Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease
Mucopolysaccharidosis I, Hurler Syndrome, Hurler-Scheie Syndrome
About this trial
This is an interventional treatment trial for Mucopolysaccharidosis I focused on measuring MPS I Disease
Eligibility Criteria
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.
Sites / Locations
- Osaka City University Hospital
- National Center for Child Health and Development
Arms of the Study
Arm 1
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.