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Ataluren

Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

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

Más ensayos de: Distrofia muscular de Duchenne.

Fase

Fase 3

Estado

Completado

Participantes

270

previstos

Centros

34

Fin del objetivo primario

feb 2026

fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, de un solo grupo, abierto. Comenzó en nov 2010.

Qué mide

Numero de participantes con acontecimientos adversos (Baseline up to end of study (up to approximately 8 years)) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroThe objective of this study is to assess the safety and tolerability of 10, 10, 20 milligrams per kilogram (mg/kg) ataluren in participants with nmDBMD who had prior exposure to ataluren in a PTC sponsored clinical trial or treatment plan, and siblings of those participants (provided those participants have completed the placebo-controlled portion of the trial). The treatment will continue under this protocol until consent withdrawal by participants, withdrawal due to worsen condition after initiating ataluren treatment, withdrawal by investigator, withdrawal due to participant unable to tolerate ataluren, participant is eligible to participate in another ataluren nmDBMD clinical trial program initiated by sponsor, study is discontinued by the relevant regulatory authority and/or sponsor, or until ataluren becomes commercially available.

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Todos los de PTCT, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT01247207), actualizada en mar 2026.