Phase 3 Extension Study of the Safety and Efficacy of Aldurazyme® (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients

Phase 3

Completed

Conditions: Hurler-Scheie Syndrome
Mucopolysaccharidosis I
Hurler's Syndrome
Scheie Syndrome

Brief Summary: This study is being conducted to collect additional long-term efficacy and safety data of Aldurazyme® (laronidase) patients with MPS I disease. Patients who were previously enrolled in the Phase 3 Double-Blind Study will be enrolled in this study.

Recruitment & Eligibility

Inclusion Criteria

The patient or patient's legal guardian must provide written informed consent prior to any protocol-related procedures being performed.
The patient must have successfully completed Study ALID-003-99 (who received 21 of 26 consecutive weekly infusions).
The patient has not experienced any safety issues that would contraindicate participation in the Extension study.
A female patient of childbearing potential must have a negative pregnancy test at entry

Exclusion Criteria

The patient is pregnant or lactating.
The patient has received an investigational drug within 30 days prior to the study enrollment.
The patient has a medical condition, serious intercurrent illness, or other extenuating circumstance that may significantly interfere with study compliance including all prescribed evaluations and follow-up activities

Primary Outcome Measures

Name	Time	Method
Change From Baseline to Week 182 in Six Minute Walk Test (6MWT)	Baseline to Week 182	Six Minute Walk Test: Distance walked (measured in Meters) in 6 minutes. A longer distance indicates a greater response.
Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC)	Baseline to Week 182	Percent Predicted Forced Vital Capacity: the maximal exhaled breath volume following a maximal inhaled breath. Overall change from Baseline to Week 182 in percent predicted FVC = (observed value)/(predicted value) \* 100%). A higher value indicates a greater response.

Secondary Outcome Measures

Name	Time	Method
Change From Baseline to Week 182 in Liver Volume	Baseline to Week 182	Liver Organ Volume: Volume of liver measured by Magnetic Resonance Imaging (MRI). Greater decrease in volume indicates a greater response.
Change From Baseline to Week 182 in Child Health Assessment Questionnaire/Health Assessment Questionnaire (CHAQ/HAQ) Disability Index Score	Baseline to Week 182	CHAQ/HAQ = Patient questionnaire that measures the degree of disability on a scale of 0 (no disability) to 3 (maximal disability). A lower score indicates a greater response.
Change From Baseline to Week 182 in Active Joint Range of Motion (ROM)	Baseline to Week182	Active Joint Range of Motion (ROM): Shoulder Flexion Ability to maximally raise one's arm overhead without assistance. Shoulder range of motion (mean of left and right arms) measured in degrees (0-180) by goniometry. Greater degree of flexion indicates greater response.
Change From Baseline to Week 182 in Apnea/Hypopnea Index (AHI)	Baseline to Week 182	Apnea/Hypopnea Index (AHI): Number of absent (apnea) and shallow (hypopnea) breaths per hour of sleep. A greater decrease in events indicates a greater response.

Locations (25): University of South Alabama
🇺🇸
Mobile, Alabama, United States
Emory University School of Medicine
🇺🇸
Atlanta, Georgia, United States
University of Rochester
🇺🇸
Rochester, New York, United States
University of North Carolina at Chapel Hill
🇺🇸
Chapel Hill, North Carolina, United States
Toledo Children's Hospital
🇺🇸
Toledo, Ohio, United States
Merle West Medical Center
🇺🇸
Klamath Falls, Oregon, United States
The Childrens Hospital of Philadelphia
🇺🇸
Philadelphia, Pennsylvania, United States
Rhode Island Hospital
🇺🇸
Providence, Rhode Island, United States
Hospital Universatario de Universidade Federal de Santa Catarina
🇧🇷
Florianopolis, Brazil
Hospital Infantil Joana de Gusmao
🇧🇷
Florianopolis, Brazil
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University of South Alabama
🇺🇸Mobile, Alabama, United States