Phase Ib Study of AC-003 Capsules for Adults With Acute Graft-Versus-Host Disease After Stem Cell Transplant

What this Trial Is About

Researchers are evaluating AC-003 capsules in adults with grade II-IV steroid-refractory acute graft-versus-host disease SR-aGVHD following allogeneic hematopoietic stem cell transplantation. This Phase Ib, open-label, single-arm study aims to investigate the safety, pharmacokinetics, pharmacodynamics, and early signs of effectiveness of AC-003 in this patient group. The study is conducted at multiple centers and includes approximately 24 participants. The trial consists of two parts. Part A focuses on dose escalation through four sequential cohorts receiving increasing doses of AC-003 orally for 28 days, to determine safety and recommended dosing. Part B involves dose expansion to evaluate preliminary efficacy at the recommended dose, also given orally for 28 days. Safety follow-up occurs on Day 35 after dosing in both parts. Participants will receive specified doses of AC-003 capsules either once or twice daily for 28 days, depending on their cohort. Researchers will monitor safety by recording adverse events up to Day 35 and assess treatment response rates at Days 14 and 28. The study includes pharmacokinetic and pharmacodynamic evaluations, with ongoing safety follow-up and assessments during treatment to measure overall and complete response rates.

A Phase Ib Clinical Study of AC-003 Capsules in Subjects With aGVHD.