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

Acceleron Pharma

Status and phase: Terminated; Phase 2

Conditions: Duchenne Muscular Dystrophy

Treatments: Biological: ACE-031 (Extension of cohort 3 from core study, A031-03); Biological: ACE-031 (Extension of cohort 2 from core study, A031-03); Biological: ACE-031 (Extension of cohort 1 from core study, A031-03)

Study type: Interventional

Funder types: Industry

Identifiers

NCT01239758

A031-06

Details and patient eligibility

About

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.]

Enrollment: 11 patients

Sex: Male

Ages: 4+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Parallel Assignment

Masking: None (Open label)

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11 participants in 3 patient groups

ACE-031 (Extension of cohort 1 from core study, A031-03)

Experimental group

Treatment:

Biological: ACE-031 (Extension of cohort 1 from core study, A031-03)

ACE-031 (Extension of cohort 2 from core study, A031-03)

Experimental group

Treatment:

Biological: ACE-031 (Extension of cohort 2 from core study, A031-03)

ACE-031 (Extension of cohort 3 from core study, A031-03)

Experimental group

Treatment:

Biological: ACE-031 (Extension of cohort 3 from core study, A031-03)