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Updated June 2026 · ClinicalTrials.gov

RECRUITINGPhase 2INTERVENTIONAL

Clinical Trial of BP1001 in Combination With With Venetoclax Plus Decitabine in AML

A Phase IIa, Open-label, Clinical Trial to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of BP1001 (a Liposomal Grb2 Antisense Oligonucleotide) in Combination With Venetoclax Plus Decitabine in Patients With AML Who Are Ineligible for Intensive Induction Therapy

Clinical Trial of BP1001 in Combination With With Venetoclax Plus Decitabine in AML (NCT02781883) is a Phase 2 interventional studying Acute Myeloid Leukemia (AML), sponsored by Bio-Path Holdings, Inc.. RECRUITING as of the most recent ClinicalTrials.gov update. Talk to your doctor before contacting the trial site.

Important: This information is not medical advice. Talk to your doctor about whether a clinical trial is right for you.

About This Trial

The primary objectives of this study are to assess: (1) whether the combination of BP1001 plus venetoclax plus decitabine provides greater efficacy (Complete Remission \[CR\], Complete Remission with incomplete hematologic recovery \[CRi\], Complete Remission with partial hematologic recovery \[CRh\], than venetoclax plus decitabine alone (by historical comparison) in participants with untreated AML that cannot or elect not to be treated with more intensive chemotherapy; (2) whether BP1001-based treatment provides greater efficacy (CR, CRi, CRh) than intensive chemotherapy (by historical comparison) in participants with refractory/relapsed AML.

What Stage of Research Is This?

Phase 2 trials evaluate whether a treatment actually works against Acute Myeloid Leukemia (AML) and continue monitoring side effects. Phase 2 enrolls larger groups (typically 100–300 patients) and produces the first real efficacy signal. A successful Phase 2 readout is what unlocks the much larger Phase 3 confirmatory trials needed for FDA approval.

This trial is currently recruiting participants. The sponsor has registered the study with ClinicalTrials.gov as actively enrolling, which means new applicants who meet the eligibility criteria can be considered for screening. Trial status can change between updates — confirm current recruiting status with the study contact before traveling for a screening visit.

Target enrollment of 108 participants puts this in the typical range for a Phase 2-style efficacy study or a moderate Phase 3 trial in a focused Acute Myeloid Leukemia (AML) subpopulation. At this scale, the study has enough statistical power to detect a clear treatment effect but is not the largest cohort in the field.

Who May Be Eligible (Plain English)

Inclusion Criteria At the time of Screening, participants must meet all of the following criteria to be considered eligible to participate in the study: 1. Adults ≥18 years of age 2. Females must be of non-childbearing potential, surgically sterile, postmenopausal, or practice adequate methods of contraception during the study and for 30 days after the last dose of study drug or decitabine 3. Males must agree to use an adequate method of contraception during the study and for at least 30 days after the last dose of study drug or decitabine 4. Histologically documented diagnosis (based on the 2008 World Health Organization \[WHO\] Classification) (Vardiman et al. 2009) of one of the following: 1. Newly diagnosed untreated AML; or 2. Untreated secondary AML, including AML that has progressed from MDS 3. In some cases of AML associated with specific genetic abnormalities, however, the diagnosis of AML may be made if the blast count is less than 20% (Dohner et al. 2017) - specifically AML with t(15;17), t(8;21), inv(16), or t(16;16)) 4. Relapsed or Refractory AML 5. Investigator considers previously untreated participant ineligible for (or unwilling to receive) intensive induction therapy based on medical reasons, disease characteristics such as genetics, type of AML (untreated or secondary), or participant characteristics such as age, performance status, co-morbidities, organ dysfunctions, or patient election of low-intensity treatment 6. Eligible for venetoclax and decitabine therapy, based on Investigator assessment 7. Participant's WBC count is 25 x 10\^9/L or less at study initiation. The use of leukapheresis or hydroxyurea before treatment initiation to achieve this is permitted. 8. 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 ...See full criteria on ClinicalTrials.gov Always talk to your doctor about whether this trial is right for you.

These are translations of the protocol\'s inclusion and exclusion criteria, simplified for patients and caregivers. The original clinical text appears below. Eligibility is ultimately confirmed by the trial site\'s screening process — this summary is a starting point for a conversation with your doctor, not a final determination.

Original Eligibility Criteria

View original clinical language
Inclusion Criteria At the time of Screening, participants must meet all of the following criteria to be considered eligible to participate in the study: 1. Adults ≥18 years of age 2. Females must be of non-childbearing potential, surgically sterile, postmenopausal, or practice adequate methods of contraception during the study and for 30 days after the last dose of study drug or decitabine 3. Males must agree to use an adequate method of contraception during the study and for at least 30 days after the last dose of study drug or decitabine 4. Histologically documented diagnosis (based on the 2008 World Health Organization \[WHO\] Classification) (Vardiman et al. 2009) of one of the following: 1. Newly diagnosed untreated AML; or 2. Untreated secondary AML, including AML that has progressed from MDS 3. In some cases of AML associated with specific genetic abnormalities, however, the diagnosis of AML may be made if the blast count is less than 20% (Dohner et al. 2017) - specifically AML with t(15;17), t(8;21), inv(16), or t(16;16)) 4. Relapsed or Refractory AML 5. Investigator considers previously untreated participant ineligible for (or unwilling to receive) intensive induction therapy based on medical reasons, disease characteristics such as genetics, type of AML (untreated or secondary), or participant characteristics such as age, performance status, co-morbidities, organ dysfunctions, or patient election of low-intensity treatment 6. Eligible for venetoclax and decitabine therapy, based on Investigator assessment 7. Participant's WBC count is 25 x 10\^9/L or less at study initiation. The use of leukapheresis or hydroxyurea before treatment initiation to achieve this is permitted. 8. 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 glomerular filtration rate (eGFR) of at least 40 ml/min. These estimations can be calculated using any of the following methods (Appendix E: Formulas): i. Chronic Kidney Disease Epidemiology Collaboration (CKD-Epi) equation * GFR = 141 × min (Scr /κ, 1)\^α × max(Scr /κ, 1)\^-1.209 × 0.993\^Age × 1.018 \[if female\] × 1.159 \[if black\] ii. Cockcroft gault equation * Cockcroft Gault equation utilizing the TBW (Total body weight) to calculate an estimated creatinine clearance * CrCl = \[(140 - age) x TBW\] / (Scr x 72) x 0.85 \[if female\] iii. Modification of Diet in Renal Disease (MDRD) Study equation * GFR (mL/min/1.73 m\^2) = 175 × (Scr)\^-1.154 × (Age)\^-0.203 × 0.74 \[if female\] x 1.212 \[if African American (AA)\] iv. Creatinine clearance estimated by 24-hr urine collection for creatinine clearance 9. Documented Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2 10. Recovered from the effects of any prior surgery, radiotherapy, or antineoplastic treatment (with the exception of alopecia), based on Investigator assessment 11. Willing and able to provide written informed consent 7.2. Exclusion Criteria At the time of Screening, participants who meet any of the following criteria will be excluded from participating in the study: 1. Active non-hematologic or lymphoid malignancy other than AML treated with immuno- or chemotherapy within the previous 12 months except active non- melanoma, non-invasive skin cancer will be allowed. 2. Known, active leptomeningeal leukemia requiring intrathecal therapy. NOTE: Patients 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 extramedullary leukemia without also meeting bone marrow criteria for acute leukemia (for AML usually = 20% blasts in BMA or BMB). Patients may have leukemia with lower blast counts (Dohner et al. 2010). Bio-Path Holdings and Investigator concurrence required. 4. Acute promyelocytic leukemia (APL) with t(15;17)(q22;q12) PML-RARA 5. Chronic myeloid leukemia (CML) in any phase 6. Receipt of any anti-cancer therapy within 14 days prior to C1D1, with the exception of hydroxyurea or anagrelide (within 24 hours), TKI (within 1day), a single dose of cytarabine (for proliferative disease) 7. Uncontrolled active, untreated, or progressive infection 8. Receipt of any investigational agent or study treatment within 30 days prior to C1D1 9. Females who are capable of becoming pregnant, test positive for pregnancy, or are breast-feeding during the Screening period, or intend to become pregnant or breast-feed during the course of the study or within 30 days after last dose of study drug 10. Serious intercurrent medical or psychiatric illness which, in the opinion of the Investigator, would interfere with the ability of the participant to complete the study 11. Known active or clinically significant hepatitis B infection (based on positive surface antigen \[HBsAg\]), hepatitis C infection (based on positive antibody \[HCV Ab\]), or human immunodeficiency virus (HIV-1 or HIV-2, based on positive antibody) 12. History of any hypersensitivity to venetoclax or decitabine, unless reaction is deemed irrelevant to the study by the Investigator and Medical Monitor 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. Cannot receive live attenuated vaccine immunization prior to, during, or after treatment with venetoclax until B-cell recovery occurs 17. Unable or unwilling to communicate or cooperate with the Investigator or follow the protocol for any reason

Treatments Being Tested

DRUG

BP1001 in combination with Ventoclax plus decitabine

BP1001 in combination with Ventoclax plus decitabine

DRUG

BP1001 plus decitabine

BP1001 plus decitabine in ventoclax intolerant or resistant subjects

Locations (9)

Trial sites listed on ClinicalTrials.gov for this study. Site activation status can vary — confirm with the specific site before traveling for a screening visit.

UCLA Medical Center
Los Angeles, California, United States
Georgia Cancer Center at Augusta University
Augusta, Georgia, United States
University of Kansas Cancer Center
Fairway, Kansas, United States
New Jersey Hematology Oncology Associates
Brick, New Jersey, United States
Laura & Isaac Pe lmutter Cancer Center at NYU Langone Health
New York, New York, United States
Weill Cornell Medical College - New York - Presbyterian Hospital
New York, New York, United States
University of Texas M.D. Anderson Cancer Center
Houston, Texas, United States
Baylor Scott & White Research Institute
Temple, Texas, United States
West Virginia University/Mary Babb Randolph Cancer Center
Morgantown, West Virginia, United States

How to Talk to Your Doctor About This Trial

Bring the printable summary of this trial — including the NCT ID (NCT02781883), the sponsor (Bio-Path Holdings, Inc.), and the key eligibility criteria — to your next appointment. Your doctor can review the inclusion and exclusion criteria against your medical history, lab values, and current treatments to assess whether you are likely to qualify. They can also help you weigh whether trial participation makes sense alongside your existing care plan.

Useful questions to walk through together: What does the trial protocol require beyond standard care? How long is the active treatment phase, and how long is follow-up? Are there study visits at sites I can reach? Who pays for the trial-specific procedures, and who pays for standard-of-care portions? See our 25 questions to ask about clinical trials guide for a more complete checklist.

Authoritative Sources

The official record for this trial lives on ClinicalTrials.gov — the federal registry maintained by the National Library of Medicine at NIH. For background on how this trial fits into the FDA approval pathway, see the FDA drug approval process. For oncology-specific guidance for patients considering trials, the National Cancer Institute publishes patient-oriented overviews. International trial registries are aggregated by the WHO ICTRP.

Frequently Asked Questions

What is the NCT02781883 clinical trial studying?

The primary objectives of this study are to assess: (1) whether the combination of BP1001 plus venetoclax plus decitabine provides greater efficacy (Complete Remission \[CR\], Complete Remission with incomplete hematologic recovery \[CRi\], Complete Remission with partial hematologic recovery \[CRh\], than venetoclax plus decitabine alone (by historical comparison) in participants with untreated AML that cannot or elect not to be treated with more intensive chemotherapy; (2) whether BP1001-based treatment provides greater efficacy (CR, CRi, CRh) than intensive chemotherapy (by historical compa… The full protocol is registered on ClinicalTrials.gov and includes the primary outcome measures, eligibility criteria, and study endpoints.

Who can participate in NCT02781883?

Eligibility for this trial depends on the specific inclusion and exclusion criteria set by the sponsor. The plain-English summary above translates the most important criteria into accessible language; the official clinical text is preserved in the collapsible section underneath. Whether you fit any specific trial is a medical decision your doctor needs to confirm — bring the trial information to your treating physician for a full review against your medical history.

How do I contact the trial site for NCT02781883?

Contact information registered with ClinicalTrials.gov is shown in the sidebar of this page. Before reaching out, confirm with your treating physician that this trial is appropriate for your situation. The trial site will then walk you through the screening process to determine final eligibility.

Is participating in a clinical trial safe?

Clinical trials in the United States are regulated by the FDA and overseen by Institutional Review Boards (IRBs) that review the protocol for safety. Risk varies by trial — Phase 1 studies test new treatments in humans for the first time, while Phase 3 trials use treatments that have already passed earlier safety screening. The informed consent document for any specific trial details the known risks and what to expect. Discuss those risks with your physician before deciding whether to participate.

Where can I verify the data on this page?

Every detail on this page comes directly from the ClinicalTrials.gov API. Click "View on ClinicalTrials.gov" in the sidebar to see the official, unmodified record. The federal record is always authoritative; this page is a structured presentation with a plain-English eligibility translation. For background on how clinical trials are regulated, see the FDA drug approval process documentation.

How This Page Is Built

Every field on this page is pulled directly from the ClinicalTrials.gov API v2 — no estimates, no proxies. The plain-English eligibility translation is generated from the original protocol text and reviewed for fidelity to the underlying clinical criteria. The original clinical text remains visible in the collapsible section above so users and clinicians can verify the translation. Read the full methodology for the data pipeline and known limitations.

Source: ClinicalTrials.gov API v2 record for NCT02781883. Maintained by the National Library of Medicine at NIH. Public domain. Cite as: "TrialFinderData. NCT02781883. Data: ClinicalTrials.gov."

Medical disclaimer: This page is informational, not medical advice. Talk to your doctor about whether a clinical trial is right for you.

Last updated 2026-06-26 · Data from ClinicalTrials.gov.