NCT05732103

A Study of CTX-712 in Relapsed/Refractory Acute Myeloid Leukemia and Higher Risk Myelodysplastic Syndromes

Phase 1/2 Multicenter, Open-Label Study of CTX-712 in Patients With Relapsed/Refractory Acute Myeloid Leukemia and Higher Risk Myelodysplastic Syndromes

• Status: Recruiting
• Phase: Phase 1 / Phase 2
• Study type: Interventional
• Enrollment: 225 (estimated)
• Sponsor: Chordia Therapeutics, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The goal of this phase 1/2 multicenter, open-label, single-arm dose escalation and expansion study is to assess the safety, tolerability, pharmacokinetic and pharmacodynamic profile of CTX-712 in patients with relapsed/refractory (R/R) acute myeloid leukemia (AML) and higher risk myelodysplastic syndromes (HR-MDS), or MDS/MPN (including CMML). The phase 1 part of the study consists of sequential standard 3 + 3 dose escalation, where patients will receive ascending doses of CTX-712 to determine the recommended phase 2 dose (RP2D) for further clinical development. This is followed by initial expansion cohorts in AML and/or HR-MDS where patients will be treated with CTX-712 at the RP2D to gain further confidence in the selected dose level. Additional expansion cohorts may be initiated if considered necessary. After RP2D is determined, Drug-Drug-Interaction cohorts will be started. The phase 2 part of the study will commence after the RP2D has been identified and confirmed and will evaluate therapeutic activity in R/R AML or R/R HR-MDS, in addition to confirmation of the safety profile.

Conditions

• Acute Myeloid Leukemia
• Myelodysplastic Syndromes

Interventions

Type	Name	Description
DRUG	CTX-712	CTX-712 will be provided as a 20 mg tablet for oral administration. Patients will take CTX-712 once or twice weekly, depending on their dose level assignment, during each 28-day cycle.

Timeline

• Start date: 2023-04-25
• Primary completion: 2028-06-01
• Completion: 2029-02-01
• First posted: 2023-02-16
• Last updated: 2026-04-13

Locations

8 sites across 1 country: United States

Regulatory

• FDA-regulated drug study