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

Duchenne Muscular Dystrophy Clinical Trial

Official title:

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01239758
• Other study ID #: A031-06
• Status: Terminated
• Phase: Phase 2
• First received: November 1, 2010
• Last updated: January 30, 2013
• Start date: October 2010
• Est. completion date: May 2011

Study information

• Verified date: January 2013
• Source: Acceleron Pharma, Inc.
• Contact: n/a
• Is FDA regulated: No
• Health authority: Canada: Health CanadaUnited States: Food and Drug Administration
• Study type: Interventional

Clinical Trial 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.]

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 11
• Est. completion date: May 2011
• Est. primary completion date: May 2011
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 4 Years and older

Eligibility

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

Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Duchenne Muscular Dystrophy
• Muscular Dystrophies
• Muscular Dystrophy, Duchenne

Intervention

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.
• 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.
• 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.

Locations

Country	Name	City	State
Canada	Acceleron Investigative Site	Calgary	Alberta
Canada	Acceleron Investigative Site	Hamilton	Ontario
Canada	Acceleron Investigative Site	London	Ontario
Canada	Acceleron Investigative Site	Ottawa	Ontario

Sponsors (1)

Lead Sponsor	Collaborator
Acceleron Pharma, Inc.

Country where clinical trial is conducted

Canada, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of patients with adverse events.		From treatment initiation to End-of-Study Visit, approximately 24 weeks later.	Yes
Primary	Change in laboratory parameters and vital signs.		Baseline to End-of-Study Visit, approximately 24 weeks later.	Yes
Secondary	Percent change in total lean body mass by DXA scan.		Baseline to End-of-Study Visit, approximately 24 weeks later.	No
Secondary	Percent change in total body and lumbar spine bone mineral density by DXA scan.		Baseline to End-of-Study Visit, approximately 24 weeks later.	No
Secondary	Percent change in muscle strength score by hand-held myometry.		Baseline to End-of-Study Visit, approximately 24 weeks later.	No
Secondary	Change in distance traveled in 6 minutes (standardized 6-Minute-Walk Test).		Baseline to End-of-Study Visit, approximately 24 weeks later.	No
Secondary	Change in time to travel 10 meters (standardized 10-Meter-Walk/Run test).		Baseline to End-of-Study Visit, approximately 24 weeks later.	No
Secondary	Change in pulmonary function tests.		Baseline to End-of-Study Visit, approximately 24 weeks later.	No