Calendario›SNY›Olipudase alfa · ASCEND
Olipudase alfa · ASCEND
Efficacy, Safety, Pharmacodynamic, and Pharmacokinetics Study of Olipudase Alfa in Patients With Acid Sphingomyelinase Deficiency
Ensayo de Sanofi en Sphingomyelin Lipidosis (del registro, en inglés).
- Fase
- Fase 2/3
- Estado
- Completado
- Participantes
- 36
- Centros
- 24
- Fin del objetivo primario
- mar 2021
previstos
fecha real
Qué significa cada fase y cada estado.
Estudio de intervención, aleatorizado, cuádruple ciego. Comenzó en dic 2015.
Qué mide
Percent Predicted (% Predicted) Hemoglobin (Hb) and Altitude-Adjusted Diffusing Capacity of the Lung for Carbon Monoxide (DLco) at Baseline (Baseline (Day 1)) (del registro, en inglés)
Cómo lo describe el promotor
En el documentoEn inglés, del registroPrimary Objective: The primary objective of this phase 2/3 study was to evaluate the efficacy of olipudase alfa (recombinant human acid sphingomyelinase) administered intravenously once every 2 weeks for 52 weeks in adult participants with acid sphingomyelinase deficiency (ASMD) by assessing changes in: 1) spleen volume as measured by abdominal magnetic resonance imaging (MRI) (and, for the United States \[US\] only, in association with participant perception related to spleen volume as measured by splenomegaly-related score \[SRS\]); and 2) infiltrative lung disease as measured by the pulmonary function test, diffusing capacity of the lung for carbon monoxide (DLCO). Secondary Objectives: * To confirm the safety of olipudase alfa administered intravenously once every 2 weeks for 52 weeks. * To characterize the effect of olipudase alfa on the participant perception related to spleen volume as measured by the SRS after 52 weeks of study drug administration. (For the US, the effect of olipudase alfa on the SRS is part of the primary objective). * To characterize the effect of olipudase alfa after 52 weeks of study drug administration on the following outcome measures assessed sequentially: * The effect of olipudase alfa on liver volume; * The effect of olipudase alfa on platelet count; * The effect of olipudase alfa on fatigue; * The effect of olipudase alfa on pain; * The effect of olipudase alfa on dyspnea.
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Todos los de SNY, en su ficha.
Ficha completa en ClinicalTrials.gov (NCT02004691), actualizada en oct 2024.