Pompe Disease Clinical Trial
Official title:
An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme®-Naive, CRIM(-) Patients With Infantile-onset Pompe Disease
The 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.
n/a
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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