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

Study Status: Completed
Recruit Status: Completed
Study Type: Interventional

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

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 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.


Detailed Summary:
Sponsor: Genzyme, a Sanofi Company

Current Primary Outcome: 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.


Original Primary Outcome: Safety assessment of JC0498 (laronidase)

Current Secondary Outcome: 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.


Original Secondary Outcome:

  • Pharmacodynamic assessment of JC0498 (laronidase) with urinary glycosaminoglycan(GAG) excretion.
  • Tertiary objective: Efficacy assessment of JC0498 (laronidase).


Information By: Sanofi

Dates:
Date Received: November 22, 2005
Date Started: December 2005
Date Completion:
Last Updated: February 4, 2014
Last Verified: February 2014