Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease
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.
|Mucopolysaccharidosis I Hurler Syndrome Hurler-Scheie Syndrome Scheie Syndrome||Biological: Aldurazyme (Recombinant Human Alpha-L-Iduronidase)||Phase 3|
|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|
- 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.
- 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.
|Study Start Date:||December 2005|
|Study Completion Date:||October 2006|
|Primary Completion Date:||October 2006 (Final data collection date for primary outcome measure)|
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
Please refer to this study by its ClinicalTrials.gov identifier: NCT00258011
|Osaka City University Hospital|
|Osaka, Japan, 545-8586|
|National Center for Child Health and Development|
|Tokyo, Japan, 157-8535|
|Study Director:||Shigetoyo Oguri||Corp. GCP Compliance - Clinical Affairs, Genzyme Japan K.K.|