NCT07250646

A Study of Rebecsinib for Patients With Relapsed/Refractory Secondary Acute Myeloid Leukemia or High Risk Myelofibrosis

A Phase 1 Study of Rebecsinib, a Spliceosome Modulator That Inhibits ADAR1, in Patients With Relapsed or Refractory Secondary Acute Myeloid Leukemia or Higher-Risk Myelofibrosis

• Status: Not Yet Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 28 (estimated)
• Sponsor: Aspera Biomedicines, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to test the safest and most effective dose of a new investigational drug, rebecsinib. Participants in this study will have either Secondary Acute Myeloid Leukemia (sAML) that has either returned (relapsed) or not responded to treatment (refractory) or have higher risk Myelofibrosis (MF). Participants will receive a study drug infusion on Day 1, Day 4, Day 8 and Day 11 of each 28-day cycle for a total of 6 cycles.

Detailed description

The primary objective is to determine the maximum tolerated dose or biologically active dose of rebecsinib when given on days 1, 4, 8, and 11 of each 28 days cycle to patients with relapsed or refractory secondary acute myeloid leukemia (sAML), who have evolved from Myelodysplastic Syndrome (MDS) or myeloproliferative neoplasms (MPNs), or patients with higher-risk myelofibrosis. The other primary objective is to determine the safety and tolerability of rebecsinib by ongoing evaluation of adverse events (AEs), as assessed according to the NCI Common Terminology Criteria for Adverse Events (NCI CTCAE v5.0). Study outcomes will be measured by adverse event data, response rates, progression free survival and overall survival. Pharmacodynamics and plasma pharmacokinetics of rebecsinib will be explored.

Conditions

• Secondary AML
• Myelofibrosis

Interventions

Type	Name	Description
DRUG	rebecsinib	Rebecsinib is administered by intravenous infusion on Days 1, 4, 8, and 11 of the 28 day cycle. Doses to be tested are 0.2, 0.4, 0.6, 0.8, 1.0, 1.5, 2.0, 3.0, 4.0, 6.0, and 8.0 mg/kg based in ideal body weight (IBW).

Timeline

• Start date: 2026-04-01
• Primary completion: 2027-10-01
• Completion: 2028-04-01
• First posted: 2025-11-26
• Last updated: 2026-01-27

Locations

1 site across 1 country: United States

Regulatory

• FDA-regulated drug study