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Clinical Intramuscular Gene Transfer of rAAV1.CMV.huFollistatin344 Trial to Patients With Duchenne Muscular Dystrophy

NCT02354781 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2023-10-11

Study results available
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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.

Conditions

Interventions

BIOLOGICAL

rAAV1.CMV.huFollistin344

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

Sponsors & Collaborators

  • Duchenne Alliance

    collaborator OTHER

  • Milo Therapeutics

    collaborator INDUSTRY

  • Jerry R. Mendell

    lead OTHER

Principal Investigators

  • Jerry R Mendell, MD · Director, Center for Gene Therapy, Nationwide Children's Hospital

Study Design

Allocation
NA
Purpose
OTHER
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
7 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2015-01-31
Primary Completion
2017-11-30
Completion
2017-11-30

Countries

  • United States

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT02354781 on ClinicalTrials.gov