Researchers at PTC Therapeutics are looking for participants with Duchenne muscular dystrophy to participate in a Phase 3 study. The goal of the study is to evaluate the safety and efficacy of Translarna (ataluren). The drug is believed to promote the formation of full-length and functional dystrophin protein in boys with a nonsense (or premature stop codon) mutation. Participants will be randomized to receive either ataluren or placebo, to determine whether treatment with ataluren is associated with stabilized or slowed disease progression.
Total study duration for each patient will be approximately 144 weeks, with 12 clinic visits (one every three months) involving a series of functional tests including a six-minute walk test, timed function tests, the North Star Ambulatory Assessment, performance of upper limb in patients 7 years old or younger at baseline, myometry in patients younger than 7 years old at baseline, magnetic resonance imaging (MRI) and the EQ-5D in addition to laboratory blood tests and physical examination to monitor for adverse events. Throughout the study, participants will continue to see their regular doctor for routine care.
In order to be eligible to participate, individuals must be:
- Male, older than 5 years old
- Genetically diagnosed with a nonsense (premature stop codon) mutation in the dystrophin gene.
- Receiving systemic corticosteroids (prednisone/prednisolone or deflazacort) for a minimum of 12 months (with no significant change in dosage or dosing regimen for a minimum of three months) prior to the start of the study
- Able to walk 150 meters or more within six minutes (6MWD≥150 meters)
- Able to perform timed function tests within 30 seconds
- Able to meet additional study criteria
To learn more or to inquire about participation, contact Mary Frances Harmon at 908-912-9256 or firstname.lastname@example.org.