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

Mucopolysaccharidosis I Clinical Trial

Official title:

A Safety Confirmatory Study of JC0498 (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT00258011
• Other study ID #: ALID02205
• Status: Completed
• Phase: Phase 3
• First received: November 22, 2005
• Last updated: February 4, 2014
• Start date: December 2005
• Est. completion date: October 2006

Study information

• Verified date: February 2014
• Source: Sanofi
• Contact: n/a
• Is FDA regulated: No
• Health authority: Japan: Ministry of Health, Labor and Welfare
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 3
• Est. completion date: October 2006
• Est. primary completion date: October 2006
• Accepts healthy volunteers: No
• Gender: Both
• Age group: N/A and older

Eligibility

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.

Study Design

Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Hurler Syndrome
• Hurler-Scheie Syndrome
• Mucopolysaccharidoses
• Mucopolysaccharidosis I
• Scheie Syndrome
• Syndrome

Intervention

Biological:
• Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
0.58 mg/kg every week

Locations

Country	Name	City	State
Japan	Osaka City University Hospital	Osaka
Japan	National Center for Child Health and Development	Tokyo

Sponsors (2)

Lead Sponsor	Collaborator
Genzyme, a Sanofi Company	BioMarin/Genzyme LLC

Country where clinical trial is conducted

Japan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Safety Evaluation	Overall Safety Summary of Adverse Events (AEs) during Treatment Safety assessment was based on the incidence of AE reports.	Up to 73 Weeks	Yes
Secondary	Urinary Glycosaminoglycan (GAG) Excretion	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.	Up to 73 Weeks	No