Efficacy and safety of leuprorelin in patients with spinal and bulbar muscular atrophy (JASMITT study): a multicentre, randomised, double-blind, placebo-controlled trial

What Was Tested

Researchers studied Leuprolide in this randomized controlled trial publication.

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

At present there are no treatments for spinal and bulbar muscular atrophy, although leuprorelin suppressed the accumulation of pathogenic androgen receptors in a phase 2 trial.

Who Took Part

By PubMed's age tags, the people studied were adults, middle-aged adults and older adults, both men and women.

Patients and investigators were masked to treatment allocation.

All patients who were randomly assigned and who were assessed with videofluorography at least once were included in the analyses.

How the Study Was Run

The design is described as randomized, double-blind and placebo-controlled.

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

Neither the participants nor the staff measuring them knew who was on Leuprolide, so expectations could not easily colour the readings.

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

The Japan SBMA Interventional Trial for TAP-144-SR (JASMITT) was a 48-week, randomised, double-blind, placebo-controlled trial done at 14 hospitals between August, 2006, and March, 2008.

The primary endpoint was pharyngeal barium residue, which indicates incomplete bolus clearance, measured at week 48 by videofluorography.

This study is registered with the JMACCT clinical trials registry, number JMA-IIA00009, and the UMIN clinical trials registry, number UMIN000000465.

What Was Measured

Topics PubMed links to this paper include Antineoplastic Agents, Hormonal, Japan, Leuprolide, Muscular Atrophy, Muscular Atrophy, Spinal and Statistics, Nonparametric.

What This Means in Plain Language

The paper is a randomized, double-blind and placebo-controlled study of Leuprolide.

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 2010 (The Lancet. Neurology), a journal based in England.

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