CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 6 enrolled
Drug / intervention
SRP-9004genetic
Likely dose
Not stated in 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/NCT01976091
NCT01976091Phase 2Completed
CompareCT.gov

Phase I/IIA Gene Transfer Clinical Trial for LGMD2D (Alpha-Sarcoglycan Deficiency) Using scAAVrh74.tMCK.hSGCA

Sarepta Therapeutics, Inc.·interventional·Posted Nov 5, 2013·Updated Jun 15, 2023

In Brief

A Phase 2 clinical trial evaluating SRP-9004 for Limb-Girdle Muscular Dystrophy, Type 2D. Completed, enrolled 6 participants.

Detailed Summary

This is an open-label, dose escalation gene transfer therapy study evaluating the safety of SRP-9004 (patidistrogene bexoparvovec) via isolated limb infusion (ILI) administration in approximately 6 participants with LGMD2D.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsLimb-Girdle Muscular Dystrophy, Type 2D
Countries--
CollaboratorsNationwide Children's Hospital

Timeline

Phase 2CompletedFinished
2014201520162017201820192020202120222023202420252026
First PostedNov 5, 2013
Enrollment StartFeb 1, 2015
Primary CompletionMar 14, 2019
TodayJul 2, 2026
Enrollment to primary: 4.1 yearsPosted 12.7 years ago

Interventions

SRP-9004genetic

Isolated Limb Infusion (ILI)