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Ataluren

Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (DMD)

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

Más ensayos de: Distrofia muscular de Duchenne.

Fase

Fase 2

Estado

Interrumpido

Participantes

36

previstos

Centros

3

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 ago 2008.

Este ensayo se detuvo. Motivo declarado por el promotor (del registro, en inglés): «Terminated early because similar study with Ataluren (PTC124-GD-007-DMD; NCT00592553) 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 Week 89) (del registro, en inglés)

Cómo lo describe el promotor

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

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Todos los de PTCT, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT00759876), actualizada en oct 2020.