AzTrials
AzTrials

Acute Myeloid Leukemia (AML)Phase 2NCT02781883

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

A Phase 2 study testing BP1001 in combination with Ventoclax plus decitabine and BP1001 plus decitabine in people with Acute Myeloid Leukemia (AML), run by Bio-Path Holdings, Inc. at 9 sites in United States.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
Search all trials

Trial 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… Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Acute Myeloid Leukemia (AML)
What is being tested?
BP1001 in combination with Ventoclax plus decitabine and BP1001 plus decitabine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
108 participants
Where
9 sitesUnited States
Study dates
May 2016to Dec 2028
Phase
Phase 2
Are there results?
Not yet: the study is under way

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

  • 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
Show all 42Show fewer
  • 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;
  • 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):
  • 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
  • Documented Eastern Cooperative Oncology Group (ECOG) performance status of 0, 1, or 2
  • Recovered from the effects of any prior surgery, radiotherapy, or antineoplastic treatment (with the exception of alopecia), based on Investigator assessment
  • 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:
  • 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
  • Uncontrolled seizure disorder (i.e., seizures within the past 2 months)
  • Cannot receive live attenuated vaccine immunization prior to, during, or after treatment with venetoclax until B-cell recovery occurs
  • Unable or unwilling to communicate or cooperate with the Investigator or follow the protocol for any reason

Who cannot join

None listed.

Trial locations and contacts

9 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Maro Ohanian, DO contact

713-792-2631

mohanian@mdanderson.org

9 sites in 1 country

United States9 sites, 6 recruiting

  • Georgia Cancer Center at Augusta University

    Augusta, Georgia, 30912

    Vamsi Kota, MD, 706-721-2505, vkota@augusta.edu

    Vamsi Kota, MD

    Recruiting

  • New Jersey Hematology Oncology Associates

    Brick, New Jersey, 08724

    Terminated

  • University of Kansas Cancer Center

    Fairway, Kansas, 66205

    Kerry Hepler, RN, 913-945-7552, khepler@kumc.edu

    Jecinta Scott, MS, 913-945-7505, jscott11@kumc.edu

    Recruiting

  • University of Texas M.D. Anderson Cancer Center

    Houston, Texas, 77030

    Maro Ohanian, DO, 713-792-2631, mohanian@mdanderson.org

    Maro Ohanian, MD

    Recruiting

  • UCLA Medical Center

    Los Angeles, California, 90095

    Bruck Habtemariam, 310-528-2565, BHabtemariam@mednet.ucla.edu

    Rika Galias, 310-206-5756, RGalias@mednet.ucla.edu

    Recruiting

  • West Virginia University/Mary Babb Randolph Cancer Center

    Morgantown, West Virginia, 26506

    Terminated

  • Laura & Isaac Pe lmutter Cancer Center at NYU Langone Health

    New York, New York, 10016

    Mohammad Maher Abdul Hay, MD, 646-501-4818, Maher.Abdulhay@nyumc.org

    Mohammad Maher Abdul Hay, MD

    Recruiting

  • Weill Cornell Medical College - New York - Presbyterian Hospital

    New York, New York, 10021

    Gail J Roboz, MD, 646-962-2700, gar2001@med.cornell.edu

    Gail J Roboz, MD

    Recruiting

  • Baylor Scott & White Research Institute

    Temple, Texas, 76508

    Terminated

Other trials in United States

About this study

Official title: 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

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.

Read the detailed description

Improvement of clinical benefit in fragile AML patients while maintaining tolerability is an important area of further clinical development. Modern aggressive combination chemotherapy can induce CR in a significant proportion of patients with previously untreated AML, but relapse occurs in most unless patients undergo intensive allogeneic hematopoietic stem cell transplantation. Novel therapies are needed for these patients

The Grb2 gene has been mapped to the human chromosome region 17q22-qter, a region that is duplicated in leukemias and solid tumors, which may result in an increased copy number of the Grb2 gene product. As Grb2 is important for the transformation of murine hematopoietic cells, and the proliferation of human leukemia cells that express high levels of oncogenic tyrosine kinases, inhibition of Grb2 may have a significant impact on the natural history of leukemias. The study drug (BP1001) may be able to inhibit the cells from making Grb-2. Researchers hope that without this protein, the leukemia cells will die.

This represents an area in which targeted therapies might be of benefit to these patients. One such potential treatment is BP1001, liposomal anti-sense treatment directed against Growth Factor Receptor-Bound Protein 2 (Grb2). Decitabine is approved in Europe for the treatment of adult patients with newly diagnosed de novo or secondary acute myeloid leukemia (AML). In vitro studies in AML cells co-incubated with BP1001 and decitabine suggests that treatment of AML patients with decitabine followed by BP1001 may be a combination that could benefit patients with AML.

This Phase IIa, multicenter, study of BP1001 in combination with Ventoclax plus decitabine will enroll participants with AML who are not otherwise eligible for for intensive induction therapy.

This trial will utilize an open label design to assess the safety profile, PK, PD, and efficacy of of BP1001 in combination with Ventoclax plus decitabine to assess whether the combination of either provides greater efficacy than intensive chemotherapy alone.

There are 3 cohorts exploring three-drug combinations of BP1001, venetoclax and decitabine.

  • Untreated AML patients will be treated with BP1001 plus venetoclax plus decitabine.
  • Refractory/relapsed AML patients will also be treated with BP1001 plus venetoclax plus decitabine.
  • A third cohort of BP1001 + decitabine is offered to refractory/relapsed AML patients who are venetoclax resistant or intolerant, or not considered by the investigator as optimal candidates for venetoclax-based therapy.

Each cohort will continue until approximately 19 evaluable participants have been investigated. At that point, enrollment will be placed on hold so that the Sponsor can perform an administrative review of the data to determine which treatment cohorts should continue with enrollment.

Should one or more cohorts continue with enrollment, the sample size will be increased up to 54 in the refractory/relapsed AML cohorts and 98 in the untreated AML cohort. These sample sizes for the study are based on the primary endpoint.

Being tested
BP1001 in combination with Ventoclax plus decitabine, BP1001 plus decitabine
Keywords
Liposomal Grb-2 treatment of AML, Liposomal Grb-2 with Venetoclax plus decitabine for AML
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.

  • BP1001 in combination with Ventoclax plus decitabineDrug

    Also called Liposomal Grb-2, L-Grb-2

    BP1001 in combination with Ventoclax plus decitabine

  • BP1001 plus decitabineDrug

    Also called Liposomal Grb-2, L-Grb-2

    BP1001 plus decitabine in ventoclax intolerant or resistant subjects

Study groups in NCT02781883
Study groupRoleWhat they receive
Untreated AMLExperimentalBP1001 in combination with Ventoclax plus decitabine
Receives: Drug: BP1001 in combination with Ventoclax plus decitabine
Refractory/Relapsed AMLExperimentalBP1001 in combination with Ventoclax plus decitabine
Receives: Drug: BP1001 in combination with Ventoclax plus decitabine
Refractory/Relapsed AML (ventoclax-intolerant or resistant)ExperimentalBP1001 + decitabine combination in patients who are resistant or intolerant of venetoclax-based treatment, or considered not optimal candidates for a venetoclax-based therapy.
Receives: Drug: BP1001 plus decitabine

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. 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

    Measured: 180 days

  2. 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.

    Measured: 180 days

Secondary outcomes (5)

  1. Assessment of safety of BP1001 in combination with Ventoclax plus decitabine

    Evaluate safety and toxicity of the combination of BP1001 with Ventoclax plus decitabine using non-hematologic and hematologic measures per NCI CTCAE criteria (with a safety run-in)

    Measured: 30 days

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

    Evaluate the in vivo PK using plasma to compute half life and elimination.

    Measured: 30 days

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

    Assess time to response from administration of BP1001-based treatment to CR, CRi, or CRh, negative MRD status as defined by the site and as available (Usually ≤ 0.1% by multi-parameter flow cytometry).

    Measured: 30 days

  4. Assessment of Partial Remissions and blast count reductions.

    Assessment of Partial Remissions (per Cheson, 2003), and blast count reductions (per Williams, 2016)

    Measured: 30 days

  5. Assessment of overall survival

    To assess event-free survival, overall survival, time to response, 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.

  1. First posted25 May 2016 (estimated)
  2. Study startMay 2016
  3. Primary completionDec 2027 (estimated)
  4. Study completionDec 2028 (estimated)

Resources

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 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
108 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02781883
Sponsor's protocol ID
BP1001-201-AML
Status last verified
Mar 2025
First submitted
18 May 2016
Last updated
7 Mar 2025
Results posted
No
Data monitoring committee
Yes
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing BP1001 in combination with Ventoclax plus decitabine and BP1001 plus decitabine in people with Acute Myeloid Leukemia (AML), run by Bio-Path Holdings, Inc. at 9 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 42 inclusion and 0 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: looking for participants now, as of the sponsor's last update on 7 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 9 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Maro Ohanian, DO (713-792-2631, mohanian@mdanderson.org), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Assessment of efficacy in untreated AML subjects by bone marrow aspirate or biopsy (time frame: 180 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02781883.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 7 Mar 2025.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in