NCT00258011

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

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

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 3 (actual)
• Sponsor: Genzyme, a Sanofi Company · Industry
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

Conditions

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

Interventions

Type	Name	Description
BIOLOGICAL	Aldurazyme (Recombinant Human Alpha-L-Iduronidase)	0.58 mg/kg every week

Timeline

• Start date: 2005-12-01
• Primary completion: 2006-10-01
• Completion: 2006-10-01
• First posted: 2005-11-24
• Last updated: 2014-03-07
• Results posted: 2009-02-12

Locations

2 sites across 1 country: Japan