Advanced Hematologic MalignancyPhase 1NCT04891757
FHD-286 as Monotherapy or Combination Therapy in Subjects With Advanced Hematologic Malignancies
A Phase 1 study testing FHD-286, Low Dose Cytarabine and Decitabine in people with Advanced Hematologic Malignancy and Relapsed Acute Myeloid Leukemia, run by Foghorn Therapeutics Inc. at 5 sites in United States.
Trial summary
This Phase 1, multicenter, open-label, dose escalation study is designed to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of FHD-286 administered orally as monotherapy or combination therapy, in subjects with advanced hematologic malignancies. Read more about this study
- Who is it for?
- Ages 16 years and older
- Conditions the study is for
- Advanced Hematologic Malignancy, Relapsed Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia
- What is being tested?
- FHD-286, Low Dose Cytarabine and Decitabine
- Type of treatment
- Pill or oral medicine, Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 144 participants
- Where
- 5 sitesUnited States
- Study dates
- 14 Jun 2021to Jun 2027
- Phase
- Phase 1
- 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 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Subject must be ≥16 years of age.
- Subject must:
- Have a confirmed diagnosis of R/R AML, R/R MDS, or R/R CMML not in blast crisis AND
- Have received ≤4 prior lines of systemic anticancer therapy for their disease under study; subjects who have received >4 prior lines of systemic anticancer therapy for their disease under study must receive Sponsor approval. AND
- Be an appropriate candidate for treatment with LDAC (Arm A) or decitabine (Arm B)
- Subject or their parent or legal guardian (when applicable) must be able to understand and be willing to sign an informed consent and, when applicable, subject must sign an assent form.
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- Subject must be willing and able to comply with scheduled study visits and treatment plans.
- Subject must be willing to undergo all study procedures unless contraindicated due to medical risk.
- Subject must have an ECOG PS of ≤2.
- Subject must have a life expectancy of ≥3 months.
- Subject must have adequate hepatic function.
- Subject must have adequate renal function.
- Subject must have a WBC count ≤20×109/L; treatment with a stable dose of hydroxyurea or other cytoreductive agent (eg, cytarabine) to achieve this count is allowed.
- Subject must have adequate cardiovascular, respiratory, and immune system function.
- Subject must agree to abide by dietary and other considerations required during the study.
- Subject must meet timing requirements with respect to prior therapy and surgery
- Toxicity related to prior therapy must have returned to Grade ≤2 by CTCAE by approximately 14 days before the start of study treatment or be deemed irreversible and stable by the Investigator. Exceptions include alopecia, neuropathy, appropriately controlled endocrine toxicities, and other well-controlled stable toxicities with discussion with the Sponsor.
- Female subjects must be:
- postmenopausal; or
- permanently sterile, or, if sexually active with male partners, these partners must be azoospermic; or
- nonpregnant, nonlactating, and, if sexually active with fertile male partners, having agreed to use a highly effective method of contraception
- Male subjects must have documented azoospermia or, if fertile and sexually active, must agree to use a highly effective method of contraception with their partners of childbearing potential
Who cannot join
- Subject is unable to provide informed consent and/or to follow protocol requirements.
- Subject:
- Has undergone chimeric antigen receptor T cell therapy or HSCT within 60 days of the first dose of study treatment OR
- Has clinically significant GVHD
- Subject has evidence (or suspicion) of extramedullary involvement, unless approved by Sponsor.
- Subject has an immediately life-threatening, severe complication(s) of advanced myeloid malignancy, such as uncontrolled bleeding, pneumonia with hypoxia or shock, and/or disseminated intravascular coagulation.
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- Subject has other malignancy that may interfere with the diagnosis and/or treatment of advanced hematologic malignancies.
- Subject has active HBV or HCV infections; Subject has known positive HIV antibody results, or AIDS-related illness.
- Subject has an active severe infection that requires anti-infective therapy or has an unexplained temperature of >38.5°C during screening visits or on their first day of study treatment.
- Subject has an uncontrolled intercurrent illness.
- Subject has QTcF >470 msec or other factors that increase the risk of QTc prolongation or arrhythmic events.
- Subject has any other medical or psychological condition, deemed by the Investigator to be likely to interfere with a subject's ability to sign informed consent/assent, cooperate, or participate in the study.
- Subject has known allergies or hypersensitivities to:
- All subjects: components of the FHD-286 formulation
- Arm A: cytarabine or any of the excipients
- Arm B: decitabine or any of the excipients
- Subject is unable to tolerate the administration of oral medication or has GI dysfunction that would preclude adequate absorption, distribution, metabolism, or excretion of FHD-286.
- Subject is receiving any other anticancer investigational agents. Investigational agents to treat non-cancer indications may be permitted with Sponsor approval.
- Exclusion Criteria #14 was removed.
- Subject is on medications classified as:
- Strong CYP3A inhibitors [Exception: Triazole antifungal agents, including those classified as strong CYP3A inhibitors, are permitted.]
- Strong CYP3A inducers
- Sensitive CYP3A substrates with narrow TIs [Stable doses of immunosuppressant medications that are sensitive CYP3A4 substrates may be permitted with Sponsor approval.]
- Subject is on medications with narrow TIs that are sensitive P-gp or BCRP substrates and are administered orally or on medications classified as strong inhibitors of P-gp or BCRP.
- Administration of PPIs should be stopped or switched to another ARA 7 days before administration of FHD-286.
- Subject is requiring clinically significant or increasing doses of systemic steroid therapy or any other systemic immunosuppressive medication. Local or targeted steroid and immunosuppressive therapies are acceptable. Appropriate steroid replacement to manage endocrine toxicities resulting from prior anticancer systemic therapy is permitted.
- Subject has undergone any prior treatment with a BRG1/BRM inhibitor.
- Subject is pregnant or breastfeeding or is planning to become pregnant within 1 year of the start of study treatment.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Dana Farber Cancer Institute
Boston, Massachusetts, 02215
City of Hope National Medical Center
Duarte, California, 91010
MD Anderson Cancer Center
Houston, Texas, 77030
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
Memorial Sloan Kettering Cancer Center
New York, New York, 10065
About this study
Official title: A Phase 1, Multicenter, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Clinical Activity of Orally Administered FHD-286, as Monotherapy or Combination Therapy, in Subjects With Advanced Hematologic Malignancies
This Phase 1, multicenter, open-label, dose escalation study is designed to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of FHD-286 administered orally as monotherapy or combination therapy, in subjects with advanced hematologic malignancies.
Read the detailed description
This study is primarily intended to evaluate the safety and tolerability of FHD-286 when administered orally as monotherapy or in combination with either LDAC or decitabine to subjects with R/R AML, R/R MDS, and R/R CMML not in blast crisis. In each arm of the study, successive cohorts of participants will receive increasing oral doses of FHD-286 as a single agent or in combination with LDAC or decitabine to determine the RP2D(s) in this population.
The data from this study in subjects with advanced hematologic malignancies, including safety, tolerability, PK/PD findings, and antitumor activity, will form the basis for subsequent clinical development of FHD-286.
- Conditions
- Advanced Hematologic Malignancy, Relapsed Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia, Relapsed Myelodysplastic Syndromes, Refractory Myelodysplastic Syndromes, Relapsed Chronic Myelomonocytic Leukemia
- Being tested
- FHD-286, Low Dose Cytarabine, Decitabine
- Keywords
- AML, Relapsed Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia, MDS, Relapsed Myelodysplastic Syndromes, Refractory Myelodysplastic Syndromes, Phase 1, FHD-286, Foghorn, CMML, Refractory Chronic Myelomonocytic Leukemia, Relapsed Chronic Myelomonocytic Leukemia
- Medical subject headings
- Leukemia, Myeloid, Acute, Myelodysplastic Syndromes, Leukemia, Myelomonocytic, Chronic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FHD-286Drug
FHD-286 administered orally
Low Dose CytarabineDrug
Also called LDAC
LDAC administered subcutaneously (SC)
DecitabineDrug
Decitabine administered intravenously
| Study group | Role | What they receive |
|---|---|---|
| FHD-286 Monotherapy | Experimental | Closed to Enrollment Receives: Drug: FHD-286 |
| FHD-286 in Combination with LDAC | Experimental | FHD-286 administered orally + LDAC administered subcutaneously Receives: Drug: FHD-286; Drug: Low Dose Cytarabine |
| FHD-286 in Combination with Decitabine | Experimental | FHD-286 administered orally + decitabine administered intravenously (IV) Receives: Drug: FHD-286; Drug: Decitabine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of AEs, dose-limiting toxicities (DLTs), serious AEs (SAEs), AEs leading to discontinuation, and adverse events of special interest (AESIs); safety laboratory assessments
Measured: Up to 18 months
Secondary outcomes (18)
AML: Complete remission (CR) rate
Measured: Up to 18 months
AML: Duration of CR
Measured: Up 18 months
AML: CR + CR with partial hematologic recovery (CRh) rate
Measured: Up 18 months
AML: Duration of CR + CRh
Measured: Up 18 months
AML: Transfusion independence rate
Measured: Up 18 months
AML: Event free survival (EFS)
Measured: Up 42 months
AML: Overall survival (OS)
Measured: Up to 42 months
MDS: CR rate
Measured: Up to 18 months
MDS & CMML: Duration of CR
Measured: Up to 18 months
MDS & CMML: Partial remission (PR) rate
Measured: Up to 18 months
MDS & CMML: Duration of PR
Measured: Up to 18 months
MDS & CMML: CR + PR
Measured: Up to 18 months
MDS & CMML: Duration of CR + PR
Measured: Up to 18 months
MDS & CMML: Hematologic Improvement rate
Measured: Up to 18 months
MDS & CMML: EFS
Measured: Up to 42 months
MDS: OS
Measured: Up to 42 months
PK parameter: Area under the plasma concentration time curve (AUC)
Measured: Day 1 and day 8 of cycle 1 (each cycle is 28 days)
Plasma concentration vs. time profiles
Measured: Day 1 and day 8 of cycle 1 (each cycle is 28 days)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted18 May 2021
- Study start14 Jun 2021
- Primary completionSep 2025 (estimated)
- Study completionJun 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Advanced Hematologic MalignancyStudies 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
- Single group
- Masking
- None (open label)
- Enrolment
- 144 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Foghorn Therapeutics Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sarah Reilly, MD, Study director, Foghorn Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04891757
- Sponsor's protocol ID
- FHD-286-C-002
- Status last verified
- Mar 2025
- First submitted
- 13 May 2021
- Last updated
- 10 Jul 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
DiNardo CD, Fathi AT, Kishtagari A, Bhalla KN, Quintas-Cardama A, Reilly SA, Almon C, Patriquin C, Nabhan S, Healy K, Hickman D, Collins MP, Khalil A, Corrigan D, Zhao T, Piel J, Lyons K, Horrigan K, Schuck V, Martin P, Elliott G, Lahr DL, Bosinger M, D'Aco K, Smolen GA, Hentemann M, Loghavi S, Agresta S, Savona MR, Stein EM. A Phase I Study of FHD-286, a Dual BRG1/BRM (SMARCA4/SMARCA2) Inhibitor, in Patients with Advanced Myeloid Malignancies. Clin Cancer Res. 2025 Jun 13;31(12):2327-2338. doi: 10.1158/1078-0432.CCR-24-3790.
PubMed 40238563
Study at a glance
From the registry record, last updated by the sponsor on 10 Jul 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1, Multicenter, Open-Label, Dose Escalation Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Clinical Activity of Orally Administered FHD-286, as Monotherapy or Combination Therapy, in Subjects With Advanced Hematologic Malignancies
- ClinicalTrials.gov ID
- NCT04891757
- Acronym / sponsor ID
- FHD-286-C-002
- Conditions
- Advanced Hematologic Malignancy, Relapsed Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia, Relapsed Myelodysplastic Syndromes, Refractory Myelodysplastic Syndromes, Relapsed Chronic Myelomonocytic Leukemia
- Keywords
- AML, Relapsed Acute Myeloid Leukemia, Refractory Acute Myeloid Leukemia, MDS, Relapsed Myelodysplastic Syndromes, Refractory Myelodysplastic Syndromes, Phase 1, FHD-286
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 144 participants (estimated)
- Lead sponsor
- Foghorn Therapeutics Inc.Industry (pharma, biotech, devices)
- Study start
- 14 Jun 2021 (actual)
- Primary completion
- Sep 2025 (estimated)
- Study completion
- Jun 2027 (estimated)
- First posted
- 18 May 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This Phase 1, multicenter, open-label, dose escalation study is designed to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of FHD-286 administered orally as monotherapy or combination therapy, in subjects with advanced hematologic malignancies.
Detailed description
This study is primarily intended to evaluate the safety and tolerability of FHD-286 when administered orally as monotherapy or in combination with either LDAC or decitabine to subjects with R/R AML, R/R MDS, and R/R CMML not in blast crisis. In each arm of the study, successive cohorts of participants will receive increasing oral doses of FHD-286 as a single agent or in combination with LDAC or decitabine to determine the RP2D(s) in this population.
The data from this study in subjects with advanced hematologic malignancies, including safety, tolerability, PK/PD findings, and antitumor activity, will form the basis for subsequent clinical development of FHD-286.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| FHD-286 Monotherapy | Experimental | Drug: FHD-286Closed to Enrollment |
| FHD-286 in Combination with LDAC | Experimental | Drug: FHD-286, Drug: Low Dose CytarabineFHD-286 administered orally + LDAC administered subcutaneously |
| FHD-286 in Combination with Decitabine | Experimental | Drug: FHD-286, Drug: DecitabineFHD-286 administered orally + decitabine administered intravenously (IV) |
Who may qualify
- Ages
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (22 listed)
- Subject must be ≥16 years of age.
- Subject must:
- Subject or their parent or legal guardian (when applicable) must be able to understand and be willing to sign an informed consent and, when applicable, subject must sign an assent form.
- Subject must be willing and able to comply with scheduled study visits and treatment plans.
- Subject must be willing to undergo all study procedures unless contraindicated due to medical risk.
Key exclusion criteria (28 listed)
- Subject is unable to provide informed consent and/or to follow protocol requirements.
- Subject:
- Subject has evidence (or suspicion) of extramedullary involvement, unless approved by Sponsor.
- Subject has an immediately life-threatening, severe complication(s) of advanced myeloid malignancy, such as uncontrolled bleeding, pneumonia with hypoxia or shock, and/or disseminated intravascular coagulation.
- Subject has other malignancy that may interfere with the diagnosis and/or treatment of advanced hematologic malignancies.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| FHD-286FHD-286 administered orally | Drug | FHD-286 Monotherapy, FHD-286 in Combination with Decitabine, FHD-286 in Combination with LDAC |
| Low Dose CytarabineAlso: LDACLDAC administered subcutaneously (SC) | Drug | FHD-286 in Combination with LDAC |
| DecitabineDecitabine administered intravenously | Drug | FHD-286 in Combination with Decitabine |
Primary outcome
Incidence of AEs, dose-limiting toxicities (DLTs), serious AEs (SAEs), AEs leading to discontinuation, and adverse events of special interest (AESIs); safety laboratory assessments
Time frame: Up to 18 months
18 secondary outcomes
AML: Complete remission (CR) rate
Time frame: Up to 18 months
AML: Duration of CR
Time frame: Up 18 months
AML: CR + CR with partial hematologic recovery (CRh) rate
Time frame: Up 18 months
AML: Duration of CR + CRh
Time frame: Up 18 months
AML: Transfusion independence rate
Time frame: Up 18 months
AML: Event free survival (EFS)
Time frame: Up 42 months
AML: Overall survival (OS)
Time frame: Up to 42 months
MDS: CR rate
Time frame: Up to 18 months
MDS & CMML: Duration of CR
Time frame: Up to 18 months
MDS & CMML: Partial remission (PR) rate
Time frame: Up to 18 months
MDS & CMML: Duration of PR
Time frame: Up to 18 months
MDS & CMML: CR + PR
Time frame: Up to 18 months
MDS & CMML: Duration of CR + PR
Time frame: Up to 18 months
MDS & CMML: Hematologic Improvement rate
Time frame: Up to 18 months
MDS & CMML: EFS
Time frame: Up to 42 months
MDS: OS
Time frame: Up to 42 months
PK parameter: Area under the plasma concentration time curve (AUC)
Time frame: Day 1 and day 8 of cycle 1 (each cycle is 28 days)
Plasma concentration vs. time profiles
Time frame: Day 1 and day 8 of cycle 1 (each cycle is 28 days)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jul 2025.
- Results publication: PubMed 40238563DiNardo CD, Fathi AT, Kishtagari A, Bhalla KN, Quintas-Cardama A, Reilly SA, Almon C, Patriquin C, Nabhan S, Healy K, Hickman D, Collins MP, Khalil A, Corrigan D, Zhao T, Piel J, Lyons K, Horrigan K, Schuck V, Martin P, Elliott G, Lahr DL, Bosinger M, D'Aco K, Smolen GA, Hentemann M, Loghavi S, Agresta S, Savona MR, Stein EM. A Phase I Study of FHD-286, a Dual BRG1/BRM (SMARCA4/SMARCA2) Inhibitor, in Patients with Advanced Myeloid Malignancies. Clin Cancer Res. 2025 Jun 13;31(12):2327-2338. doi: 10.1158/1078-0432.CCR-24-3790.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Sarah Reilly, MD study director
Foghorn Therapeutics
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing FHD-286, Low Dose Cytarabine and Decitabine in people with Advanced Hematologic Malignancy and Relapsed Acute Myeloid Leukemia, run by Foghorn Therapeutics Inc. at 5 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 22 inclusion and 28 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: under way, not looking for participants, as of the sponsor's last update on 10 Jul 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 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 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of AEs, dose-limiting toxicities (DLTs), serious AEs (SAEs), AEs leading to discontinuation, and adverse events of special interest (AESIs); safety laboratory assessments (time frame: Up to 18 months). 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 NCT04891757, which also lists 1 linked publication.
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 10 Jul 2025.