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

What Was Tested

The trial (phase 2) is built around ACE-031.

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.\].

It had three arms (groups): ACE-031 (Extension of cohort 1 from core study, A031-03) (experimental), ACE-031 (Extension of cohort 2 from core study, A031-03) (experimental) and ACE-031 (Extension of cohort 3 from core study, A031-03) (experimental).

The registry classes the interventions as biological.

Who Took Part

Enrollment: 11 actual participants took part.

It was open to men only aged from 4 years with no upper age limit.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Completion of participation in Study A031-03 and Investigator approval.

The full record lists 2 inclusion and 2 exclusion criteria; among the exclusions: participation in any other therapeutic clinical trial; plans to have surgery during the course of the study.

How the Study Was Run

Design: non-randomized, parallel-group and open-label.

Groups ran in parallel: each of the participants stayed in one arm throughout.

Everyone knew when ACE-031 was being given (open label), which is simpler to run but leaves more room for expectations to affect what is reported.

It started in 2010-10 with completion listed for 2011-05.

It ran at 4 sites in Canada (4).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Number of patients with adverse events (time frame: From treatment initiation to End-of-Study Visit, approximately 24 weeks later). Change in laboratory parameters and vital signs (time frame: Baseline to End-of-Study Visit, approximately 24 weeks later).

It also tracked 6 secondary outcomes, for example Percent change in total lean body mass by DXA scan, Percent change in total body and lumbar spine bone mineral density by DXA scan, Percent change in muscle strength score by hand-held myometry, Change in distance traveled in 6 minutes (standardized 6-Minute-Walk Test), Change in time to travel 10 meters (standardized 10-Meter-Walk/Run test) and Change in pulmonary function tests.

What This Means in Plain Language

Put simply, this is a phase 2, non-randomized trial of ACE-031 in 11 people.

Its size is small, so on its own it can only give an early look.

It ended early (terminated); no results are posted to the registry yet, so nothing here describes outcomes.

More context: the ACE-031 compound page, all human trials.