A Randomized Study of ASTX727 With or Without Iadademstat in Advanced Myeloproliferative Neoplasms (MPNs)

Conditions

• Accelerated Phase Myeloproliferative Neoplasm
• Blast Phase Myeloproliferative Neoplasm
• Essential Thrombocythemia
• Myelodysplastic/Myeloproliferative Neoplasm
• Myeloproliferative Neoplasm, Not Otherwise Specified
• Polycythemia Vera
• Primary Myelofibrosis
• Secondary Myelofibrosis

Eligibility Criteria

Sex

ALL

Age

18 Years - N/A

Healthy Volunteers

Not accepted

Interventions

• Biospecimen Collection — PROCEDURE
  Undergo buccal swab and blood sample collection
• Bone Marrow Aspiration — PROCEDURE
  Undergo bone marrow aspiration and biopsy
• Bone Marrow Biopsy — PROCEDURE
  Undergo bone marrow aspiration and biopsy
• Decitabine and Cedazuridine — DRUG
  Given PO
• Iadademstat — DRUG
  Given PO

Study Details

This phase II trial compares the effect of ASTX727 in combination with iadademstat to ASTX727 alone in treating patients with accelerated or blast phase Philadelphia chromosome negative myeloproliferative neoplasms (MPNs). ASTX727 is a combination of two drugs, cedazuridine and decitabine. Cedazuridine is in a class of medications called cytidine deaminase inhibitors. It prevents the breakdown of decitabine, making it more available in the body so that decitabine will have a greater effect. Decitabine is in a class of medications called hypomethylation agents. It works by helping the bone marrow produce normal blood cells and by killing abnormal cells in the bone marrow. Iadademstat may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving ASTX727 in combination with iadademstat may be more effective than ASTX727 alone in treating patients with accelerated or blast phase Philadelphia chromosome negative MPNs.

Key Dates

First listed

Oct 28, 2024

Start date

Aug 14, 2025

Status verified

Aug 2026

Primary completion

Dec 31, 2027

Completion

Dec 31, 2027

Study Design

Enrollment

78 participants (estimated)

Allocation

RANDOMIZED

Intervention model

PARALLEL

Primary purpose

TREATMENT

Arms

• Experimental: Arm I (ASTX727)
  Patients receive ASTX727 PO QD on days 1-5 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Additionally, patients undergo buccal swab sample collection at baseline and blood sample collection and bone marrow aspiration and biopsy throughout the study.
• Experimental: Arm II (ASTX727, iadademstat)
  Patients receive ASTX727 PO QD on days 1-5 and iadademstat PO QD on days 1-5, 8-12, 15-19, and 22-26 of each cycle. Cycles repeat every 28 days in the absence of disease progression or unacceptable toxicity. Additionally, patients undergo buccal swab sample collection at baseline and blood sample collection and bone marrow aspiration and biopsy throughout the study.

Primary Outcome Measure

Acute leukemia response-complete (ALR-C) rate [ Time Frame: Within 4 cycles (cycle length = 28 days) ]

Locations (31)

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