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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 October 2017 |
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Main ID: |
NCT00299000 |
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Date of registration:
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02/03/2006 |
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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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A Phase 4 Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With MPS VI
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Scientific title:
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A Phase 4 Multi-center, Multi-national, Open-label, Randomized, Two Dose Level Study of Naglazyme(TM) (Galsulfase) in Infants With Maroteaux-Lamy Syndrome (MPS VI) |
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Date of first enrolment:
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May 2006 |
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Target sample size:
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4 |
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Recruitment status: |
Completed |
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URL:
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https://clinicaltrials.gov/show/NCT00299000 |
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Study type:
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Interventional |
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Study design:
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Phase:
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Phase 4
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Countries of recruitment
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France
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Portugal
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United States
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Contacts
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Name:
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Celeste Decker, MD |
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Address:
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Telephone:
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Email:
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Affiliation:
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BioMarin Pharmaceutical |
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Key inclusion & exclusion criteria
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Inclusion Criteria:
- Signed informed consent by a parent or legal guardian
- Parent or legal guardian willing and able to comply with all study procedures
- Equal to or greater than 36 weeks estimated gestational age by physical exam at birth
- Has a diagnosis of MPS VI based on a documented prenatal diagnosis or fibroblast or
leukocyte N-acetylgalactosamine 4-sulfatase (ASB) enzyme activity level of less than
10% of the lower limit of the normal range of the measuring laboratory
- Is less than one year of age
- Has no evidence of skeletal dysplasia based on physical exam
Exclusion Criteria:
- Parent of legal guardian perceived to be unreliable or unavailable for study
participation
- Use of any investigational drug within 30 days prior to screening, or requirement for
any investigational agent prior to completion of all scheduled study assessments
- Has concurrent disease or condition that would interfere with study participation or
safety (i.e., has previously undergone hematopoietic stem cell transplantation such as
bone marrow or cord blood transplantation, or major organ transplantation)
- Any condition that, in the view of the principle investigator, renders the subject at
high risk from treatment compliance and/or completing the study
- Has known hypersensitivity to Naglazyme
- Has previously received Naglazyme
Age minimum:
N/A
Age maximum:
1 Year
Gender:
All
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Health Condition(s) or Problem(s) studied
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Mucopolysaccharidosis VI
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Maroteaux-Lamy Syndrome
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Intervention(s)
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Drug: Naglazyme
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Primary Outcome(s)
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Change in Height
[Time Frame: 52 weeks]
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Change in Weight
[Time Frame: 52 weeks]
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Change in Haed Circumference
[Time Frame: 52 weeks]
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Secondary Outcome(s)
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Change in Urinary Glycosaminoglycan Levels
[Time Frame: minimum 52 weeks of dosing]
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Source(s) of Monetary Support
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Please refer to primary and secondary sponsors
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