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

Phase 2

Terminated

• Conditions: Duchenne Muscular Dystrophy
• Interventions: Biological: ACE-031 (Extension of cohort 1 from core study, A031-03); Biological: ACE-031 (Extension of cohort 2 from core study, A031-03); Biological: ACE-031 (Extension of cohort 3 from core study, A031-03)

• Registration Number: NCT01239758
• Lead Sponsor: Acceleron Pharma, Inc., a wholly-owned subsidiary of Merck & Co., Inc., Rahway, NJ USA

Brief 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. \[Note: This study was terminated based on preliminary safety data. Pending further analysis of safety data and discussion with health authorities, a new ACE-031 trial will be planned.\]

Detailed Description

Not available

Study Timeline

• Study Start: 2010-10
• Study First Submitted: 2010-11-01
• Study First Submitted QC: 2010-11-10
• Study First Posted: 2010-11-11
• Primary Completion: 2011-05
• Study Completion: 2011-05
• Status Verified: 2013-01
• Disposition First Submitted: 2013-01-24
• Disposition First Submitted QC: 2013-01-30
• Last Update Submitted: 2013-01-30
• Disposition First Posted: 2013-02-01
• Last Update Posted: 2013-02-01

Recruitment & Eligibility

• Status: TERMINATED
• Sex: Male
• Target Recruitment: 11

Inclusion Criteria

• Completion of participation in Study A031-03 and Investigator approval
• Continuation of corticosteroid therapy at the same absolute dose and schedule as on Study A031-03

Exclusion Criteria

• Participation in any other therapeutic clinical trial
• Plans to have surgery during the course of the study

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
ACE-031 (Extension of cohort 1 from core study, A031-03)	ACE-031 (Extension of cohort 1 from core study, A031-03)	-
ACE-031 (Extension of cohort 2 from core study, A031-03)	ACE-031 (Extension of cohort 2 from core study, A031-03)	-
ACE-031 (Extension of cohort 3 from core study, A031-03)	ACE-031 (Extension of cohort 3 from core study, A031-03)	-

Primary Outcome Measures

Name	Time	Method
Number of patients with adverse events.	From treatment initiation to End-of-Study Visit, approximately 24 weeks later.
Change in laboratory parameters and vital signs.	Baseline to End-of-Study Visit, approximately 24 weeks later.

Secondary Outcome Measures

Name	Time	Method
Percent change in total lean body mass by DXA scan.	Baseline to End-of-Study Visit, approximately 24 weeks later.
Percent change in total body and lumbar spine bone mineral density by DXA scan.	Baseline to End-of-Study Visit, approximately 24 weeks later.
Percent change in muscle strength score by hand-held myometry.	Baseline to End-of-Study Visit, approximately 24 weeks later.
Change in distance traveled in 6 minutes (standardized 6-Minute-Walk Test).	Baseline to End-of-Study Visit, approximately 24 weeks later.
Change in time to travel 10 meters (standardized 10-Meter-Walk/Run test).	Baseline to End-of-Study Visit, approximately 24 weeks later.
Change in pulmonary function tests.	Baseline to End-of-Study Visit, approximately 24 weeks later.

Trial Locations

Locations (1)

Acceleron Investigative Site; 🇨🇦 Ottawa, Ontario, Canada