Status:
COMPLETED
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
Lead Sponsor:
Genzyme, a Sanofi Company
Conditions:
Pompe Disease
Glycogen Storage Disease Type II
Eligibility:
All Genders
Phase:
PHASE4
Brief 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 pat...
Eligibility Criteria
Inclusion
- The patient's legal guardian(s) must have provided written informed consent prior to any study-related procedures being performed
- The patient must have had a clinical diagnosis of Pompe disease as defined by documented acid alpha-glucosidase (GAA) deficiency (deficient endogenous GAA activity) in skin fibroblasts, muscle, or blood, or 2 GAA mutations. Consent was also sought from the biological parent(s) for parental GAA mutational analysis, but was not a requirement for study eligibility
- The patient must have not received Myozyme® or any rhGAA therapies prior to enrollment in the study
- The patient must be CRIM negative via Western Blot analysis performed on skin fibroblasts or via 2 known CRIM negative mutations (in which case CRIM status was to be confirmed by Western Blot analysis after enrollment)
- The patient's legal guardian(s) must have the ability to comply with the clinical protocol
Exclusion
- The patient had any medical condition that, in the opinion of the Investigator, could be exacerbated/precipitated by or interfere with the study regimen or assessments; such conditions may include but were not limited to human immunodeficiency virus, cancer, Hepatitis B, Hepatitis C, Cytomegalovirus, Herpes Simplex, John Cunningham (JC) virus, Parvovirus, or Epstein Barr virus or tuberculosis
- The patient had used any investigational product within 30 days prior to study enrollment
- The patient had or was required to have any live vaccination within 1 month prior to enrollment
Key Trial Info
Start Date :
October 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 1 2013
Estimated Enrollment :
4 Patients enrolled
Trial Details
Trial ID
NCT00701129
Start Date
October 1 2009
End Date
March 1 2013
Last Update
May 13 2014
Active Locations (2)
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1
Kosair Children's Hospital
Louisville, Kentucky, United States, 40202
2
Duke University Medical Center
Durham, North Carolina, United States, 27710