Safety, tolerability, and preliminary efficacy of an IGF-1 mimetic in patients with spinal and bulbar muscular atrophy: a randomised, placebo-controlled trial

What Was Tested

This randomized controlled trial paper looks at IGF-1 LR3.

Spinal and bulbar muscular atrophy is an X-linked neuromuscular disease caused by CAG repeat expansion in the androgen receptor gene.

Who Took Part

PubMed indexes the participants as adults, middle-aged adults and older adults, both men and women.

In this randomised, double-blind, placebo-controlled trial, the researchers recruited patients from neuromuscular centres in Denmark (Copenhagen), Germany (Ulm), Italy (Padova), and three sites within the USA (Bethesda, MD; Irvine, CA; and Columbus, OH).

Eligible patients were 18 years or older with a confirmed genetic diagnosis of spinal and bulbar muscular atrophy, were ambulatory, had symptomatic weakness, and had serum IGF-1 concentrations of 170 ng/mL or lower.

How the Study Was Run

In design terms this was a randomized, double-blind and placebo-controlled study.

The participants were allocated by chance, so the IGF-1 LR3 group and the comparison group should start out alike.

As a double-blind IGF-1 LR3 study, the assignments were hidden from both participants and investigators until the end.

A placebo (an inactive look-alike) gave the IGF-1 LR3 group a baseline to be compared against.

Patients were randomly assigned (2:1) to study drug or placebo by a number scheme.

Patients, investigators, and study personnel were masked to treatment assignment.

Primary outcome measures were safety, tolerability, and the effects of BVS857 on thigh muscle volume (TMV) measured by MRI.

The ratio of TMV at day 85 to baseline was analysed with ANCOVA per protocol.

This trial was registered with ClinicalTrials.gov, NCT02024932.

What Was Measured

Topics PubMed links to this paper include Biomimetics, Bulbo-Spinal Atrophy, X-Linked, Cohort Studies, Insulin-Like Growth Factor I, International Cooperation, Magnetic Resonance Imaging and Muscular Atrophy.

What This Means in Plain Language

The paper is a randomized, double-blind and placebo-controlled study of IGF-1 LR3.

Its findings take up roughly 10 sentences of the abstract and are left out here on purpose; the source paper is the place to read them.

The publication is from 2018 (The Lancet. Neurology), a journal based in England.

More context: the IGF-1 LR3 compound page, all human trials.