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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: |
NCT00059280 |
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Date of registration:
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22/04/2003 |
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Prospective Registration:
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No |
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Primary sponsor: |
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Public title:
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A Study of the Safety and Efficacy of rhGAA in Patients With Infantile-onset 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 Recombinant Human Acid Alpha-glucosidase Treatment in Patients Less Than 6 Months Old With Infantile-onset Pompe Disease |
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Date of first enrolment:
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April 2003 |
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Target sample size:
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16 |
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Recruitment status: |
Completed |
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URL:
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http://clinicaltrials.gov/show/NCT00059280 |
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Study type:
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Interventional |
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Study design:
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Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Factorial Assignment, Masking: Open Label, Primary Purpose: Treatment
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Phase:
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Phase 2/Phase 3
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Countries of recruitment
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France
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Israel
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Taiwan
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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 clinical symptoms (documented in his or her medical record) of
infantile-onset Pompe disease. In addition, the patient must have: a. an endogenous
GAA activity less than 1% of the mean of the normal range as assessed in cultured
skin fibroblasts; AND b. cardiomyopathy (LVMI greater than 65 g/m2) by
echocardiography;
- The patient must be no older than 26 weeks and 0 days, when he/she receives 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:
- Symptoms of respiratory insufficiency, including: a. Oxygen saturation less than 90%
in room air as measured by pulse oximetry; OR b. venous PCO2 greater than 55 mmHg on
room air OR arterial PCO2 greater than 40 mmHg on room air; c. any ventilator use at
the time of enrollment;
- Major congenital abnormality;
- Clinically significant organic disease (with the exception of symptoms relating to
Pompe disease), 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:
N/A
Age maximum:
26 Weeks
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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Intervention(s)
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Biological: Myozyme
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Primary Outcome(s)
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Determine PK/PD profile of MZ
[Time Frame: 52 weeks]
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Evaluate the safety profile of MZ
[Time Frame: 52 weeks]
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To estimate the proportion of patients treated w/ MZ who were alive and free of ventilator support at 12 months of age; compared to historical cohort
[Time Frame: 52 weeks]
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Determine effect of different doses of MZ on safety and efficacy
[Time Frame: 52 weeks]
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Secondary ID(s)
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AGLU01602
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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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