NCT06297941

Study of REM-422 in Patients With AML or Higher Risk MDS

A Phase 1, Multicenter, Open-Label Study of REM-422, an MYB mRNA Degrader, in Patients With Relapsed/Refractory AML or Higher-Risk MDS

• Status: Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 100 (estimated)
• Sponsor: Remix Therapeutics · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The goal of this study is to determine the safety and antitumor effects of REM-422, a MYB mRNA degrader, in people with Higher Risk MDS and relapsed/refractory AML

Detailed description

This is a Phase 1, open-label, non-randomized, multicenter study investigating REM-422, a potent, selective, and oral small molecule mRNA degrader that reduces expression of the MYB transcription factor for patients with higher risk MDS or relapsed/refractory AML. This study includes a Dose Escalation Phase and a Dose Expansion Phase. The purpose of the Dose Escalation Phase is to determine the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D) of REM-422 in patients with higher risk MDS or relapsed/refractory AML. The purpose of Dose Expansion is to further evaluate the safety and anti-tumor activity of the RP2D carried forward from Dose Escalation. Participation in this study will continue until disease progression, therapy intolerance, or participant withdrawal.

Conditions

• Myelodysplastic Syndromes
• Higher Risk Myelodysplastic Syndromes
• Acute Myeloid Leukemia
• Acute Myeloid Leukemia Refractory

Interventions

Type	Name	Description
DRUG	REM-422	REM-422 is a first in class, small molecule mRNA inhibitor that reduces expression of the MYB transcription factor REM-422 will be administered orally once daily

Timeline

• Start date: 2024-04-26
• Primary completion: 2026-03-15
• Completion: 2027-06-15
• First posted: 2024-03-07
• Last updated: 2025-04-23

Locations

9 sites across 2 countries: United States, France

Regulatory

• FDA-regulated drug study