dm+d

Unassigned

New Medicines

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

Information

New molecular entity
Sarepta Therapeutics
Sarepta Therapeutics

Development and Regulatory status

None
None
Phase II Clinical Trials

Category

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
Intravenous infusion

Trial or other data

Nov 20PI/II trial (NCT03375164) has enrolled 4 patients. It expects to complete collection of primary outcome data now in Apr 23 [4].
Dec 18PI/II trial (NCT03375164) has completed recruitment. Collection of primary outcome data due to complete Jan 21 [3].
Dec 17PI/II trial (NCT03375164) starts. This is a single-dose controlled trial using rAAVrh74.MHCK7.micro-dystrophin for DMD subjects. Cohort A will include six subjects ages 3 months to 3 years, and Cohort B will include six subjects ages 4 to 7 years old. All subjects will receive intravenous micro-dystrophin vector (2X10e14 vg/kg in 10mL/kg). n the study, the gene will be infused via peripheral arm vein so that it can reach all the muscles in the body. Six DMD subjects ages 3 months to 3 years in Cohort A, and six DMD subjects ages 4 years to age 7 years in Cohort B, will be enrolled. All subjects will receive intravenous micro-dystrophin vector (2X10e14 vg/kg in 10mL/kg). Subjects will have infusions over 1 hour in the Pediatric Intensive Care Unit (PICU) at Nationwide Children Hospital in the US. Before the gene therapy a muscle biopsy will be done at the screening visit. Subjects will have a second muscle biopsy to see if the gene allowed for replacement of the missing dystrophin protein at 90 days post delivery. After the gene transfer, patients will be carefully monitored for any side effects of the treatment. This will include blood and urine tests, as well as physical examination during the screening visits and on days 0, 1, 7, 14, 30, 60, 90, and 180, and at months 9, 12, 18, 24, 30 and 36 to make sure that there are no side effects from the gene injection [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].

Evidence based evaluations