← Human Trials & Peer-Reviewed Studies
Myostatin inhibitor ACE-031 treatment of ambulatory boys with Duchenne muscular dystrophy: Results of a randomized, placebo-controlled clinical trial
Plain-language guide to PMID 27462804 (2017): double-blind randomized controlled trial study of ACE-031 in 4 boys.
Key Facts
- PMID
- 27462804
- Journal
- Muscle & nerve
- Year
- 2017
- Study type
- Randomized Controlled Trial
- Authors
- Campbell C et al. (10 authors)
- DOI
- 10.1002/mus.25268
What This Study Did, in Plain Language
What Was Tested
Researchers studied ACE-031 in this randomized controlled trial publication.
The primary objective was safety evaluation.
Why It Was Studied
ACE-031 is a fusion protein of activin receptor type IIB and IgG1-Fc, which binds myostatin and related ligands.
It aims to disrupt the inhibitory effect on muscle development and provide potential therapy for myopathies like Duchenne muscular dystrophy (DMD).
Who Took Part
According to the abstract, 4 boys were involved.
By PubMed's age tags, the people studied were young children, children and teenagers, men only.
How the Study Was Run
The design is described as randomized, double-blind and placebo-controlled.
Because people in this ACE-031 study were split into groups at random, differences between the groups are less likely to reflect who happened to join each one.
As a double-blind ACE-031 study, the assignments were hidden from both participants and investigators until the end.
Some boys received a placebo instead of ACE-031, giving a reference point.
What Was Measured
The subject headings attached to it are Activin Receptors, Type II, Body Composition, Cohort Studies, Muscular Dystrophy, Duchenne, Myostatin and Walking.
Author keywords: 6-minute walk test, Duchenne muscular dystrophy, TGF-β superfamily, lean body mass, myostatin inhibitor and placebo-controlled clinical trial.
What This Means in Plain Language
The paper is a randomized, double-blind and placebo-controlled study of ACE-031 in 4 boys.
With fewer than 40 people, it is a small study, so it is better read as an early signal for further research than as a settled answer.
Results are reported in around 7 sentences of the abstract; we leave them to the paper itself rather than summarise them.
The publication is from 2017 (Muscle & nerve), a journal based in United States.
More context: the ACE-031 compound page, all human trials.
Our Notes on the Compound
ACE-031: A soluble activin type IIB receptor fusion protein that soaks up myostatin; a Duchenne trial was stopped for bleeding-related side effects.
Related Pages
Source: PubMed record. This explanation is written by PeptideWhiz from the public record. For research and education only. Not medical advice; no dosing information is given.