Acute Myeloid Leukemia, in RelapsePhase 1NCT05190471
A Clinical Trial of BP1002 in Patients With Refractory/Relapsed Acute Myeloid Leukemia (AML)
A Phase 1 study testing BP1002; Liposomal Bcl-2 Antisense Oligodeoxynucleotide and Decitabine (in combination with BP1002) in people with Acute Myeloid Leukemia, in Relapse and Acute Myeloid Leukemia Refractory, run by Bio-Path Holdings, Inc. at 4 sites in United States.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acute Myeloid Leukemia, in Relapse, Acute Myeloid Leukemia Refractory
- What is being tested?
- BP1002; Liposomal Bcl-2 Antisense Oligodeoxynucleotide and Decitabine (in combination with BP1002)
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 48 participants
- Where
- 4 sitesUnited States
- Study dates
- 16 Aug 2022to Sep 2027
- Phase
- Phase 1
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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;
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- 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
Who cannot join
- 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
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- 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
Trial locations and contacts
4 sites in 1 country. Contact the study team or a site near you to ask about taking part.
United States4 sites, 4 recruiting
- Recruiting
MD Anderson Cancer Center
Houston, Texas, 77030
Miranda Lim, BSN, RN, 713-794-1722, MGLim@mdanderson.org
Maro Ohanian, D.O., 713-792-2631, mohanian@mdanderson.org
- Recruiting
Scripps Green Hospital
La Jolla, California, 92037
Kiley Borchard, 858-554-9253, borchard.kiley@scrippshealth.org
David Hermel, MD
- Recruiting
UCLA Medical Center
Los Angeles, California, 90024
Gary Schiller, MD
Gary Schiller, MD
- Recruiting
Weill Cornell Medical College - NewYork-Presbyterian Hospital
New York, New York, 10021
Dunay Bach, 212-746-4447, dub4001@med.cornell.edu
Gail Roboz, MD, 646-962-2700, gar2001@med.cornell.edu
About this study
Official title: 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).
- Being tested
- BP1002; Liposomal Bcl-2 Antisense Oligodeoxynucleotide, Decitabine (in combination with BP1002)
- Medical subject headings
- Leukemia, Myeloid, Acute
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BP1002; Liposomal Bcl-2 Antisense OligodeoxynucleotideDrug
Also called Liposomal Bcl-2; L-Bcl-2
Dose escalation of BP1002 monotherapy
Decitabine (in combination with BP1002)Drug
Also called Decitabine
Dose expansion of BP1002 in combination with decitabine
| Study group | Role | What they receive |
|---|---|---|
| Relapsed/Refractory AML - BP1002 monotherapy | Experimental | BP1002 monotherapy dose escalation Receives: Drug: BP1002; Liposomal Bcl-2 Antisense Oligodeoxynucleotide |
| Relapsed/Refractory AML - BP1002 in combination with decitabine | Experimental | BP1002 single dose in combination with decitabine Receives: Drug: BP1002; Liposomal Bcl-2 Antisense Oligodeoxynucleotide; Drug: Decitabine (in combination with BP1002) |
What is being measured
The primary outcome is the main question the study is designed to answer.
Identify Dose Limiting Toxicity (DLT) of BP1002
Identify DLT of BP1002 using non-hematologic and hematologic measures per NCI CTCAE criteria
Measured: 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
Measured: 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
Measured: 30 days
Recommended Phase 2 (RP2D) of BP1002
Determine RP2D by evaluating Maximally Tolerated Dose (MTD) data
Measured: 210 days
Determine plasma pharmacokinetics (PK) of BP1002 using maximum plasma drug concentration
Evaluate plasma PK of BP1002 using maximum plasma drug concentration (Cmax)
Measured: 30 days
Determine plasma pharmacokinetics (PK) of BP1002 using volume of distribution
Evaluate in vivo PK of BP1002 using volume of distribution (Vd)
Measured: 30 days
Determine plasma pharmacokinetics (PK) of BP1002 using elimination rate constant
Evaluate in vivo PK of BP1002 using elimination rate constant
Measured: 30 days
Determine half-life plasma pharmacokinetics (PK) of BP1002
Evaluate in vivo PK of BP1002 half-life (t1/2)
Measured: 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)
Measured: 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
Measured: 30 days
Determine anti-drug antibody (ADA) levels of BP1002
Evaluate ADA via peripheral blood
Measured: 30 days
Secondary outcomes (6)
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
Measured: 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
Measured: 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
Measured: 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
Measured: 180 days
Assessment of blast count reductions by complete blood counts using peripheral blood
To assess blast count reductions per Williams 2016
Measured: 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
Measured: 180 days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Jan 2022
- Study start16 Aug 2022
- Primary completionMar 2027 (estimated)
- Study completionSep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acute Myeloid Leukemia, in RelapseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- None (open label)
- Enrolment
- 48 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Bio-Path Holdings, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Gail J Roboz, MD, Principal investigator, Weill Cornell Medical College - New York-Presbyterian Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05190471
- Sponsor's protocol ID
- BP1002-102-AML
- Status last verified
- Mar 2025
- First submitted
- 8 Dec 2021
- Last updated
- 10 Mar 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No