A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Multinational, Clinical Study of Recombinant Human Alpha L-Iduronidase In Patients With Mucopolysaccharidosis I

Genzyme, a Sanofi Company0 sites45 target enrollmentDecember 2000

ConditionsMucopolysaccharidosis I Hurlers Syndrome Hurler-Scheie Syndrome

Overview

Phase: Phase 3
Intervention: Not specified
Conditions: Mucopolysaccharidosis I
Sponsor: Genzyme, a Sanofi Company
Enrollment: 45
Primary Endpoint: Overall Change From Baseline to Week 26 in Six Minute Walk Test (6MWT)
Status: Completed
Last Updated: 11 years ago

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

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

Registry

clinicaltrials.gov

Start Date

December 2000

End Date

September 2001

Last Updated

11 years ago

Study Type

Interventional

Study Design

Parallel

Sex

All

Investigators

Sponsor

Genzyme, a Sanofi Company

Eligibility Criteria

Inclusion Criteria

•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 Criteria

•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.

Outcomes

Primary Outcomes

Overall Change From Baseline to Week 26 in Six Minute Walk Test (6MWT)

Time Frame: Baseline to Week 26

Six Minute Walk Test (6MWT): Distance walked (measured in meters) in 6 minutes. A longer distance indicates a greater response.

Overall Change From Baseline to Week 26 in Percent Predicted Forced Vital Capacity (FVC)

Time Frame: Baseline to Week 26

Percent Predicted Forced Vital Capacity (FVC): the maximal exhaled breathe volume following a maximal inhaled breath. Overall Change from Baseline to Week 26 in percent predicted FVC = (observed value)/(predicted value) \* 100%). A higher value indicates a greater response.

Secondary Outcomes

Overall Change From Baseline to Week 26 in Apnea/Hypopnea Index (AHI)(Baseline to Week 26)
Overall Percent Change From Baseline to Week 26 in Liver Volume(Baseline to Week 26)
Overall Change From Baseline to Week 26 in Child Health Assessment Questionnaire/Health Assessment Questionnaire (CHAQ/HAQ) Disability Index Score(Baseline to week 26)
Overall Change From Baseline to Week 26 in Active Joint Range of Motion (ROM)(Baseline to Week 26)
Overall Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Levels(Baseline to Week 26)