Duchenne Muscular Dystrophy Clinical Trial
Official title:
A Phase 2a Study of Ataluren (PTC124) in Nonambulatory Patients With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy
Duchenne/Becker muscular dystrophy (DMD/BMD) is a genetic disorder that develops in boys. It is caused by a mutation in the gene for dystrophin, a protein that is important for maintaining normal muscle structure and function. Loss of dystrophin causes muscle fragility that leads to weakness and loss of walking ability during childhood and teenage years. A specific type of mutation, called a nonsense (premature stop codon) mutation is the cause of DMD/BMD in approximately 10-15% of boys with the disease. Ataluren (PTC124) is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. This study is a Phase 2a trial that enrolled boys with nonsense mutation DMD/BMD who have lost independent mobility due to the disease. This study evaluated the safety and tolerability of ataluren (PTC124) and also evaluated efficacy outcomes in this participant population.
It was planned that this Phase 2a, open-label, safety and efficacy study to be performed at 5
sites in the US and 1 site in the United Kingdom.
The study was to enroll ~30 boys with nonsense mutation DMD/BMD (nmDBMD) who have been
nonambulatory for at least 1 year. Enrollment was to be stratified to ensure evaluation of
~15 participants who were receiving chronic corticosteroid therapy and of ~15 participants
who were not receiving chronic corticosteroid therapy. Participants were to take ataluren 3
times per day (at breakfast, lunch, and dinner) for 48 weeks (~1 year). Study assessments
were to be performed at clinic visits during screening, every 6 weeks for 2 visits and then
every 12 weeks until the end of the study. Additional safety laboratory testing was to be
required 4 times during the course of the study; this could have been performed at the
investigational site, at an accredited local laboratory or clinic, or in the participant's
home using a nursing service. When the blind for a similar study (PTC124-GD-007-DMD;
NCT00592553) was revealed, the results indicated lack of efficacy for the high dose.
Therefore, even though an independent data monitoring committee (DMC) agreed that both
ataluren dose levels were well tolerated by the participants, the DMC recommended
discontinuing ongoing studies with participants with nmDBMD receiving high-dose ataluren.
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