rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)

This study has been completed.
Sponsor:
Information provided by:
Sanofi
ClinicalTrials.gov Identifier:
NCT00053573
First received: January 31, 2003
Last updated: February 4, 2014
Last verified: February 2014

January 31, 2003
February 4, 2014
February 2003
July 2006   (final data collection date for primary outcome measure)
  • Evaluate the safety of Myozyme [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
  • Determine proportion of patients alive over the course of treatment [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
  • PK profile of MZ [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
  • PD profile of MZ [ Time Frame: 52 weeks ] [ Designated as safety issue: No ]
Not Provided
Complete list of historical versions of study NCT00053573 on ClinicalTrials.gov Archive Site
Not Provided
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rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)
An Open-Label, Multicenter, Multinational, Study of the Safety, Efficacy, Pharmacokinetics, and Pharmacodynamics of rhGAA Treatment in Patients Greater Than 6 Months and Less Than or Equal to 36 Months Old With Infantile-Onset GSD-II

Glycogen Storage Disease Type II ("GSD-II"; also known as Pompe disease) is caused by a deficiency of a critical enzyme in the body called acid alpha-glucosidase (GAA). Normally, GAA is used by the body's cells to break down glycogen (a stored form of sugar) within specialized structures called lysosomes. In patients with GSD-II, an excessive amount of glycogen accumulates and is stored in various tissues, especially heart and skeletal muscle, which prevents their normal function. This study is being conducted to evaluate the safety and effectiveness of recombinant human acid alpha-glucosidase (rhGAA) as a potential enzyme replacement therapy for GSD-II. Patients diagnosed with infantile-onset GSD-II who are greater than 6 months old, but less than or equal to 36 months old will be studied.

Not Provided
Interventional
Phase 1
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
  • Glycogen Storage Disease Type II
  • Pompe Disease
  • Acid Maltase Deficiency Disease
  • Glycogenosis 2
Biological: Myozyme
20 mg/kg to 40 mg/kg qow
Other Name: Alglucosidase alfa
Experimental: 1
Intervention: Biological: Myozyme
Kishnani PS, Goldenberg PC, DeArmey SL, Heller J, Benjamin D, Young S, Bali D, Smith SA, Li JS, Mandel H, Koeberl D, Rosenberg A, Chen YT. Cross-reactive immunologic material status affects treatment outcomes in Pompe disease infants. Mol Genet Metab. 2010 Jan;99(1):26-33.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
20
November 2006
July 2006   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • The patient or the patient's legal guardian(s) must provide written informed consent prior to any study-related procedures being performed
  • The patient must have a clinical diagnosis of infantile GSD-II as defined by: (a) the patient has/had documented (in a medical record) onset of symptoms compatible with GSD-II by 12 months of age; (b) the patient has documented GAA deficiency as illustrated by an endogenous GAA activity less than or equal to 2% of the mean of the normal range as assessed in cultured skin fibroblasts; AND (c) the patient has a Left Ventricular Mass Index greater than 2 standard deviations above the mean for age
  • The patient is greater than 6 months old and less than or equal to 36 months old at the time of the first dose of rhGAA
  • The patient and his/her legal guardian(s) must have the ability to comply with the clinical protocol

Exclusion Criteria:

  • Signs and symptoms of cardiac failure and an ejection fraction less than 40%
  • Major congenital abnormality
  • Clinically significant organic disease (with the exception of symptoms relating to GSD-II), including clinically significant cardiovascular, hepatic, pulmonary, neurologic, or renal disease, or other medical condition, serious intercurrent illness, or extenuating circumstance that, in the opinion of the Investigator, would preclude participation in the trial or potentially decrease survival
  • Use of any investigational product within 30 days prior to study enrollment
  • Received enzyme replacement therapy with GAA from any source
Both
6 Months to 36 Months
No
Contact information is only displayed when the study is recruiting subjects
United States,   France,   Israel,   United Kingdom
 
NCT00053573
AGLU01702
Not Provided
Medical Monitor, Genzyme Corporation
Genzyme, a Sanofi Company
Not Provided
Study Director: Medical Monitor Genzyme, a Sanofi Company
Sanofi
February 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP