frame: 30 days
Flow cytometry assays to determine the effects of BP1002 on Bcl-2 protein expression
30 days
Exploratory objective to correlate treatment response with molecular characteristics
Time Frame: 30 days
Flow cytometry assays to determine the effects of BP1002 on Bcl-2 protein expression
30 days

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Bio-Path Holdings, Inc.

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Gail J Roboz, MD, Weill Cornell Medical College - New York-Presbyterian Hospital

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
August 16, 2022

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 1, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
September 1, 2027

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
December 8, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
December 29, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
January 13, 2022

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
March 25, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 6, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
March 1, 2025

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • BP1002-102-AML

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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