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
Aspera Biomedicines, Inc.
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.
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…
Eligibility
- Age range
- 18+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. Ability to understand and the willingness to sign a written informed consent. 2. Must have a refractory myeloid neoplasm (5th Edition WHO Classification of Tumors) consisting of secondary AML (sAML), which has evolved from antecedent MDS, MPN, MDS/MPN, or have higher-risk MF defined by DIPSS Plus or MIPSS70+ version 2.0, and excess bone marrow blasts (\> 5%). Patients with sAML, defined as AML with an antecedent history of MDS or MPN or WHO AML with myelodysplasia related genetic changes or WHO AML with myelodysplasia related cytogenetic abnormalities. Must be ineligibl…
Interventions
- Drugrebecsinib
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).
Location
- UC San Diego Moores Cancer CenterSan Diego, California