A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old

NCT ID: NCT00146757

Last Updated: 2015-04-03

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 20 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2002-10-31
• Study Completion Date: 2005-05-31

Brief Summary

The main objectives of this study are to evaluate the safety and pharmacokinetics (PK) of enzyme replacement therapy with recombinant human alpha-L-iduronidase [Aldurazyme® (laronidase)] in mucopolysaccharidosis I (MPS I) patients less than 5 years old. Efficacy measurements will also be evaluated in this study.

Conditions

Mucopolysaccharidosis I; Hurler Syndrome; Hurler-Scheie Syndrome; Scheie Syndrome

Study Design

• Allocation Method: NON_RANDOMIZED
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Aldurazyme (rhIDU) 100 U/kg ONLY every week

Patients received Aldurazyme (recombinant human alpha-L-iduronidase (rhIDU)) once per week at a dose of 100 Units/kg (approximately 0.58 mg/kg) for up to 52 weeks - labeled dose.

Group Type: EXPERIMENTAL

Aldurazyme (Recombinant Human Alpha-L-Iduronidase)

Intervention Type: BIOLOGICAL

100 U/kg every week

Aldurazyme (rhIDU) 100-200 U/kg every week

After receiving 100 Units/kg dose of Aldurazyme (rhIDU) for the first 25 weeks, patients enrolling after January 1, 2004 were eligible to receive an increased dose of 200 Units/kg from Week 26 onwards if the patient's urinary glycosaminoglycan (uGAG) levels were \>200µg/mg creatinine at Week 22.

Group Type: EXPERIMENTAL

Aldurazyme (Recombinant Human Alpha-L-Iduronidase)

Intervention Type: BIOLOGICAL

200 U/kg every week (Week 26 onwards)

Interventions

Aldurazyme (Recombinant Human Alpha-L-Iduronidase)

100 U/kg every week

Intervention Type: BIOLOGICAL

Aldurazyme (Recombinant Human Alpha-L-Iduronidase)

200 U/kg every week (Week 26 onwards)

Intervention Type: BIOLOGICAL

Eligibility Criteria

Inclusion Criteria

• Written informed consent is required from the parent(s) or legal guardian(s) prior to any protocol-related procedures being performed. (A separate informed consent will be requested from the parent(s) for their genotyping, which is independent of the inclusion.)
• Be less than 5 years of age at the time of enrollment.
• Have confirmed iduronidase deficiency with a fibroblast or leukocyte alpha-L-iduronidase enzyme activity level of less than 10.0 % of the lower limit of the normal range, or below the detection range of the measuring laboratory.
• Have a clinical diagnosis of MPS I based on genotyping.
• Documentation in his/her medical record that the parent(s) or legal guardian(s) have had counseling or a consultation regarding HSCT in order to assure that the parent(s) or legal guardian(s) are fully informed regarding the risks and benefits of this alternative treatment for patients eligible for the trial and with the severe manifestations of MPS I with neurodegeneration.

Exclusion Criteria

• The patient is under consideration for or has undergone hematopoietic stem cell transplantation (HSCT).
• The patient has acute hydrocephalus at the time of enrollment.
• The patient has a clinically significant organic disease (with the exception of symptoms relating to MPS I) including: cardiovascular, hepatic, pulmonary, neurologic, or renal disease, other serious intercurrent illness, or extenuating circumstances that, in the opinion of the Investigator, would preclude participation in the trial or potentially decrease survival.
• The patient has received any investigational product within 30 days prior to trial enrollment.
• The patient has known severe hypersensitivity to Aldurazyme® (laronidase) or components of the delivery solution.

• Maximum Eligible Age: 5 Years
• 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 Monitor

Role: STUDY_DIRECTOR

Genzyme, a Sanofi Company

Locations

Hôpital E. Herriot

Lyon, , France

Johannes Gutenberg Universität

Kinderklinik, Mainz, Germany

Sophia Children's Hospital

Rotterdam, , Netherlands

Willink Biochemical Genetics Unit Royal Hospital for Children

Manchester, , United Kingdom

Countries

France; Germany; Netherlands; United Kingdom

Other Identifiers

ALID-014-02

Identifier Type: -

Identifier Source: org_study_id