CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 22 enrolled
Drug / intervention
Sodium phenylbutyratedrug
Likely dose
Sodium phenylbutyrate 600 mg/kgfrom record
Structured eligibility isn't available for this trial yet — see the full criteria in the Eligibility tab below.

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT00528268
NCT00528268Phase 2Completed
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Prospective Phase I/II Study to Evaluate Effects of Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy

University of Utah·interventional·Posted Sep 12, 2007·Updated Feb 28, 2025

In Brief

A Phase 2 clinical trial evaluating Sodium phenylbutyrate for Spinal Muscular Atrophy. Completed, enrolled 22 participants across 1 site.

Detailed 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.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsSpinal Muscular Atrophy
CountriesUnited States
CollaboratorsEunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

Timeline

Phase 2CompletedFinished
200720082009201020112012201320142015201620172018201920202021202220232024202520262027
First PostedSep 12, 2007
Enrollment StartJul 1, 2007
Primary CompletionDec 1, 2013
TodayJul 2, 2026
Enrollment to primary: 6.4 yearsPosted 18.8 years ago

Interventions

Sodium phenylbutyratedrug

Sodium phenylbutyrate is dispensed as a powder, 450-600 mg/kg/day, divided into four doses. For cohort 1, treatment and monitoring continues for 18 months. For cohort 2, treatment and monitoring continues for 24 months.