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

NCT02781883 | Recruiting Phase 2 DMC

• 1 other identifier: BP1001-201-AML
• Study Type: interventional
• Target: 108
• Locations: 1 country
• Sites: 9

Brief Summary

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.

Conditions

Acute Myeloid Leukemia (AML)

Keywords

Liposomal Grb-2 treatment of AML; Liposomal Grb-2 with Venetoclax plus decitabine for AML

Outcome Measures

Primary Outcomes (2)

• Assessment of efficacy in untreated AML subjects by bone marrow aspirate or biopsy

  Assess whether the combination of BP1001 and Ventoclax plus decitabine provides greater efficacy (Complete Remission \[CR\], Complete Remission with incomplete hematologic recovery \[CRi\], Complete Remission with partial hematologic recovery \[CRh\], than Ventoclax + decitabine alone (by historical comparison) with untreated AML that cannot or elect not to be treated with more intensive chemotherapy

  180 days

• Assessment of efficacy in refractory/relapsed AML subjects by bone marrow aspirate or biopsy

  Assess whether BP1001-based treatment provides greater efficacy (CR, CRi, CRh) than intensive chemotherapy (by historical comparison) in participants with refractory/relapsed AML.

  180 days

Secondary Outcomes (5)

• Assessment of safety of BP1001 in combination with Ventoclax plus decitabine

  30 days

• Assessment of the pharmacokinetics (PK) of BP1001 when given in combination with Ventoclax plus decitabine

  30 days

• Assessment of Minimal Residual Disease (MRD) status in patients who achieve CR/CRi/CRh with BP1001-based treatment

  30 days

• Assessment of Partial Remissions and blast count reductions.

  30 days

• Assessment of overall survival

  180 days

Study Arms (3)

Untreated AML

EXPERIMENTAL

BP1001 in combination with Ventoclax plus decitabine

Drug: BP1001 in combination with Ventoclax plus decitabine

Refractory/Relapsed AML

EXPERIMENTAL

BP1001 in combination with Ventoclax plus decitabine

Drug: BP1001 in combination with Ventoclax plus decitabine

Refractory/Relapsed AML (ventoclax-intolerant or resistant)

EXPERIMENTAL

BP1001 + decitabine combination in patients who are resistant or intolerant of venetoclax-based treatment, or considered not optimal candidates for a venetoclax-based therapy.

Drug: BP1001 plus decitabine

Interventions

BP1001 in combination with Ventoclax plus decitabine DRUG

BP1001 in combination with Ventoclax plus decitabine

Also known as: Liposomal Grb-2, L-Grb-2

Refractory/Relapsed AML; Untreated AML

BP1001 plus decitabine DRUG

BP1001 plus decitabine in ventoclax intolerant or resistant subjects

Also known as: Liposomal Grb-2, L-Grb-2

Refractory/Relapsed AML (ventoclax-intolerant or resistant)

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• At the time of Screening, participants must meet all of the following criteria to be considered eligible to participate in the study:
• Adults ≥18 years of age
• 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
• 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
• Histologically documented diagnosis (based on the 2008 World Health Organization \[WHO\] Classification) (Vardiman et al. 2009) of one of the following:
• Newly diagnosed untreated AML; or
• Untreated secondary AML, including AML that has progressed from MDS
• 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))
• Relapsed or Refractory AML
• 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
• Eligible for venetoclax and decitabine therapy, based on Investigator assessment
• 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.
• 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;

You may not qualify if:

• At the time of Screening, participants who meet any of the following criteria will be excluded from participating in the study:
• 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.
• 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
• 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.
• Acute promyelocytic leukemia (APL) with t(15;17)(q22;q12) PML-RARA
• Chronic myeloid leukemia (CML) in any phase
• 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)
• Uncontrolled active, untreated, or progressive infection
• Receipt of any investigational agent or study treatment within 30 days prior to C1D1
• 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
• 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
• 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)
• History of any hypersensitivity to venetoclax or decitabine, unless reaction is deemed irrelevant to the study by the Investigator and Medical Monitor
• 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

Sponsors & Collaborators

• Bio-Path Holdings, Inc.: lead

Study Sites (9)

UCLA Medical Center

Los Angeles, California, 90095, United States

RECRUITING

Georgia Cancer Center at Augusta University

Augusta, Georgia, 30912, United States

RECRUITING

University of Kansas Cancer Center

Fairway, Kansas, 66205, United States

RECRUITING

New Jersey Hematology Oncology Associates

Brick, New Jersey, 08724, United States

TERMINATED

Laura & Isaac Pe lmutter Cancer Center at NYU Langone Health

New York, New York, 10016, United States

RECRUITING

Weill Cornell Medical College - New York - Presbyterian Hospital

New York, New York, 10021, United States

RECRUITING

University of Texas M.D. Anderson Cancer Center

Houston, Texas, 77030, United States

RECRUITING

Baylor Scott & White Research Institute

Temple, Texas, 76508, United States

TERMINATED

West Virginia University/Mary Babb Randolph Cancer Center

Morgantown, West Virginia, 26506, United States

TERMINATED

MeSH Terms

Conditions

Leukemia, Myeloid, Acute

Interventions

Decitabine

Condition Hierarchy (Ancestors)

Leukemia, Myeloid; Leukemia; Neoplasms by Histologic Type; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Azacitidine; Aza Compounds; Organic Chemicals; Cytidine; Pyrimidine Nucleosides; Pyrimidines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Nucleosides; Nucleic Acids, Nucleotides, and Nucleosides; Ribonucleosides

Study Officials

• Maro Ohanian, DO

  M.D. Anderson Cancer Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Maro Ohanian, DO

CONTACT

713-792-2631; mohanian@mdanderson.org

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 18, 2016
• First Posted: May 25, 2016
• Study Start: May 1, 2016
• Primary Completion (Estimated): December 1, 2027
• Study Completion (Estimated): December 1, 2028
• Last Updated: March 7, 2025

Record last verified: 2025-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (9)