NCT02604433

An Efficacy and Safety Study of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell Transfusions Due to Beta (β) Thalassemia

A Phase 3, Double-Blind, Placebo Controlled Multicenter Study to Determine the Efficacy and Safety of Luspatercept (ACE-536) in Adults With Transfusion Dependent Beta (B)-Thalassemia

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 336 (actual)
• Sponsor: Celgene · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This is a Phase 3, double-blind, randomized, placebo-controlled, multicenter study to determine the efficacy and safety of luspatercept (ACE-536) plus Best supportive care (BSC) versus placebo plus BSC in adults who require regular red blood cell transfusion due to (β)-thalassemia. The study is divided into the following periods: * Historical Period, * Screening/Run-in Period, * Double-blind Treatment Period (48 weeks), * Double-blind Long-term Treatment Period, (at the investigator's discretion an additional 48 weeks), * Open-Label Phase post unblinding and upon Data Monitoring Committee positive recommendation * Post-treatment Follow-up Period

Conditions

• Erythrocyte Transfusion
• Beta-Thalassemia

Interventions

Type	Name	Description
DRUG	Luspatercept	Subjects will start with luspatercept at 1 mg/kg dose level.
OTHER	Placebo	Placebo, Subcutaneous, every 21 days.

Timeline

• Start date: 2016-05-02
• Primary completion: 2021-01-05
• Completion: 2021-01-05
• First posted: 2015-11-13
• Last updated: 2023-04-18
• Results posted: 2020-01-27

Locations

76 sites across 15 countries: United States, Australia, Bulgaria, Canada, France, Greece, Israel, Italy, Lebanon, Malaysia, Taiwan, Thailand, Tunisia, Turkey (Türkiye), United Kingdom