Study to Evaluate the Effects of ACE-536 in Patients With Beta-thalassemia

B-Thalassemia Clinical Trial

Official title: A Phase 2, Open-Label, Ascending Dose Study to Evaluate the Effects of ACE-536 in Patients With Beta-Thalassemia Intermedia

Status Completed

Clinical Trial Summary

The purpose of this study is to evaluate the effects of ACE-536 in patients with beta-thalassemia.

Clinical Trial Description

To evaluate the proportion of β-thalassemia patients who have an erythroid response, defined as:

1. a hemoglobin increase of ≥ 1.5 g/dL from baseline for ≥ 14 days (in the absence of red blood cell [RBC] transfusions) in non-transfusion dependent patients, or

2. ≥ 20% reduction in RBC transfusion burden compared to pretreatment in transfusion dependent patients. ;

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• B-Thalassemia
• beta-Thalassemia
• Thalassemia

• NCT number: NCT01749540
• Study type: Interventional
• Source: Acceleron Pharma, Inc.
• Status: Completed
• Phase: Phase 2
• Start date: February 2013
• Completion date: November 2015