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A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease

This study has been completed.
Sponsor:
Collaborator:
BioMarin/Genzyme LLC
Information provided by:
Genzyme, a Sanofi Company
ClinicalTrials.gov Identifier:
NCT00144781
First received: September 2, 2005
Last updated: August 20, 2009
Last verified: April 2009
  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).


Condition Intervention Phase
Mucopolysaccharidosis I
Hurler's Syndrome
Hurler-Scheie Syndrome
Scheie Syndrome
Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
Phase 4

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Multicenter, Multinational, Randomized, Dose-Optimization Study of the Safety and Pharmacodynamic Response of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I

Resource links provided by NLM:


Further study details as provided by Genzyme, a Sanofi Company:

Primary Outcome Measures:
  • Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Level [ Time Frame: Baseline to 26 Weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Percent Change From Baseline to Week 26 in Liver Organ Volume [ Time Frame: Baseline to 26 Weeks ] [ Designated as safety issue: No ]
  • Change From Baseline to Week 26 in Six Minute Walk Test (6MWT) [ Time Frame: Baseline to 26 Weeks ] [ Designated as safety issue: No ]

Enrollment: 34
Study Start Date: December 2004
Study Completion Date: January 2006
Primary Completion Date: January 2006 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Active Comparator: 1
0.58 mg Aldurazyme/kg of body weight (100 U/kg) administered every week (labeled dose). Final Visit is Week 27 for patients randomized to every week regimen.
Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
0.58 mg/kg every week
Active Comparator: 2
1.2 mg Aldurazyme/kg of body weight (200 U/kg) administered every week. Final Visit is Week 27 for patients randomized to every week regimen.
Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
1.2 mg/kg every week
Active Comparator: 3
1.2 mg Aldurazyme/kg of body weight (200 U/kg) administered every 2 weeks. Final Visit is Week 26 for patients randomized to every 2 week regimen.
Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
1.2 mg/kg every other week
Active Comparator: 4
1.8 mg Aldurazyme/kg of body weight (300 U/kg) administered every 2 weeks. Final Visit is Week 26 for patients randomized to every 2 week regimen.
Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
1.8 mg/kg every other week

  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
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT00144781

Locations
Brazil
Hospital Infantil Joana de Gusmao
Florianopolis, Santa Catarina, Brazil, CEP 88025-601
Universidade Federal de Minas Gerais
Belo Horizonte, Brazil, CEP 30130-100
Hospital de Clinical de Porto Alegre
Porto Alegre, Brazil, CEP 90035-003
Universidade Federal de Sao Paulo
San Paulo, Brazil, CEP 04023-062
Canada, Ontario
Division of Clinical and Metabolic Genetics
Toronto, Ontario, Canada, M5G 1X8
Sponsors and Collaborators
Genzyme, a Sanofi Company
BioMarin/Genzyme LLC
Investigators
Study Director: Medical Information Genzyme, a Sanofi Company
  More Information

No publications provided

Responsible Party: Medical Monitor, Genzyme Corporation
ClinicalTrials.gov Identifier: NCT00144781     History of Changes
Other Study ID Numbers: ALID-017-03
Study First Received: September 2, 2005
Results First Received: March 27, 2009
Last Updated: August 20, 2009
Health Authority: United States: Food and Drug Administration

Additional relevant MeSH terms:
Mucopolysaccharidoses
Mucopolysaccharidosis I
Syndrome
Carbohydrate Metabolism, Inborn Errors
Connective Tissue Diseases
Disease
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Metabolic Diseases
Metabolism, Inborn Errors
Mucinoses
Pathologic Processes

ClinicalTrials.gov processed this record on November 20, 2014