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A Dose-Optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease
This study has been completed.
Sponsors and Collaborators: Genzyme
BioMarin/Genzyme LLC
Information provided by: Genzyme
ClinicalTrials.gov Identifier: NCT00144781
  Purpose

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
Mucopolysaccharidosis I
Hurler's Syndrome
Hurler-Scheie Syndrome
Scheie Syndrome
Drug: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
Phase IV

Genetics Home Reference related topics: mucopolysaccharidosis type I
Drug Information available for: Laronidase
U.S. FDA Resources
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

Further study details as provided by Genzyme:

Primary Outcome Measures:
  • To evaluate differences in the pharmacodynamic response and safety of Aldurazyme® (laronidase) does regimens

Study Start Date: April 2004
Estimated Study Completion Date: January 2006
  Eligibility

Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Have a documented diagnosis of MPS I, confirmed by measurable clinical signs and symptoms of MPS I, and a documented fibroblast or leukocyte α-L-iduronidase enzyme activity level of less than 10% of the lower limit of the normal range of the measuring laboratory.
  • Weigh at least 12.5 kg.
  • Have serum creatinine and blood urea nitrogen (BUN) values within age appropriate normal ranges.
  • Provide signed, written informed consent prior to any protocol-related procedures being performed. Consent of a legally authorized guardian(s) is (are) required for patients under 18 years. If the patient is under 18 years old and can understand the consent, written informed consent will be required from both the patient and the authorized guardian(s).

Exclusion Criteria:

  • Have previously received Aldurazyme® (laronidase).
  • Have a suspected hypersensitivity to Aldurazyme® (laronidase) or known hypersensitivity to components of the infusion solution.
  • Have previously undergone hematopoietic stem cell transplantation (HSCT; i.e., from bone marrow [BMT], peripheral blood, or umbilical cord blood) or other major organ transplantation.
  • Have a medical condition, serious inter-current illness, or other extenuating circumstance that may interfere with study compliance including all prescribed evaluations and follow-up activities, except the 6MWT.

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.

  • Have an acute illness that requires surgical intervention, and/or anticipates surgery during study participation, and/or has had surgery within 30 days prior to study enrollment.
  • Have received an investigational drug within 30 days prior to study enrollment.
  • Is pregnant or lactating. Female patients of childbearing potential must have a negative pregnancy test [urine β-human chronic gonadotropin (hCG)] at entry (prior to the first infusion). Note: All female patients of childbearing potential and sexually mature males must be advised to use a medically accepted method of contraception throughout the study.
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00144781

Locations
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
Sponsors and Collaborators
Genzyme
BioMarin/Genzyme LLC
  More Information

US FDA Approved Full Prescribing Information for Aldurazyme®  This link exits the ClinicalTrials.gov site

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

Study placed in the following topic categories:
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

Additional relevant MeSH terms:
Pathologic Processes
Disease
Syndrome
Mucinoses
Carbohydrate Metabolism, Inborn Errors

ClinicalTrials.gov processed this record on January 16, 2009