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

NCT00258011 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 3

Last updated 2014-03-07

Study results available
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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

BIOLOGICAL

Aldurazyme (Recombinant Human Alpha-L-Iduronidase)

0.58 mg/kg every week

Sponsors & Collaborators

  • BioMarin/Genzyme LLC

    collaborator INDUSTRY

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Shigetoyo Oguri · Corp. GCP Compliance - Clinical Affairs, Genzyme Japan K.K.

Study Design

Allocation
NA
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2005-12-31
Primary Completion
2006-10-31
Completion
2006-10-31

Countries

  • Japan

Study Locations

More Related Trials

Entities

Diseases

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00258011 on ClinicalTrials.gov