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Ataluren

Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada

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

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

Fase

Fase 3

Estado

Completado

Participantes

94

previstos

Centros

21

Fin del objetivo primario

ene 2018

fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, de un solo grupo, abierto. Comenzó en may 2012.

Qué mide

Numero de Participantes con Acontecimientos Adversos Surgidos del Tratamiento (TEAE) (Baseline up to Week 246) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroDuchenne/Becker muscular dystrophy (DBMD) 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 DBMD in approximately 10-15% 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 comprises a Phase 3, open-label study of ataluren in participants with nmDBMD who previously received ataluren at an Investigator site in a prior PTC-sponsored clinical study. A separate open-label study (PTC124-GD-016-DMD; NCT01247207) is being conducted for nmDBMD participants who previously received ataluren at an Investigator site in the United States (US).

Más ensayos de PTCT

Todos los de PTCT, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT01557400), actualizada en nov 2020.