Actively Recruiting

Phase 1
Age: 18Years +
All Genders
NCT06327685

Avapritinib With Decitabine in Patients With SM-AHN

Led by H. Lee Moffitt Cancer Center and Research Institute · Updated on 2026-04-01

34

Participants Needed

7

Research Sites

154 weeks

Total Duration

On this page

Sponsors

H

H. Lee Moffitt Cancer Center and Research Institute

Lead Sponsor

B

Blueprint Medicines Corporation

Collaborating Sponsor

AI-Summary

What this Trial Is About

Systemic mastocytosis with an associated hematologic neoplasm (SM-AHN) is a challenging disease to treat. Targeted KIT inhibitors have been approved for this indication based on their ability to control the mastocytosis portion of the disease, but patients frequently experience progression of the concomitant myeloid malignancy (i.e. the AHN). Using a combination approach to treat both aspects of the disease has the potential to provide enhanced disease control; however, overlapping toxicity is a concern. In this study, investigators aim to study the safety and tolerability of combined avapritinib and decitabine for the treatment of SM-AHN.

CONDITIONS

Official Title

Avapritinib With Decitabine in Patients With SM-AHN

Who Can Participate

Age: 18Years +
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • Diagnosis of SM-AHN defined by World Health Organization 2022 criteria.
  • ECOG performance status 0 to 3.
  • Ability to understand and willingness to sign informed consent.
  • Ability to follow study visits and protocol requirements.
  • Willingness to receive blood products if needed.
  • Adequate organ and bone marrow function as defined by the protocol.
  • HIV-infected participants on effective anti-retroviral therapy with undetectable viral load within 6 months.
  • For chronic hepatitis B infection, undetectable viral load on suppressive therapy if needed.
  • Participants with hepatitis C must be treated and cured or have undetectable viral load if currently treated.
  • Participants with cardiac disease history should be New York Heart Association Functional Classification class 2B or better.
  • Women of child-bearing potential and men must use adequate contraception before, during, and after study participation as specified.
Not Eligible

You will not qualify if you...

  • History of decitabine use with disease progression of AHN while on decitabine.
  • History of avapritinib use with progression of mastocytosis while on avapritinib.
  • Prior treatment with decitabine combined with avapritinib.
  • Use of azacitidine within 4 weeks before first study drug dose.
  • Diagnosis of acute myeloid leukemia with 20% or more myeloblasts or myeloid sarcoma.
  • Receiving other investigational agents or participating in another interventional study.
  • Allergic reactions to similar compounds including azacitidine, decitabine, cedazuridine, avapritinib, propylene glycol, or mannitol.
  • History of intracranial hemorrhage or conditions increasing risk of brain bleeding.
  • History of seizure disorder or need for antiseizure medication.
  • QTcF interval greater than 480 msec.
  • Recent allogeneic stem cell transplant within 6 months, active graft versus host disease, or transplant immunosuppression.
  • Use of strong or moderate CYP3A inhibitors or inducers.
  • Uncontrolled intercurrent illness.
  • Psychiatric or social conditions limiting compliance.
  • Pregnant or breastfeeding women.
  • Inability or unwillingness to comply with study visits, drug plan, tests, or restrictions.
  • Primary brain malignancy or brain metastases.
  • Major surgery within 14 days before first study drug dose (minor procedures allowed).
  • Eosinophilia with FIP1L1-PDGFRA fusion unless relapse or progression on imatinib.
  • Participation in another interventional clinical study.

AI-Screening

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Trial Site Locations

Total: 7 locations

1

Mayo Clinic - Arizona

Phoenix, Arizona, United States, 85054

Not Yet Recruiting

2

Stanford University Medical Center

Palo Alto, California, United States, 94305

Not Yet Recruiting

3

Moffitt Cancer Center

Tampa, Florida, United States, 33612

Actively Recruiting

4

Dana-Farber Cancer Institute

Boston, Massachusetts, United States, 02215

Not Yet Recruiting

5

University of Michigan

Ann Arbor, Michigan, United States, 48109

Not Yet Recruiting

6

Memorial Sloan Kettering Cancer Center

New York, New York, United States, 10065

Not Yet Recruiting

7

University of Utah Health

Salt Lake City, Utah, United States, 84132

Actively Recruiting

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Research Team

P

Paul Ciero

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

SEQUENTIAL

Primary Purpose

TREATMENT

Number of Arms

2

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