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Main
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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register. |
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Register:
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ClinicalTrials.gov |
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Last refreshed on:
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19 February 2015 |
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Main ID: |
NCT00053573 |
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Date of registration:
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31/01/2003 |
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Prospective Registration:
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Yes |
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Primary sponsor: |
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Public title:
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rhGAA in Patients With Infantile-onset Glycogen Storage Disease-II (Pompe Disease)
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Scientific title:
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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 |
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Date of first enrolment:
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February 2003 |
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Target sample size:
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20 |
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Recruitment status: |
Completed |
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URL:
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http://clinicaltrials.gov/show/NCT00053573 |
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Study type:
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Interventional |
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Study design:
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Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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Phase:
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Phase 1/Phase 2
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Countries of recruitment
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France
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Israel
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United Kingdom
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United States
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Contacts
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Name:
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Medical Monitor |
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Address:
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Telephone:
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Email:
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Affiliation:
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Genzyme, a Sanofi Company |
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Key inclusion & exclusion criteria
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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
Age minimum:
6 Months
Age maximum:
36 Months
Gender:
Both
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Health Condition(s) or Problem(s) studied
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Glycogen Storage Disease Type II
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Glycogenosis 2
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Acid Maltase Deficiency Disease
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Pompe Disease
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Intervention(s)
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Biological: Myozyme
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Primary Outcome(s)
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Determine proportion of patients alive over the course of treatment
[Time Frame: 52 weeks]
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Evaluate the safety of Myozyme
[Time Frame: 52 weeks]
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PK profile of MZ
[Time Frame: 52 weeks]
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PD profile of MZ
[Time Frame: 52 weeks]
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Secondary ID(s)
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AGLU01702
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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Results
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Results available:
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Date Posted:
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Date Completed:
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URL:
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