New Medicines

Duchenne muscular dystrophy in children aged 3 months to 7 years with mutations between exons 18 and 58


New molecular entity
Sarepta Therapeutics
Sarepta Therapeutics

Development and Regulatory status

Phase II Clinical Trials


Mutation agnostic gene therapy. Recombinant adeno‐associated virus (rAAV) is the preferred vehicle for delivery due to the persistence of injected AAV in striated muscles and lack of pathogenicity.
Duchenne Muscular Dystrophy (DMD) is one of the most common and severe forms of muscular dystrophy, affecting 15.9 to 19.5 per 100,000 live births. It almost exclusively occurs in males. People with the condition will usually only live into their 20s or 30s [1]
Duchenne muscular dystrophy in children aged 3 months to 7 years with mutations between exons 18 and 58

Trial or other data

Nov 21PI/II trial (NCT03333590) is due to complete [3].
Feb 20PI/II trial (NCT03333590) is active but no longer recruiting [3].
Nov 17PI/II trial of rAAVrh74.MCK.GALGT2 for DMD patients starts (NCT03333590). This is an open-label, dose escalation trial where the vector will be delivered via the femoral artery to the muscles of both legs of DMD subjects. The primary objective of this study is the assessment of the safety of intravascular administration of rAAVrh74.MCK.GALGT2 to DMD patients. Safety endpoints will be assessed by changes in hematology, serum chemistry, urinalysis, immunologic response to rAAVrh74 and GALGT2, and reported history and observations of symptoms. Efficacy measures will be used as secondary outcome for this disorder including a combination of functional 6 minute walk test (6MWT) and direct muscle testing for strength (MVICT) of lower limb muscles. Subjects will be evaluated at baseline, infusion visit (days 0-2), and return for follow up visits on days 7, 14, 30, 60, 90, and 180 and months 12, 18 and 24. Six patients aged 4 years and older will be recruited at the Nationwide Childrens Hospital in Ohio. Collection of primary outcome data is due to complete Nov 20 [3].
Jan 17Nationwide Children’s Hospital, in collaboration with Sarepta Therapeutics, is planning a phase I/II trial to assess the safety of microdystrophin gene therapy in patients with Duchenne muscular dystrophy. The trial is scheduled to initiate in late 2017 [2].