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Sponsors and Collaborators: |
Genzyme BioMarin/Genzyme LLC |
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Information provided by: | Genzyme |
ClinicalTrials.gov Identifier: | NCT00144781 |
The main purpose of this study is to evaluate differences in the pharmacodynamic response of 4 Aldurazyme® (laronidase) dose regimens in patients with Mucopolysaccharidosis I (MPS I) disease through measuring urinary GAG levels, liver volume, and functional tests (6-minute walk test).
Condition | Intervention | Phase |
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Mucopolysaccharidosis I Hurler's Syndrome Hurler-Scheie Syndrome Scheie Syndrome |
Drug: Aldurazyme (Recombinant Human Alpha-L-Iduronidase) |
Phase IV |
Study Type: | Interventional |
Study Design: | Treatment, Randomized, Open Label, Dose Comparison, Parallel Assignment, Safety/Efficacy Study |
Official Title: | A Multicenter, Multinational, Randomized, Dose-Optimization Study of the Safety and Pharmacodynamic Response of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Note: All patients may not be capable of performing the 6MWT due to age and/or maturity level. Exemption from performing the 6MWT must be obtained in writing by the investigator from the sponsor's medical monitor prior to enrollment.
Brazil | |
Hospital de Clinical de Porto Alegre | |
Porto Alegre, Brazil, CEP 90035-003 | |
Universidade Federal de Sao Paulo | |
San Paulo, Brazil, CEP 04023-062 | |
Hospital das Clinicas de Faculdade de Medicina da Universidade Federal de Minas Gerais | |
Belo Horizonte, Brazil, CEP 30130-100 | |
Brazil, Santa Catarina | |
Hospital Infantil Joana de Gusmao | |
Florianopolis, Santa Catarina, Brazil, CEP 88025-601 | |
Canada, Ontario | |
Division of Clinical and Metabolic Genetics | |
Toronto, Ontario, Canada, M5G 1X8 |
Study ID Numbers: | ALID-017-03 |
Study First Received: | September 2, 2005 |
Last Updated: | December 14, 2007 |
ClinicalTrials.gov Identifier: | NCT00144781 |
Health Authority: | United States: Food and Drug Administration |
Alpha-L-iduronidase deficiency Metabolism, Inborn Errors Mucopolysaccharidoses Metabolic Diseases Genetic Diseases, Inborn Lysosomal Storage Diseases |
Connective Tissue Diseases Mucopolysaccharidosis Metabolic disorder Hurler syndrome Mucopolysaccharidosis I |
Pathologic Processes Disease Syndrome Mucinoses Carbohydrate Metabolism, Inborn Errors |