Calendario›PTCT›Ataluren

Ataluren

Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystrophy (DMD/BMD)

Ensayo de PTC THERAPEUTICS, INC. en Distrofia muscular de Duchenne · Distrofia muscular de Becker.

Más ensayos de: Distrofia muscular de Duchenne · Distrofia muscular de Becker.

Fase

Fase 2

Estado

Interrumpido

Participantes

173

previstos

Centros

37

Fin del objetivo primario

may 2010

fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, de un solo grupo, abierto. Comenzó en ene 2009.

Qué mide

Number of Participants With Treatment-Emergent Adverse Events (AEs) (Baseline (Week 48 of Study 007) up to Week 102) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroDuchenne/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 2b extension trial that will evaluate the long-term safety of ataluren (PTC124) in boys with nonsense mutation DMD/BMD, as determined by adverse events and laboratory abnormalities. The study will also assess changes in walking, muscle function, and other important clinical and laboratory measures.

Más ensayos de PTCT

Todos los de PTCT, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT00847379), actualizada en jun 2020.