Resumen

This is an open-label extension study for participants who completed a Phase 3, placebo-controlled study of ataluren in participants with nonsense mutation cystic fibrosis (nmCF) not receiving chronic inhaled aminoglycosides.

Descripción detallada

The primary objective of this Phase 3 extension study will be to obtain long-term safety data to augment the overall safety database. The secondary objectives will be to augment the efficacy data collected in the double-blind study (PTC124-GD-021-CF; NCT02139306).

Elegibilidad

Edad mínima: 6 YearsSexo: ALL

Criterios de inclusión

Completion of study treatment (placebo or active) in the previous Phase 3, double-blind study protocol (Protocol PTC124-GD-021-CF)
Evidence of signed and dated informed consent/assent document(s) indicating that the participant (and/or the participant's parent/legal guardian) has been informed of all pertinent aspects of the trial.

Criterios de exclusión

Known hypersensitivity to any of the ingredients or excipients of the study drug.
Ongoing participation in any other therapeutic clinical trial.

Intervenciones

drug

Ataluren

Ataluren will be provided as a vanilla-flavored powder to be mixed with water or milk.

Ubicaciones

Hospital de Ninos Ricardo Gutierrez

Buenos Aires, Argentina

Hospital Universitario Austral