NCT00701129

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

An Exploratory Study of the Safety and Efficacy of Prophylactic Immunomodulatory Treatment in Myozyme®-Naive, CRIM(-) Patients With Infantile-onset Pompe Disease

• Status: Completed
• Phase: Phase 4
• Study type: Interventional
• Enrollment: 4 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

Summary

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.

Conditions

• Pompe Disease
• Glycogen Storage Disease Type II

Interventions

Type	Name	Description
BIOLOGICAL	Alglucosidase Alfa	Administered as IV infusion.
DRUG	Methotrexate	Administered subcutaneously.
DRUG	Rituximab	Administered as IV infusion.

Timeline

• Start date: 2009-10-01
• Primary completion: 2013-03-01
• Completion: 2013-03-01
• First posted: 2008-06-19
• Last updated: 2014-05-13
• Results posted: 2014-05-13

Locations

2 sites across 1 country: United States