NCT06846606

Phase 1 Study of AUTX-703 in Relapsed/Refractory AML and MDS

A Phase 1 Study of AUTX-703 in Participants With Relapsed/Refractory Acute Myeloid Leukemia and Myelodysplastic Syndromes

• Status: Active Not Recruiting
• Phase: Phase 1
• Study type: Interventional
• Enrollment: 34 (actual)
• Sponsor: Auron Therapeutics, Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This Phase 1, multicenter, open-label, dose escalation and dose optimization study is designed to assess the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of AUTX-703 administered orally in subjects with advanced hematologic malignancies.

Detailed description

This is a first-in-human, Phase 1, multicenter study to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and preliminary clinical activity of AUTX-703, an orally bioavailable lysine acetyltransferase 2A (KAT2A) and lysine acetyltransferase 2B (KAT2B) degrader, in participants with relapsed/refractory acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). The study consists of two parts: Part A (Dose Escalation) to determine the maximum tolerated dose (MTD) and/or recommended Phase 2 dose (RP2D), and Part B (Dose Optimization) to further evaluate safety, PK, PD and efficacy at selected dosages.

Conditions

• Relapsed Acute Myeloid Leukemia (AML)
• Refractory Acute Myeloid Leukemia (AML)
• Relapsed/Refractory AML
• Relapsed Myelodysplastic Syndromes
• Refractory Myelodysplastic Syndromes
• Relapsed/Refractory (R/R) Acute Myeloid Leukemia (AML)

Interventions

Type	Name	Description
DRUG	AUTX-703	AUTX-703 administered orally

Timeline

• Start date: 2025-05-01
• Primary completion: 2027-04-01
• Completion: 2028-06-01
• First posted: 2025-02-26
• Last updated: 2026-03-03

Locations

9 sites across 1 country: United States

Regulatory

• FDA-regulated drug study