Phase 2 trial of leuprorelin in patients with spinal and bulbar muscular atrophy

What Was Tested

Researchers studied Leuprolide in this randomized controlled trial publication.

Spinal and bulbar muscular atrophy (SBMA) is a hereditary motor neuron disease caused by the expansion of a polyglutamine tract in the androgen receptor (AR).

Who Took Part

The abstract describes 19 patients taking part.

The age groups tagged for this paper are adults, middle-aged adults, older adults and people over 80, men only.

The patients who did not participate in the open-label trial were also followed up for the 96-week period (UMIN000000474).

How the Study Was Run

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

Random assignment meant chance, not the researchers, decided who got Leuprolide and who got the comparison, which limits bias.

Blinding on both sides kept the patients and researchers unaware of who received Leuprolide.

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

Some patients received a placebo instead of Leuprolide, giving a reference point.

What Was Measured

Topics PubMed links to this paper include Androgen Antagonists, Cineradiography, Japan, Leuprolide, Microscopy, Video, Muscular Atrophy, Spinal and Mutation.

What This Means in Plain Language

The paper is a randomized, double-blind and open-label study of Leuprolide in 19 patients.

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 5 sentences of the abstract; we leave them to the paper itself rather than summarise them.

The publication is from 2009 (Annals of neurology), a journal based in United States.

More context: the Leuprolide compound page, all human trials.