Status:
COMPLETED
Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I
Lead Sponsor:
Genzyme, a Sanofi Company
Collaborating Sponsors:
BioMarin/Genzyme LLC
Conditions:
Mucopolysaccharidosis I
Hurlers Syndrome
Eligibility:
All Genders
5+ years
Phase:
PHASE3
Brief Summary
This study is being conducted to demonstrate the safety and clinical efficacy of Aldurazyme treatment in MPS I patients
Eligibility Criteria
Inclusion
- The patient had a documented diagnosis of MPS I confirmed by measurable clinical signs and symptoms of MPS I and a fibroblast or leukocyte alpha-L-iduronidase enzyme activity level of less than 10% of the lower limit of the normal range of the measuring laboratory.
- Female patients of childbearing potential had a negative pregnancy test (urine-beta-human chorionic gonadotropin (hCG)) at baseline (all female patients of childbearing potential and sexually mature male patients were advised to use a medically accepted method of contraception throughout the study).
- The patient was capable of standing independently for 6 minutes and walking a minimum of 5 meters within 6 minutes.
- The patient was capable of performing a reproducible FVC maneuver.
- The patient had a baseline FVC value that was less than or equal to 80% of the patient's predicted normal FVC value based on polgar predicted values for standing height for children 5 through 7 years of age and the Hankinson predicted values for ages 8 and above.
Exclusion
- The patient had undergone a tracheostomy.
- The patient had previously undergone a bone marrow transplantation.
- The patient was pregnant or lactating.
- The patient has received an investigational drug within 30 days prior to study enrollment.
- The patient had a medical condition, serious intercurrent illness, or other extenuating circumstance that could have significantly interfered with study compliance including all prescribing evaluations and follow-up activities.
- The patient had a known hypersensitivity to rhIDU or to components of the active or placebo test solutions.
Key Trial Info
Start Date :
December 1 2000
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
September 1 2001
Estimated Enrollment :
45 Patients enrolled
Trial Details
Trial ID
NCT00912925
Start Date
December 1 2000
End Date
September 1 2001
Last Update
April 7 2015
Active Locations (4)
Enter a location and click search to find clinical trials sorted by distance.
1
New York, New York, United States
2
Chapel Hill, North Carolina, United States
3
Vancouver, British Columbia, Canada
4
Mainz, Germany