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

Information source: Sanofi
ClinicalTrials.gov processed this data on August 20, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Mucopolysaccharidosis I; Hurlers Syndrome; Hurler-Scheie Syndrome

Intervention: rhIDU (recombinant human-Alpha-L-Iduronidase) (Biological); Placebo (Biological)

Phase: Phase 3

Status: Completed

Sponsored by: Genzyme, a Sanofi Company

Official(s) and/or principal investigator(s):
Medical Monitor, Study Director, Affiliation: Genzyme, a Sanofi Company

Summary

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

Clinical Details

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

Study design: Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Primary outcome:

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

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

Secondary outcome:

Overall Change From Baseline to Week 26 in Apnea/Hypopnea Index (AHI)

Overall Percent Change From Baseline to Week 26 in Liver Volume

Overall Change From Baseline to Week 26 in Child Health Assessment Questionnaire/Health Assessment Questionnaire (CHAQ/HAQ) Disability Index Score

Overall Change From Baseline to Week 26 in Active Joint Range of Motion (ROM)

Overall Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Levels

Eligibility

Minimum age: 5 Years. Maximum age: N/A. Gender(s): Both.

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.

Locations and Contacts

Mainz, Germany

Vancouver, British Columbia, Canada

New York, New York, United States

Chapel Hill, North Carolina, United States

Additional Information

Starting date: December 2000
Last updated: March 19, 2015

Page last updated: August 20, 2015

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