CompletedPhase 1/2

Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy

United States22 participantsStarted 2007-07

Plain-language summary

In this single-center trial, we will evaluate the effects of NaPB on presymptomatic Spinal Muscular Atrophy (SMA) type I (cohort 1)and presymptomatic SMA type II (cohort 2) infants. A variety of outcome measures will be performed at each study visit to follow the course of the disease. Total duration of the study for type I infants will be 18 months, for type II infants, 24 months.

Who can participate

Age range1 Day – 6 Months

SexALL

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Exclusion criteria

✕Evidence of hepatic insufficiency, renal insufficiency, edema with sodium retention, known seizure disorder, urea cycle disorder, cardiac arrhythmia, congenital heart defect, hypertension, significant central nervous system (CNS) impairment, or neurodegenerative or neuromuscular disease other than SMA.

What they're measuring

Safety and Tolerability of Sodium Phenylbutyrate in Neonates and Infants With SMA

Timeframe: 24 months

Trial details

NCT IDNCT00528268

SponsorUniversity of Utah

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2013-12

Results submitted2015-06-14

View on ClinicalTrials.gov More Spinal Muscular Atrophy trials