NCT01749540

Study to Evaluate the Safety and Efficacy of Luspatercept (ACE-536) in Participants With Beta-thalassemia (A536-04/MK-6143-002)

A Phase 2, Open-Label, Ascending Dose Study to Evaluate the Effects of ACE-536 in Patients With β-Thalassemia

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 64 (actual)
• Sponsor: Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the efficacy, safety, and pharmacokinetics, of ascending doses of luspatercept in participants with β-thalassemia. The primary objective of this study is to evaluate 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 participants, or 2. a ≥ 20% reduction in RBC transfusion burden compared to pretreatment in transfusion dependent participants.

Conditions

• B-Thalassemia

Interventions

Type	Name	Description
DRUG	luspatercept	subcutaneous injection

Timeline

• Start date: 2013-02-28
• Primary completion: 2015-11-11
• Completion: 2015-11-11
• First posted: 2012-12-13
• Last updated: 2024-07-18
• Results posted: 2024-07-18

Locations

8 sites across 2 countries: Greece, Italy