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Ataluren
Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation-Mediated Duchenne/Becker Muscular Dystrophy (nmDMD/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
- 6
- Centros
- 6
- Fin del objetivo primario
- mar 2010
Qué significa cada fase y cada estado.
Estudio de intervención, de un solo grupo, abierto. Comenzó en ene 2010.
Este ensayo se detuvo. Motivo declarado por el promotor (del registro, en inglés): «Terminated early because a similar study with Ataluren exhibited lack of efficacy at the high dose (not due to safety concerns).».
Qué mide
Number of Participants With Treatment Emergent Adverse Events (TEAEs) (Baseline up to Day 50) (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 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.
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Ficha completa en ClinicalTrials.gov (NCT01009294), actualizada en jul 2020.