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Ataluren

Phase 2B Study of PTC124 (Ataluren) 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

Completado

Participantes

174

previstos

Centros

37

Fin del objetivo primario

dic 2009

fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, aleatorizado, cuádruple ciego. Comenzó en feb 2008.

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 registroDMD/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 13 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. This study is a Phase 2b trial that will evaluate the clinical benefit of ataluren in boys with DMD/BMD due to a nonsense mutation. The main goals of the study are to understand whether ataluren can improve walking, activity, muscle function, and strength and whether the drug can safely be given for a long period of time.

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Todos los de PTCT, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT00592553), actualizada en mar 2020.