SMA CARNI-VAL trial part I: double-blind, randomized, placebo-controlled trial of L-carnitine and valproic acid in spinal muscular atrophy

What Was Tested

The paper reports a study of L-Carnitine, published as a randomized controlled trial.

Who Took Part

The age groups tagged for this paper are infants, young children and children, both men and women.

Two cohorts of subjects were enrolled in the SMA CARNIVAL TRIAL, a non-ambulatory group of "sitters" (cohort 1) and an ambulatory group of "walkers" (cohort 2).

Here, the researchers present results for cohort 1: a multicenter phase II randomized double-blind intention-to-treat protocol in non-ambulatory SMA subjects 2-8 years of age.

How the Study Was Run

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

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

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

Some participants received a placebo instead of L-Carnitine, giving a reference point.

More than one site took part, so the participants came from several places.

Sixty-one subjects were randomized 1:1 to placebo or treatment for the first six months; all received active treatment the subsequent six months.

The primary outcome was change in the modified Hammersmith Functional Motor Scale (MHFMS) score following six months of treatment.

Secondary outcomes included safety and adverse event data, and change in MHFMS score for twelve versus six months of active treatment, body composition, quantitative SMN mRNA levels, maximum ulnar CMAP amplitudes, myometry and PFT measures.

What Was Measured

PubMed files the paper under topics including Age Factors, Body Composition, Body Mass Index, Bone Density, Carnitine, Cohort Studies and Drug-Related Side Effects and Adverse Reactions.

What This Means in Plain Language

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

The abstract goes on to report its results in about 7 sentences; this page does not restate them, so read the paper for the findings and their limits.

The publication is from 2010 (PloS one), a journal based in United States.

More context: the L-Carnitine compound page, all human trials.