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Sponsors and Collaborators: |
Genzyme BioMarin/Genzyme LLC |
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Information provided by: | Genzyme |
ClinicalTrials.gov Identifier: | NCT00146757 |
The main objectives of this study are to evaluate the safety and pharmacokinetics (PK) of enzyme replacement therapy with recombinant human alpha-L-iduronidase [Aldurazyme® (laronidase)] in mucopolysaccharidosis I (MPS I) patients less than 5 years old. Efficacy measurements will also be evaluated in this study.
Condition | Intervention | Phase |
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Mucopolysaccharidosis I Hurler Syndrome Hurler-Scheie Syndrome Scheie Syndrome |
Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase) |
Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | A Phase II Open-Label Clinical Trial of Recombinant Human Alpha-L-iduronidase (Aldurazyme®) to Evaluate the Safety and Pharmacokinetics in Mucopolysaccharidosis I (MPS I) Patients Less Than 5 Years Old |
Enrollment: | 20 |
Study Start Date: | October 2002 |
Study Completion Date: | May 2005 |
Primary Completion Date: | May 2005 (Final data collection date for primary outcome measure) |
Arms | Assigned Interventions |
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Aldurazyme (rhIDU) 100 U/kg ONLY every week: Experimental
Patients received Aldurazyme (recombinant human alpha-L-iduronidase (rhIDU)) once per week at a dose of 100 Units/kg (approximately 0.58 mg/kg) for up to 52 weeks - labeled dose.
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Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
100 U/kg every week
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Aldurazyme (rhIDU) 100-200 U/kg every week: Experimental
After receiving 100 Units/kg dose of Aldurazyme (rhIDU) for the first 25 weeks, patients enrolling after January 1, 2004 were eligible to receive an increased dose of 200 Units/kg from Week 26 onwards if the patient's urinary glycosaminoglycan (uGAG) levels were >200µg/mg creatinine at Week 22.
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Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
200 U/kg every week (Week 26 onwards)
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Ages Eligible for Study: | up to 5 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
France | |
Hôpital E. Herriot | |
Lyon, France | |
Germany, Mainz | |
Johannes Gutenberg Universität | |
Kinderklinik, Mainz, Germany | |
Netherlands | |
Sophia Children's Hospital | |
Rotterdam, Netherlands | |
United Kingdom | |
Willink Biochemical Genetics Unit Royal Hospital for Children | |
Manchester, United Kingdom |
Study Director: | Medical Monitor | Genzyme |
Responsible Party: | Genzyme Corporation ( Medical Monitor ) |
Study ID Numbers: | ALID-014-02 |
Study First Received: | September 2, 2005 |
Results First Received: | November 20, 2008 |
Last Updated: | August 20, 2009 |
ClinicalTrials.gov Identifier: | NCT00146757 History of Changes |
Health Authority: | European Union: European Medicines Agency |
Metabolism, Inborn Errors Mucopolysaccharidoses Metabolic Diseases Genetic Diseases, Inborn |
Lysosomal Storage Diseases Connective Tissue Diseases Metabolic Disorder Mucopolysaccharidosis I |
Metabolism, Inborn Errors Mucopolysaccharidoses Pathologic Processes Disease Metabolic Diseases Genetic Diseases, Inborn |
Lysosomal Storage Diseases Syndrome Connective Tissue Diseases Mucinoses Carbohydrate Metabolism, Inborn Errors Mucopolysaccharidosis I |