Study of ACE-031 in Subjects With Duchenne Muscular Dystrophy

What Was Tested

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

It had three arms (groups): one (experimental), one (experimental) and Placebo (placebo comparator).

The registry classes the interventions as biological and other.

More on the Plan

ACE-031, a soluble form of the human activin receptor type IIB, was administered once every 2 to 4 weeks by subcutaneous (SC) injection to boys with DMD.

A total of 24 subjects were enrolled into the study; 18 received ACE-031 and 6 placebo.

Muscle strength was assessed by hand-held myometry and fixed system testing.

Body composition (i.e., spine BMD, lean mass, and fat mass) was assessed by whole body and lumbar spine DXA scans.

Who Took Part

Enrollment: 24 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: Diagnosis of DMD confirmed. Evidence of muscle weakness by clinical assessment.

The full record lists 4 inclusion and 3 exclusion criteria; among the exclusions: any previous treatment with another investigational product within 6 months prior to study day 1; inability to perform a whole body dual x-ray absorptiometry (dxa) scan.

How the Study Was Run

Design: randomized, parallel-group and quadruple-blind.

The participants were allocated by chance, so the ACE-031 group and the comparison group should start out alike.

The ACE-031 group and the comparison group ran side by side rather than switching.

Blinding on both sides kept the participants and researchers unaware of who received ACE-031.

Those kept unaware of assignments: participant, care provider, investigator and outcomes assessor.

It started in 2010-04 with completion listed for 2011-06.

It ran at 5 sites in Canada (5).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Number of Subjects With Adverse Reactions (time frame: From treatment initiation to End-of-Study Visit, approximately 24 weeks later). Number of Subjects With Clinical Laboratory Adverse Reactions (time frame: Baseline to End-of-Study Visit, approximately 24 weeks later).

It also tracked 8 secondary outcomes, for example Percent Change in Total Lean Body Mass by DXA Scan, Percent Change in 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 From Baseline in Time to Travel 10 Meters (Standardized 10-Meter-Walk/Run Test) and Change in Pulmonary Function Tests (FVC).

What This Means in Plain Language

Put simply, this is a phase 2, randomized trial of ACE-031 in 24 people.

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

It ended early (terminated); results are posted on the registry, and this page does not restate them.

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