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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00258011
Recruitment Status : Completed
First Posted : November 24, 2005
Results First Posted : February 12, 2009
Last Update Posted : March 7, 2014
BioMarin/Genzyme LLC
Information provided by:

Brief Summary:

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 or disease Intervention/treatment Phase
Mucopolysaccharidosis I Hurler Syndrome Hurler-Scheie Syndrome Scheie Syndrome Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase) Phase 3

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 3 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Safety Confirmatory Study of JC0498 (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients
Study Start Date : December 2005
Actual Primary Completion Date : October 2006
Actual Study Completion Date : October 2006

Arm Intervention/treatment
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

Primary Outcome Measures :
  1. 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 :
  1. 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.

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   Child, Adult, Older Adult
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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00258011

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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
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Study Director: Shigetoyo Oguri Corp. GCP Compliance - Clinical Affairs, Genzyme Japan K.K.
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Responsible Party: Medical Monitor, Genzyme Corporation Identifier: NCT00258011    
Other Study ID Numbers: ALID02205
First Posted: November 24, 2005    Key Record Dates
Results First Posted: February 12, 2009
Last Update Posted: March 7, 2014
Last Verified: February 2014
Keywords provided by Sanofi:
MPS I Disease
Additional relevant MeSH terms:
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Mucopolysaccharidosis I
Pathologic Processes
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lysosomal Storage Diseases
Connective Tissue Diseases
Metabolic Diseases