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Ataluren

Study of Ataluren (PTC124™) in Cystic Fibrosis

Ensayo de PTC THERAPEUTICS, INC. en Fibrosis quistica.

Más ensayos de: Fibrosis quística.

Fase

Fase 3

Estado

Completado

Participantes

238

previstos

Centros

38

Fin del objetivo primario

nov 2011

fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, aleatorizado, cuádruple ciego. Comenzó en sep 2009.

Qué mide

Percentage of Predicted Function (Percent-Predicted) of Forced Expiratory Volume in One Second (FEV1) at Baseline (Baseline (Week 1)) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroCystic fibrosis (CF) is a genetic disorder caused by a mutation in the gene that makes the cystic fibrosis transmembrane conductance regulator (CFTR) protein. A specific type of mutation called a nonsense (premature stop codon) mutation is the cause of CF in approximately 10% of patients 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 3 trial that will evaluate the clinical benefit of ataluren in adult and pediatric participants with CF due to a nonsense mutation. The main goals of the study are to understand whether ataluren can improve pulmonary function and whether the drug can safely be given for a long period of time. The study will also assess the effects of ataluren on CF pulmonary exacerbation frequency, cough frequency, health-related quality of life, antibiotic use for CF-related infections, CF-related disruptions to daily living, body weight, and CF pathophysiology.

Más ensayos de PTCT

Todos los de PTCT, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT00803205), actualizada en may 2020.