Status:
COMPLETED
Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease
Lead Sponsor:
Genzyme, a Sanofi Company
Collaborating Sponsors:
BioMarin/Genzyme LLC
Conditions:
Mucopolysaccharidosis I
Hurler Syndrome
Eligibility:
All Genders
Phase:
PHASE3
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 evaluati...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
December 1 2005
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 1 2006
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT00258011
Start Date
December 1 2005
End Date
October 1 2006
Last Update
March 7 2014
Active Locations (2)
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1
Osaka City University Hospital
Osaka, Japan, 545-8586
2
National Center for Child Health and Development
Tokyo, Japan, 157-8535