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Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease

This study has been completed.
BioMarin/Genzyme LLC
Information provided by:
Sanofi Identifier:
First received: November 22, 2005
Last updated: February 4, 2014
Last verified: February 2014

This is a multi-center, open label, study conducted to evaluate the safety of laronidase administered by intravenous drip infusion in Japanese patients with MPS I disease.

Following baseline evaluation, patients will receive weekly infusions of JC0498 at an intravenous dose of 100 units/kg. Patient safety will be monitored continuously throughout the trial. In addition, the effects of JC0498 treatment in this patient population will be assessed by periodically evaluating aspects of MPS I disease in patients at scheduled intervals over the duration of the trial.

Since patients may be eligible for the trial if they have received JC0498, a portion of the data may be captured retrospectively and recorded onto the case report forms (CRFs).

This study represents the first good clinical practice (GCP) effort to characterize MPS I in the Japanese population and evaluate the effects of JC0498 on disease manifestations.

Condition Intervention Phase
Mucopolysaccharidosis I
Hurler Syndrome
Hurler-Scheie Syndrome
Scheie Syndrome
Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
Phase 3

Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Safety Confirmatory Study of JC0498 (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients

Resource links provided by NLM:

Further study details as provided by Sanofi:

Primary Outcome Measures:
  • Safety Evaluation [ Time Frame: Up to 73 Weeks ]
    Overall Safety Summary of Adverse Events (AEs) during Treatment Safety assessment was based on the incidence of AE reports.

Secondary Outcome Measures:
  • Urinary Glycosaminoglycan (GAG) Excretion [ Time Frame: Up to 73 Weeks ]
    Percentage change in the concentration of GAG relative to creatinine in urine (ug GAG/mg creatinine) from baseline to last study visit. Greater decrease indicates greater response.

Enrollment: 3
Study Start Date: December 2005
Study Completion Date: October 2006
Primary Completion Date: October 2006 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Aldurazyme (laronidase) treatment
Patients received weekly infusions of JC0498 (laronidase) at an intravenous dose of 100 Units/kg (0.58 mg/kg) body weight for up to 73 weeks.
Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
0.58 mg/kg every week


Ages Eligible for Study:   Child, Adult, Senior
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Written informed consent/assent of the patient or written informed consent of the parent(s) or the legal guardian(s), depending on the age of the patient, is required prior to any protocol-related procedures being performed; this includes information regarding hematopoietic stem cell transplantation (HSCT) in order to assure that the guardian(s) is fully informed regarding the risks and benefits of this alternative treatment for patients eligible for the trial and who have severe manifestations of MPS I with neurodegeneration.
  • Have a clinical diagnosis of MPS, confirmed by measurable clinical signs and symptoms of MPS I.
  • Have confirmed iduronidase deficiency with a leukocyte alpha-L-iduronidase enzyme activity level of less than 10.0% of the lower limit of the normal range of the measuring laboratory (SRL)

Exclusion Criteria:

  • The patient is under consideration for or has previously undergone hematopoietic stem cell transplantation.
  • 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 (exception: JC0498).
  • The patient has known severe hypersensitivity to JC0498 or components of the delivery solution.
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Please refer to this study by its identifier: NCT00258011

Osaka City University Hospital
Osaka, Japan, 545-8586
National Center for Child Health and Development
Tokyo, Japan, 157-8535
Sponsors and Collaborators
Genzyme, a Sanofi Company
BioMarin/Genzyme LLC
Study Director: Shigetoyo Oguri Corp. GCP Compliance - Clinical Affairs, Genzyme Japan K.K.
  More Information

Responsible Party: Medical Monitor, Genzyme Corporation Identifier: NCT00258011     History of Changes
Other Study ID Numbers: ALID02205
Study First Received: November 22, 2005
Results First Received: January 13, 2009
Last Updated: February 4, 2014

Keywords provided by Sanofi:
MPS I Disease

Additional relevant MeSH terms:
Mucopolysaccharidosis I
Pathologic Processes
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Connective Tissue Diseases
Metabolic Diseases processed this record on April 28, 2017