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Ataluren

Study of Ataluren (PTC124) in Hemophilia A and B

Ensayo de PTC THERAPEUTICS, INC. en Hemophilia A · Hemophilia B (del registro, en inglés).

Más ensayos de: Hemofilia A.

Fase

Fase 2

Estado

Interrumpido

Participantes

13

previstos

Centros

13

Fin del objetivo primario

ago 2011

fecha real

Qué significa cada fase y cada estado.

Estudio de intervención, de un solo grupo, abierto. Comenzó en oct 2009.

Este ensayo se detuvo. Motivo declarado por el promotor (del registro, en inglés): «Study was terminated early due to Sponsor decision and not reflective of adverse safety findings.».

Qué mide

Number of Participants With a Plasma FVIII/FIX Activity Response at Day 14 (Baseline up to Day 14) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroHemophilia A (HA) and hemophilia B (HB) are inherited bleeding disorders caused by mutations in the gene for factor VIII (FVIII) and factor IX (FIX), respectively. These proteins are essential for blood clotting. The lack of FVIII/FIX can produce bleeding episodes that cause damage of the bone, muscles, joints, and tissues. A specific type of mutation, called a nonsense (premature stop codon) mutation, is the cause of the disease in approximately 10-30% of participants with hemophilia and results in severe manifestations. Ataluren (PTC124) is an orally delivered, investigational drug that acts to overcome the effects of the premature stop codon, potentially enabling the production of functional FVIII/FIX. This study is a Phase 2a trial evaluating the safety and efficacy of ataluren in participants with HA or HB due to a nonsense mutation. The main purpose of this study is to understand whether ataluren can safely increase FVIII/FIX activity levels.

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Todos los de PTCT, en su ficha.

Ficha completa en ClinicalTrials.gov (NCT00947193), actualizada en may 2020.