Clinical trial NCT00144781: A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease
Official title: A Multicenter, Multinational, Randomized, Dose-Optimization Study of the Safety and Pharmacodynamic Response of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE4
Conditions: Mucopolysaccharidosis I
Hurler's Syndrome
Hurler-Scheie Syndrome
Scheie Syndrome
Lead sponsor: Sanofi
Sponsor class: INDUSTRY
Collaborators: name: BioMarin/Genzyme LLC
First posted: 2005-09-05
Enrollment: 34
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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 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.

Primary outcomes:
measure: Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Level
timeFrame: Baseline to 26 Weeks
Secondary outcomes:
measure: Percent Change From Baseline to Week 26 in Liver Organ Volume
timeFrame: Baseline to 26 Weeks
measure: Change From Baseline to Week 26 in Six Minute Walk Test (6MWT)
timeFrame: Baseline to 26 Weeks
Other outcomes:
Locations:
city: Florianopolis
country: Brazil
facility: Hospital Infantil Joana de Gusmao
state: Santa Catarina
city: Belo Horizonte
country: Brazil
facility: Universidade Federal de Minas Gerais
city: Porto Alegre
country: Brazil
facility: Hospital de Clinical de Porto Alegre
city: San Paulo
country: Brazil
facility: Universidade Federal de Sao Paulo
city: Toronto
country: Canada
facility: Division of Clinical and Metabolic Genetics
state: Ontario
Study officials:
affiliation: Genzyme, a Sanofi Company
name: Medical Information
role: STUDY_DIRECTOR
References: