CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 3 enrolled
Drug / intervention
rAAV1.CMV.huFollistin344biological
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/NCT02354781
NCT02354781Phase 2Completed
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Phase I/II Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy

Jerry R. Mendell·interventional·Posted Feb 3, 2015·Updated Oct 11, 2023

In Brief

A Phase 2 clinical trial evaluating rAAV1.CMV.huFollistin344 for Duchenne Muscular Dystrophy. Completed, enrolled 3 participants across 1 site.

Detailed Summary

The proposed clinical trial is an outgrowth of the safety record and functional improvement seen in the BMD follistatin gene therapy trial. In this study the investigators propose to inject AAV1.CMV.huFS344 at a total dose of 2.4E12 vg/kg to six DMD patients. This dose will be divided between gluteal muscles, quadriceps and tibialis anterior. This is a wider distribution of vector than given to BMD patients, who overall improved the distance walked on the 6MWT without adverse events related to viral transduction into a single muscle.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsDuchenne Muscular Dystrophy
CountriesUnited States
CollaboratorsDuchenne Alliance, Milo Therapeutics

Timeline

Phase 2CompletedFinished
201520162017201820192020202120222023202420252026
First PostedFeb 3, 2015
Enrollment StartJan 1, 2015
Primary CompletionNov 1, 2017
TodayJul 2, 2026
Enrollment to primary: 2.8 yearsPosted 11.4 years ago

Interventions

rAAV1.CMV.huFollistin344biological

Six DMD patients will receive rAAV1.CMV.huFollistatin344 to both limbs by multiple injections to gluteal muscles, quadriceps and tibialis anterior muscles.