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Alglucosidase Alfa

An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme-naive Cross-Reacting Immunologic Material (CRIM[-]) Patients With Infantile-Onset Pompe Disease

Ensayo de Sanofi en Pompe Disease · Glycogen Storage Disease Type II (del registro, en inglés).

Fase

Fase 4

Estado

Completado

Participantes

4

previstos

Centros

2

Fin del objetivo primario

mar 2013

fecha real

Qué significa cada fase y cada estado.

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

Qué mide

Change From Baseline in Number of Patients With Anti-Recombinant Human Acid Alfa-glucosidase (Anti-rhGAA) Immunoglobulin G (IgG) Antibody at End of Study (Baseline, End of Study (up to Week 79 or early termination)) (del registro, en inglés)

Cómo lo describe el promotor

En el documentoEn inglés, del registroThe purpose of this study was to evaluate the efficacy, clinical benefits and safety of a prophylactic immunomodulatory regimen given prior to first treatment with alglucosidase alfa (Myozyme®) in patients with infantile-onset Pompe disease. The objectives were to assess the efficacy of a prophylactic immunomodulatory regimen given prior to first treatment with alglucosidase alfa, as assessed by anti-recombinant human acid alpha-glucosidase (anti-rhGAA) antibody titers, and antibodies that inhibit the activity and/or uptake of alglucosidase alfa; to evaluate the clinical benefit as measured by overall survival, ventilator-free survival, left ventricular mass index (LVMI), gross motor function and development, disability index and the incidence of adverse events (AEs), serious adverse events (SAEs), and clinical laboratory abnormalities.

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Ficha completa en ClinicalTrials.gov (NCT00701129), actualizada en abr 2014.