CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 4 enrolled
Drug / intervention
delandistrogene moxeparvovecgenetic
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/NCT03375164
NCT03375164Phase 2Completed
CompareCT.gov

Systemic Gene Delivery Phase I/IIa Clinical Trial for Duchenne Muscular Dystrophy Using rAAVrh74.MHCK7.Micro-dystrophin (microDys-IV-001)

Sarepta Therapeutics, Inc.·interventional·Posted Dec 15, 2017·Updated Nov 14, 2024

In Brief

A Phase 2 clinical trial evaluating delandistrogene moxeparvovec for Duchenne Muscular Dystrophy. Completed, enrolled 4 participants across 1 site.

Detailed Summary

This study was an open-label single-dose gene transfer therapy study evaluating the safety of delandistrogene moxeparvovec intravenous (IV) administration in boys with DMD. This study was originally designed to consist of 12 patients across 2 Cohorts. Cohort A would have included participants ages 3 months to 3 years, and Cohort B included participants ages 4 to 7 years old. No participants were enrolled in Cohort A.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsDuchenne Muscular Dystrophy
CountriesUnited States
Collaborators--

Timeline

Phase 2CompletedFinished
201820192020202120222023202420252026
First PostedDec 15, 2017
Enrollment StartJan 4, 2018
Primary CompletionApr 25, 2023
TodayJul 2, 2026
Enrollment to primary: 5.3 yearsPosted 8.5 years ago

Interventions

delandistrogene moxeparvovecgenetic

Single IV infusion of delandistrogene moxeparvovec.