CARNIVAL Type I: Valproic Acid and Carnitine in Infants With Spinal Muscular Atrophy (SMA) Type I

What Was Tested

In this phase 1, phase 2 trial, researchers set out to test L-Carnitine.

This is a multi-center trial to test safety and evaluate early treatment intervention with valproic acid and carnitine in moderating SMA symptoms of Type I infants.

It had 1 arm (groups): 1 (experimental).

All patients will receive VPA and carnitine.

The registry classes the interventions as drug.

More on the Plan

It is one of the most common conditions causing muscle weakness in children.

Patients with SMA most often develop weakness as babies or young children.

Most people with SMA gradually lose muscle strength and abilities over time.

Babies with the severe infantile form of SMA, SMA type I, usually lose abilities and strength quickly over a few weeks or months.

Who Took Part

Enrollment: 40 actual participants took part.

It was open to people of any sex aged from 2 Weeks up to 12 months.

Healthy volunteers were not accepted; participants needed the condition being studied.

Examples of who could join: Laboratory documentation of SMN mutation/deletion consistent with a genetic diagnosis of SMA. Clinical diagnosis of SMA type I. Age 2 weeks to 12 months. Written informed consent of parents/guardian.

The full record lists 4 inclusion and 7 exclusion criteria; among the exclusions: any clinical or laboratory evidence of hepatic or pancreatic insufficiency; serious illness requiring systemic treatment and/or hospitalization within two weeks prior to study entry; unwillingness to travel for study assessments.

How the Study Was Run

It was run as a single-group and open-label study.

Being open-label, the L-Carnitine study did not mask who received what.

Everyone received the same intervention, so there was no separate comparison group inside the trial.

It started in 2008-04 with completion listed for 2012-05.

It ran at 8 sites in United States (6), Canada (1) and Germany (1).

Its stated primary purpose is treatment.

What Was Measured

The main thing measured (primary outcome): Laboratory Safety Data (time frame: -2 weeks, + 2 weeks, 3 months, 6 months). Anthropometric Measures of Nutritional Status (Body Mass Index [BMI] Z-scores, Weight for Length Ratios, Lean/Fat Mass Via DEXA, Growth Parameters, and Triceps Skinfold Measures) (time frame: -2 weeks, time 0, 3 months, 6 months).

It also tracked 6 secondary outcomes, for example Primary Caregiver Functional Rating Scale for SMA Type I Subjects (PCFRS), Functional Motor Assessments: TIMPSI Scores, Quantitative SMN mRNA and Protein Measures, Maximum Ulnar CMAP Amplitude/Area and MUNE and Whole Body DEXA Scanning for Lean Body Mass and Total Bone Mineral Density/ Content.

What This Means in Plain Language

Put simply, this is a phase 1, phase 2 trial of L-Carnitine in 40 people.

The trial is marked completed; results are posted on the registry, and this page does not restate them.

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