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

NCT ID: NCT00144781

Last Updated: 2015-04-03

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE4
• Total Enrollment: 34 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2004-12-31
• Study Completion Date: 2006-01-31

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).

Conditions

Mucopolysaccharidosis I; Hurler's Syndrome; Hurler-Scheie Syndrome; Scheie Syndrome

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

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.

Group Type: ACTIVE_COMPARATOR

Aldurazyme (Recombinant Human Alpha-L-Iduronidase)

Intervention Type: BIOLOGICAL

0.58 mg/kg every week

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.

Group Type: ACTIVE_COMPARATOR

Aldurazyme (Recombinant Human Alpha-L-Iduronidase)

Intervention Type: BIOLOGICAL

1.2 mg/kg every week

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.

Group Type: ACTIVE_COMPARATOR

Aldurazyme (Recombinant Human Alpha-L-Iduronidase)

Intervention Type: BIOLOGICAL

1.2 mg/kg every other week

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.

Group Type: ACTIVE_COMPARATOR

Aldurazyme (Recombinant Human Alpha-L-Iduronidase)

Intervention Type: BIOLOGICAL

1.8 mg/kg every other week

Interventions

Aldurazyme (Recombinant Human Alpha-L-Iduronidase)

0.58 mg/kg every week

Intervention Type: BIOLOGICAL

Aldurazyme (Recombinant Human Alpha-L-Iduronidase)

1.2 mg/kg every week

Intervention Type: BIOLOGICAL

Aldurazyme (Recombinant Human Alpha-L-Iduronidase)

1.2 mg/kg every other week

Intervention Type: BIOLOGICAL

Aldurazyme (Recombinant Human Alpha-L-Iduronidase)

1.8 mg/kg every other week

Intervention Type: BIOLOGICAL

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.

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

BioMarin/Genzyme LLC

INDUSTRY

Sponsor Role: collaborator

Genzyme, a Sanofi Company

INDUSTRY

Sponsor Role: lead

Responsible Party

Genzyme Corporation

Principal Investigators

Medical Information

Role: STUDY_DIRECTOR

Genzyme, a Sanofi Company

Locations

Hospital Infantil Joana de Gusmao

Florianópolis, Santa Catarina, Brazil

Universidade Federal de Minas Gerais

Belo Horizonte, , Brazil

Hospital de Clinical de Porto Alegre

Porto Alegre, , Brazil

Universidade Federal de Sao Paulo

São Paulo, , Brazil

Division of Clinical and Metabolic Genetics

Toronto, Ontario, Canada

Countries

Brazil; Canada

Other Identifiers

ALID-017-03

Identifier Type: -

Identifier Source: org_study_id