Boys between the ages of seven and 16 with Duchenne muscular dystrophy are being recruited to participate in a phase 3 clinical trial to evaluate the effectiveness and safety of eteplirsen in treating DMD.
Update (Oct. 27, 2014): In an Oct. 27, 2014, press release and conference call, Sarepta Therapeutics announced that it has revised its timeline for submission of a new drug application to the U.S. Food and Drug Administration for eteplirsen to mid-2015, based on guidance from the agency. The company had hoped to submit the eteplirsen application by the end of 2014. The conference call will be available as a webcast on the Sarepta site for 90 days.
Boys with Duchenne muscular dystrophy between the ages of seven and 16 are now being recruited to participate in a phase 3 clinical trial to evaluate the effectiveness and safety of eteplirsen in treating DMD.
Eteplirsen is an experimental drug in development by Cambridge, Mass.-based Sarepta Therapeutics to treat about 13 percent of people with DMD — those who have specific mutations (flaws) in the gene for the muscle protein dystrophin. The goal of treatment with eteplirsen — known as an exon-skipping drug — is to coax muscle cells to reinterpret genetic information for the dystrophin protein so that functional dystrophin protein can be made even though the dystrophin gene is flawed.
MDA has funded the development of exon skipping as a treatment for DMD since the 1990s and has helped support the clinical testing of eteplirsen.
Sarepta currently has several sites open for enrollment and is working to have 38 sites participate in the trial (known as the PROMOVI study). They expect to continue initiating study sites on a rolling basis between now and early 2015. In August, Sarepta announced that it was on track to submit a new drug application for eteplirsen to treat a subset of patients with DMD to the U.S. Food and Drug Administration by the end of 2014. (Editor's note: On Oct. 27, 2014, Sarepta announced that it estimates it will submit a new drug application to the U.S. Food and Drug Administration in mid-2015.)
Full details about the trial and eligibility requirements can be found at clinicaltrials.gov. Enter NCT02255552 into the search box.
About the PROMOVI Study
This is an open-label, multi-center, 48-week study to evaluate the efficacy and safety of eteplirsen in patients with genotypically confirmed Duchenne muscular dystrophy with genetic deletions amenable to exon 51 skipping (treated group), with a concurrent control arm of DMD patients not amenable to exon 51 skipping (untreated group). Patients in the treated group will receive once weekly intravenous (IV) infusions of 30 mg/kg eteplirsen. Patients in the untreated group will not receive treatment.
Clinical efficacy will be assessed at regularly scheduled study visits, including functional tests such as the six-minute walk test. Patients in the treated group will undergo a muscle biopsy at baseline and a second muscle biopsy over the course of the study. Patients in the untreated group will not undergo muscle biopsy.
Safety, including adverse event monitoring and routine laboratory assessments, will be continuously monitored for all patients.
For more information about the study or specific questions, contact Celeste DiJohnson or Charlene Brathwaite at firstname.lastname@example.org.