Phase 2 Study of AND017 Capsules to Treat Adults With Transfusion-Dependent and Non-Transfusion-Dependent -Thalassemia

What this Trial Is About

Researchers are evaluating the safety and effectiveness of a drug called AND017 in adults aged 18 to 65 who have transfusion-dependent or non-transfusion-dependent beta-thalassemia. This phase II study compares different oral doses of AND017 with a placebo, alongside optimal supportive care such as blood transfusions and iron removal based on the clinicians judgment. The goal is to understand how well AND017 works and how safe it is for these patients. Participants will be randomly assigned to receive one of several doses of AND017 capsules 8 mg, 12 mg, or 16 mg or matching placebo capsules, taken once daily. The study treatment will continue for up to 24 weeks. Throughout the treatment period, patients will continue to receive their usual supportive care including blood transfusions as needed. During the study, participants will undergo regular assessments including blood tests to measure hemoglobin levels, red blood cell counts, iron-related markers, and changes in transfusion needs. These evaluations will occur at multiple visits from baseline through week 24. Researchers will closely monitor safety and tolerability and track changes from baseline in various blood parameters to assess the impact of AND017. The total participation time includes treatment and follow-up visits lasting up to 24 weeks or until early discontinuation.

A Study of Safety and Efficiency of AND017 in Patients With β-thalassemia