MedTrace Logo

A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old

NCT00146757 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 20

Last updated 2015-04-03

Study results available
· View outcomes & findings →

Summary

The main objectives of this study are to evaluate the safety and pharmacokinetics (PK) of enzyme replacement therapy with recombinant human alpha-L-iduronidase \[Aldurazyme® (laronidase)\] in mucopolysaccharidosis I (MPS I) patients less than 5 years old. Efficacy measurements will also be evaluated in this study.

Conditions

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

Interventions

BIOLOGICAL

Aldurazyme (Recombinant Human Alpha-L-Iduronidase)

100 U/kg every week

BIOLOGICAL

Aldurazyme (Recombinant Human Alpha-L-Iduronidase)

200 U/kg every week (Week 26 onwards)

Sponsors & Collaborators

  • BioMarin/Genzyme LLC

    collaborator INDUSTRY

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Medical Monitor · Genzyme, a Sanofi Company

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Max Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2002-10-31
Primary Completion
2005-05-31
Completion
2005-05-31

Countries

  • France
  • Germany
  • Netherlands
  • United Kingdom

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 NCT00146757 on ClinicalTrials.gov