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Ataluren · ACT DMD
Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
Ensayo de PTC THERAPEUTICS, INC. en Muscular Dystrophy, Duchenne · Muscular Dystrophies · Muscular Disorders, Atrophic · Muscular Diseases · Musculoskeletal Diseases · Neuromuscular Diseases · Nervous System Diseases · Genetic Diseases, X-Linked · Genetic Diseases, Inborn (del registro, en inglés).
Más ensayos de: Distrofia muscular de Duchenne · Distrofias musculares · Enfermedades neuromusculares · Enfermedades del sistema nervioso.
- Fase
- Fase 3
- Estado
- Completado
- Participantes
- 230
- Centros
- 55
- Fin del objetivo primario
- ago 2015
Qué significa cada fase y cada estado.
Estudio de intervención, aleatorizado, cuádruple ciego. Comenzó en mar 2013.
Qué mide
Change From Baseline in 6MWD at Week 48 (del registro, en inglés) (inicio y semana 48)
Cómo lo describe el promotor
En el documentoEn inglés, del registroDystrophinopathy is a disease continuum that includes Duchenne muscular dystrophy, which 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. A specific type of mutation, called a nonsense (premature stop codon) mutation is the cause of dystrophinopathy in approximately 10-15 percent (%) of boys with the disease. Ataluren is an orally delivered, investigational drug that has the potential to overcome the effects of the nonsense mutation. The main goal of this Phase 3 study is to evaluate the effect of ataluren on walking ability. The effect of ataluren on physical function, quality of life, and activities of daily living will be evaluated. This study will also provide additional information on the long-term safety of ataluren.
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Todos los de PTCT, en su ficha.
Ficha completa en ClinicalTrials.gov (NCT01826487), actualizada en jul 2020.