Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I - Full Text View

Purpose

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

Condition	Intervention	Phase
Mucopolysaccharidosis I Hurlers Syndrome Hurler Scheie Syndrome Scheie	Biological: rhIDU (recombinant human-Alpha-L-Iduronidase) Biological: Placebo	Phase 3

Study Type:	Interventional
Study Design:	Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment
Official Title:	A Randomized, Double-Blind, Placebo-Controlled, Multicenter, Multinational, Clinical Study of Recombinant Human Alpha L-Iduronidase In Patients With Mucopolysaccharidosis I

Resource links provided by NLM:

Drug Information available for: Laronidase

U.S. FDA Resources

Further study details as provided by Genzyme:

Primary Outcome Measures:

Overall Change From Baseline to Week 26 in Percent Predicted Forced Vital Capacity (FVC) [ Time Frame: Baseline to Week 26 ] [ Designated as safety issue: No ]
Overall Change From Baseline to Week 26 in Six Minute Walk Test (6MWT) [ Time Frame: Baseline to Week 26 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:

Overall Change From Baseline to Week 26 in Apnea/Hypopnea Index (AHI) [ Time Frame: Baseline to Week 26 ] [ Designated as safety issue: No ]
Overall Percent Change From Baseline to Week 26 in Liver Volume [ Time Frame: Baseline to Week 26 ] [ Designated as safety issue: No ]
Overall Change From Baseline to Week 26 in Child Health Assessment Questionnaire/Health Assessment Questionnaire (CHAQ/HAQ) Disability Index Score [ Time Frame: Baseline to week 26 ] [ Designated as safety issue: No ]
Overall Change From Baseline to Week 26 in Active Joint Range of Motion (ROM) [ Time Frame: Baseline to Week 26 ] [ Designated as safety issue: No ]
Overall Percent Change From Baseline to Week 26 in Urinary Glycosaminoglycan (GAG) Levels [ Time Frame: Baseline to Week 26 ] [ Designated as safety issue: No ]

Enrollment:	45
Study Start Date:	December 2000
Study Completion Date:	September 2001
Primary Completion Date:	September 2001 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Placebo Comparator: Placebo Patients in the placebo-control group were administered a solution of 100 millimolar (mM) sodium phosphate, 150 mM sodium chloride, and 0.001% polysorbate-80, adjusted to a pH of 5.8 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.
Active Comparator: Aldurazyme treatment Patients in the active treatment group received Aldurazyme intravenously at a dose of 100 Units/kg (approximately 0.58 mg/kg = labeled dose) administered intravenously over approximately 4 hours once weekly for 26 weeks.	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.

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.

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT00912925

Locations

United States, New York

New York, New York, United States
United States, North Carolina

Chapel Hill, North Carolina, United States
Canada, British Columbia

Vancouver, British Columbia, Canada
Germany

Mainz, Germany

Sponsors and Collaborators

Genzyme

Genzyme-Biomarin LLC

Investigators

Study Director:

Medical Monitor

Genzyme

More Information

No publications provided

Responsible Party:	Medical Monitor, Genzyme Corporation
ClinicalTrials.gov Identifier:	NCT00912925 History of Changes
Other Study ID Numbers:	ALID-003-99
Study First Received:	June 2, 2009
Results First Received:	July 2, 2009
Last Updated:	August 21, 2009
Health Authority:	United States: Food and Drug Administration Canada: Health Canada European Union: European Medicines Agency Germany: Federal Institute for Drugs and Medical Devices

Additional relevant MeSH terms:

Mucopolysaccharidosis I
Mucopolysaccharidoses
Carbohydrate Metabolism, Inborn Errors
Metabolism, Inborn Errors
Genetic Diseases, Inborn

Lysosomal Storage Diseases
Mucinoses
Connective Tissue Diseases
Metabolic Diseases

ClinicalTrials.gov processed this record on May 19, 2013