Status:
COMPLETED
A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease
Lead Sponsor:
Genzyme, a Sanofi Company
Collaborating Sponsors:
BioMarin/Genzyme LLC
Conditions:
Mucopolysaccharidosis I
Hurler's Syndrome
Eligibility:
All Genders
Phase:
PHASE4
Brief Summary
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).
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
December 1 2004
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
January 1 2006
Estimated Enrollment :
34 Patients enrolled
Trial Details
Trial ID
NCT00144781
Start Date
December 1 2004
End Date
January 1 2006
Last Update
April 3 2015
Active Locations (5)
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1
Hospital Infantil Joana de Gusmao
Florianópolis, Santa Catarina, Brazil, CEP 88025-601
2
Universidade Federal de Minas Gerais
Belo Horizonte, Brazil, CEP 30130-100
3
Hospital de Clinical de Porto Alegre
Porto Alegre, Brazil, CEP 90035-003
4
Universidade Federal de Sao Paulo
São Paulo, Brazil, CEP 04023-062