NCT01239758

Extension Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy

An Open-Label Extension Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of ACE-031 (ActRIIB-IgG1) in Subjects With Duchenne Muscular Dystrophy

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 11 (actual)
• Sponsor: Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA · Industry
• Sex: Male
• Age: 4 Years
• Healthy volunteers: Not accepted

Summary

To evaluate the long-term safety and tolerability of ACE-031 administration in subjects with Duchenne muscular dystrophy (DMD) who participated in Study A031-03. \[Note: This study was terminated based on preliminary safety data. Pending further analysis of safety data and discussion with health authorities, a new ACE-031 trial will be planned.\]

Conditions

• Duchenne Muscular Dystrophy

Interventions

Type	Name	Description
BIOLOGICAL	ACE-031 (Extension of cohort 1 from core study, A031-03)	ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 24 weeks.
BIOLOGICAL	ACE-031 (Extension of cohort 2 from core study, A031-03)	Up to 1.0 mg/kg subcutaneously once every 2 weeks for 24 weeks.
BIOLOGICAL	ACE-031 (Extension of cohort 3 from core study, A031-03)	Up to 2.5 mg/kg subcutaneously once every 4 weeks for 24 weeks.

Timeline

• Start date: 2010-10-01
• Primary completion: 2011-05-01
• Completion: 2011-05-01
• First posted: 2010-11-11
• Last updated: 2013-02-01

Locations

4 sites across 1 country: Canada