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

Study Overview
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Brief Summary
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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.]

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

Conditions
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Duchenne Muscular Dystrophy

Intervention / Treatment
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* 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)


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 4 Years

Sexes Eligible for Study
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Male

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ACE-031 (Extension of cohort 1 from core study, A031-03)<br> | Biological: ACE-031 (Extension of cohort 1 from core study, A031-03)<br>* ACE-031 0.5 mg/kg subcutaneously once every 4 weeks for 24 weeks.<br>|
| Experimental: ACE-031 (Extension of cohort 2 from core study, A031-03)<br> | Biological: ACE-031 (Extension of cohort 2 from core study, A031-03)<br>* Up to 1.0 mg/kg subcutaneously once every 2 weeks for 24 weeks.<br>|
| Experimental: ACE-031 (Extension of cohort 3 from core study, A031-03)<br> | Biological: ACE-031 (Extension of cohort 3 from core study, A031-03)<br>* Up to 2.5 mg/kg subcutaneously once every 4 weeks for 24 weeks.<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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. |