|
Home
Search
Study Topics
Glossary
|
![]() |
![]() |
|
![]() |
|
![]() |
|
![]() |
![]() |
![]() |
|
![]() |
![]() |
||||||||||||||||||||||||||||||||||||
| Sponsor: | Genzyme |
|---|---|
| Collaborator: |
BioMarin/Genzyme LLC |
| Information provided by: | Genzyme |
| ClinicalTrials.gov Identifier: | NCT00146757 |
Purpose
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 |
|---|---|---|
|
Mucopolysaccharidosis I Hurler Syndrome Hurler-Scheie Syndrome Scheie Syndrome |
Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase) |
Phase II |
| Study Type: | Interventional |
| Study Design: | Allocation: Non-Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| 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 |
|---|---|
|
Experimental: Aldurazyme (rhIDU) 100 U/kg ONLY every week
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.
|
Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
100 U/kg every week
|
|
Experimental: Aldurazyme (rhIDU) 100-200 U/kg every week
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.
|
Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
200 U/kg every week (Week 26 onwards)
|
Eligibility| Ages Eligible for Study: | up to 5 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Contacts and Locations| France | |
| Hôpital E. Herriot | |
| Lyon, France | |
| Germany | |
| 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 |
More Information
| Responsible Party: | Medical Monitor, Genzyme Corporation |
| ClinicalTrials.gov Identifier: | NCT00146757 History of Changes |
| Other Study ID Numbers: | ALID-014-02 |
| Study First Received: | September 2, 2005 |
| Results First Received: | November 20, 2008 |
| Last Updated: | August 20, 2009 |
| Health Authority: | European Union: European Medicines Agency |
|
Mucopolysaccharidosis I Mucopolysaccharidoses Carbohydrate Metabolism, Inborn Errors Metabolism, Inborn Errors Genetic Diseases, Inborn |
Lysosomal Storage Diseases Mucinoses Connective Tissue Diseases Metabolic Diseases |