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Ataluren

Extension 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

191

previstos

Centros

31

Fin del objetivo primario

dic 2013

fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, no aleatorizado, abierto. Comenzó en ago 2010.

Qué mide

Porcentaje de participantes con acontecimientos adversos surgidos del tratamiento (TEAE) (Baseline (Week 1 [Total Study Week 48]) up to 4 Weeks Post-Treatment (Week 100 [Total Study Week 148]) or Premature Discontinuation (PD) (whichever occurred first)) (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 extension trial that will evaluate the long-term safety of ataluren in adult and pediatric participants with nonsense mutation CF (nmCF), as determined by adverse events and laboratory abnormalities. The study will also assess changes in pulmonary function, CF pulmonary exacerbations, health-related quality of life, antibiotic use for CF-related infections, CF-related disruptions to daily living, body weight, and CF pathophysiology. Funding source for this study is the FDA OOPD.

Más ensayos de PTCT

Todos los de PTCT, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT01140451), actualizada en oct 2020.