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

PHASE1/PHASE2CompletedINTERVENTIONAL
Enrollment

3

Participants

Timeline

Start Date

January 31, 2015

Primary Completion Date

November 30, 2017

Study Completion Date

November 30, 2017

Conditions
Duchenne Muscular Dystrophy
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.

Trial Locations (1)

43205

Nationwide Children's Hospital, Columbus

Sponsors
All Listed Sponsors
collaborator

Duchenne Alliance

OTHER

collaborator

Milo Therapeutics

INDUSTRY

lead

Jerry R. Mendell

OTHER