FHD-286 With Low-Dose Weekly Decitabine/Venetoclax in Patients With Acute Myeloid Leukemia

NCT07283094 | Recruiting Phase 1 DMC FDA Drug

• 2 other identifiers: 2025-166612516661-FHD-286
• Study Type: interventional
• Target: 33
• Locations: 1 country
• Sites: 1

Brief Summary

This is a Phase 1, uncontrolled, single-arm, open-label, nonrandomized, dose escalation, study of Decitabine (DAC)+Venetoclax (VEN)+FHD-286 in participants with newly diagnosed Acute Myeloid Leukemia (AML) classified as adverse risk per the 2022 European Leukemia Net (ELN) criteria or AML that has progressed after one prior line of therapy.

Conditions

Acute Myeloid Leukemia; Myelodysplastic Syndrome

Outcome Measures

Primary Outcomes (3)

• Dose-limiting toxicities (DLTs)

  Dose limiting toxicity is defined as any adverse event (AE) that occurs during the DLT evaluation period that also meets any one of the criteria for hematologic and non-hematologic AEs as defined by the protocol and determined by the Data and Safety Monitoring Committee (DSMC), with input from the clinical study team. All AEs that cannot clearly be determined to be unrelated to FHD-286 or the combination of FHD-286 with DAC and VEN will be considered relevant to determining DLTs and any other emergent toxicities that are not explicitly defined by the DLT criteria to determine if any warrant a DLT designation, including toxicities that begin after the DLT evaluation period will be reviewed by the DSMC with input from the clinical study team. The percentage of participants with DLTs will be summarized by cohort.

  Through the 12-week induction period

• Frequency and severity of the adverse event of special interest (AESI)

  Differentiation syndrome is an adverse event of special interest for FHD-286. Suspected or confirmed differentiation syndrome will be reported, at minimum, as an important medical event. All Grade ≥2 events of Differentiation syndrome will be reported. The percentage of participants with DLTs will be summarized by cohort.

  Through the 12-week induction period

• Percentage of participants who are able to continue treatment

  The percentage of participants who are able to continue treatment without dose interruptions, reductions, or delays during the 12-week induction period will be summarized. Dose interruptions and delays are defined as delaying or interrupting treatment due to toxicity or intolerability for \>2 weeks.

  After 12-week induction period

Secondary Outcomes (6)

• Hemoglobin (Hgb) laboratory level for safety assessments

  Weekly during 12-week induction period

• Platelet (Plt) laboratory level for safety assessments

  Weekly during 12-week induction period

• Absolute Neutrophil Count (ANC) laboratory level for safety assessments

  Weekly during 12-week induction period

• Overall response rate (ORR)

  End of Cycle 1 (28 days) and end of Cycle 2 (56 days)

• Duration of response

  Up to 2 years

Study Arms (1)

Decitabine, Venetoclax, and FHD-286

EXPERIMENTAL

Administration: * Decitabine is reconstituted with 5 mL of sterile water and given by subcutaneous (SC) injection at the investigational site. * Venetoclax is taken as a tablet provided by the investigational site pharmacy or another authorized specialty pharmacy. * FHD-286 is taken as a capsule provided by the investigational site pharmacy.

Drug: Decitabine; Drug: Venetoclax; Drug: FHD-286

Interventions

Decitabine DRUG

Decitabine: 0.2 mg/kg/day subcutaneously once weekly (QW) (days 1, 8, 15, 22 \[±3 days\] of each 28-day cycle) \- A second weekly dose may be added if the investigator determines that more rapid debulking is required for a participant with high disease burden. The 2 weekly DAC doses should, preferably, be given on consecutive days

Decitabine, Venetoclax, and FHD-286

Venetoclax DRUG

Venetoclax: 400 mg orally (PO) (tablets) QW, concurrent with the first weekly DAC dose (days 1, 8, 15, and 22 \[±1 day\] of each 28-day cycle) * Refer to the United States Prescribing Information (USPI) ("steady daily dose") for details regarding VEN dosage modifications. When decitabine is held, venetoclax should also be held. Based on best clinical judgment, the investigator may continue decitabine while withholding VEN for several doses to allow for improved count recovery * If treatment with a P-gp inhibitor or triazole antifungal agent classified as a moderate CYP3A inhibitor is medically necessary, reduce the VEN dose by at least 50% * If treatment with posaconazole is medically necessary, reduce the VEN dose to 70 mg * If treatment with another triazole antifungal agent classified as a strong CYP3A inhibitor is medically necessary, reduce the VEN dose to 100 mg

Decitabine, Venetoclax, and FHD-286

FHD-286 DRUG

FHD-286: 2.5 or 5 mg (based on assigned dose group) PO (capsules) once daily (QD) 5 days/week (days 3-7, 10-14, 17-21, and 24-28 of each 28-day cycle) If acceptable safety and tolerability are observed at the end of cycle 1 with at least 3 DLT-evaluable participants in cohort 1 (FHD-286 2.5 mg QD), the dose of FHD-286 will be escalated to 5 mg QD for cohort 2. Doses of DAC and VEN will not change The 2 non-dosing days must be the day of and the day after the VEN dose * If necessary to improve tolerability and/or reduce toxicity, frequency of FHD-286 dosage may be reduced to 4 days/week * FHD-286 dose level will be escalated/de-escalated as described in protocol * If treatment with a strong CYP3A inhibitor is medically necessary, discussion with the PI is required and the FHD-286 dose should be reduced to 1.5 mg QD. Dose interruption or discontinuation of FHD-286 may also be necessary * See protocol for information on prohibited concomitant therapies when medically necessary

Decitabine, Venetoclax, and FHD-286

Eligibility Criteria

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

You may qualify if:

• Newly diagnosed adverse risk AML, including Myelodysplastic Syndrome/Acute Myeloid Leukemia (MDS/AML), per the 2022 ELN criteria, with a histopathologic diagnosis confirmed by hematopathology review OR AML that has progressed after 1 prior line of therapy with ≥5% blasts
• Aged ≥75 years, or aged 18-74 years and either refuse to receive intensive induction chemotherapy or are not a candidate for intensive induction chemotherapy due to one or more of the following comorbidities:
• Eastern Cooperative Oncology Group performance status (ECOG PS) of 2 or 3
• Cardiac history of congestive heart failure requiring treatment, ejection fraction ≤50%, or chronic stable angina pectoris
• Diffusing capacity of the lung for carbon monoxide ≤65% or forced expiratory volume in 1 second ≤65%
• Creatinine clearance ≥30 mL/min to \<45 mL/min
• Moderate hepatic impairment with total bilirubin \>1.5 to ≤3.0×upper limit of normal (ULN)
• Any other comorbidity that the investigator judges to be incompatible with intensive chemotherapy
• Bone marrow blasts ≥10%
• Have not received a hypomethylating agent (HMA) or VEN for their disease under study
• No other disease-directed therapy, except hydroxyurea or cytarabine, and including experimental or investigational drug therapy, for at least 14 days before study entry
• ECOG PS:
• New diagnosed AML:
• years: ≤2
• years to \<75 years: ≤3

You may not qualify if:

• Acute promyelocytic leukemia
• Core binding factor AML who is a candidate for intensive chemotherapy
• Eligible for and willing to receive standard HMA/VEN therapy (only applicable for individuals with newly diagnosed AML)
• Evidence (or suspicion) of central nervous system (CNS) involvement
• Prior treatment with azacitidine, DAC, VEN, or FHD-286. For individuals with AML that has progressed after 1 prior line of therapy, prior treatment with Azacitidine, Decitabine and VEN is allowed
• Currently pregnant or breast-feeding. Women of child-bearing potential (WOCBP) must have negative serum pregnancy test within 72 hours before treatment start. (NOTE: WOCBP is any biological female, regardless of sexual or gender orientation, having undergone tubal ligation, or remaining celibate by choice, who has not undergone a documented hysterectomy or bilateral oophorectomy or has had a menses any time in the preceding 12 months \[therefore not naturally post-menopausal for \>12 months\].)
• Planning to become pregnant within 1 year after start of study treatment
• Uncontrolled intercurrent illness that could limit life expectancy or ability to complete study correlates. This includes, but is not limited to:
• Uncontrolled concurrent malignancy
• Heart rate-corrected QT interval (QTc) by Fridericia method (QTcF) \>470 milliseconds (ms) or other factors that increase the risk of QTc prolongation. Participants with QTcF \>470 ms and bundle branch block and/or pacemaker rhythm may be considered for the study
• Congestive heart failure of New York Heart Association class III/IV. Individuals with compensated heart failure are permitted
• Unstable angina pectoris
• New or unstable cardiac arrhythmia. Patients with stable or controlled arrhythmias may be considered for the study.
• Decompensated liver cirrhosis (Child-Pugh score ≥12 or a Model for End-Stage Liver Disease (MELD) score ≥21)
• Psychiatric illness/social situation that would limit compliance with study requirements

Sponsors & Collaborators

• Montefiore Medical Center: lead
• Foghorn Therapeutics Inc.: collaborator

Study Sites (1)

Montefiore Medical Center

The Bronx, New York, 10467, United States

RECRUITING

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  BACKGROUND

• Dinardo CD, Kishtagari A, Ball B, et al: Preliminary safety, pharmacokinetic, and clinical activity results with FHD-286, a BRG1/BRM inhibitor, plus decitabine in a Phase 1 study in patients with relapsed or refractory myeloid malignancies. Presented at the EHA2025 Congress, Milan, Italy, 12-15 Jun, 2025

  BACKGROUND

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  BACKGROUND

• Ueda M, El-Jurdi N, Cooper B, Caimi P, Baer L, Kolk M, Brister L, Wald DN, Otegbeye F, Lazarus HM, Sandmaier BM, William B, Saunthararajah Y, Woost P, Jacobberger JW, de Lima M. Low-Dose Azacitidine with DNMT1 Level Monitoring to Treat Post-Transplantation Acute Myelogenous Leukemia or Myelodysplastic Syndrome Relapse. Biol Blood Marrow Transplant. 2019 Jun;25(6):1122-1127. doi: 10.1016/j.bbmt.2018.12.764. Epub 2018 Dec 30.

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MeSH Terms

Conditions

Leukemia, Myeloid, Acute; Myelodysplastic Syndromes

Interventions

Decitabine; venetoclax

Condition Hierarchy (Ancestors)

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

• Mendel R Goldfinger, MD

  Montefiore Medical Center

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Mendel R Goldfinger, MD

CONTACT

718-920-4826; mgoldfin@montefiore.org

Akash R Shah

CONTACT

718-862-8840; ashah1@montefiore.org

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Model Details: The study will enroll approximately 3-6 participants per cohort using a 3+3 design, resulting in a total of approximately 6-12 participants. During dose escalation, up to 5 additional participants per dose level may be enrolled at dose levels that have already been cleared. After a dose(s) has been selected for further study, additional participants may be enrolled to further explore safety or preliminary clinical activity.

Study Record Dates

• First Submitted: December 2, 2025
• First Posted: December 15, 2025
• Study Start: February 3, 2026
• Primary Completion (Estimated): May 1, 2028
• Study Completion (Estimated): February 1, 2030
• Last Updated: March 3, 2026

Record last verified: 2026-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)