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Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I

NCT00912925 · Status: COMPLETED · Phase: PHASE3 · Type: INTERVENTIONAL · Enrollment: 45

Last updated 2015-04-07

Study results available
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Summary

This study is being conducted to demonstrate the safety and clinical efficacy of Aldurazyme treatment in MPS I patients

Conditions

  • Mucopolysaccharidosis I
  • Hurlers Syndrome
  • Hurler-Scheie Syndrome

Interventions

BIOLOGICAL

rhIDU (recombinant human-Alpha-L-Iduronidase)

Patients in the active treatment group received Aldurazyme intravenously at a dose of 100 units/kg (approximately 0.58mg/kg) administered intravenously over approximately 4 hours once weekly for 26 weeks.

BIOLOGICAL

Placebo

Patients in the Placebo-control group were administered a solution of 100mM sodium phosphate , 150mM sodium chloride, and 0.001% polysorbate-80, adjusted to a pH of 5.8 administered intravenously over a time period of approximately 4 hours once weekly for 26 weeks.

Sponsors & Collaborators

  • BioMarin/Genzyme LLC

    collaborator INDUSTRY

  • Genzyme, a Sanofi Company

    lead INDUSTRY

Principal Investigators

  • Medical Monitor · Genzyme, a Sanofi Company

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
5 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2000-12-31
Primary Completion
2001-09-30
Completion
2001-09-30

Countries

  • United States
  • Canada
  • Germany

Study Locations

More Related Trials

Entities

Drugs

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 NCT00912925 on ClinicalTrials.gov