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.|
|ClinicalTrials.gov Identifier: NCT00258011|
Recruitment Status : Completed
First Posted : November 24, 2005
Results First Posted : February 12, 2009
Last Update Posted : March 7, 2014
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|
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||3 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||A Safety Confirmatory Study of JC0498 (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients|
|Study Start Date :||December 2005|
|Primary Completion Date :||October 2006|
|Study Completion Date :||October 2006|
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
- 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.
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 ClinicalTrials.gov identifier (NCT number): 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.|