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

Genzyme, a Sanofi Company45 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

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

Interventions

AldurazymeBIOLOGICAL

Placebo for 26 weeks then 0.58 mg/kg Aldurazyme every week for 182 weeks

AldurazymeBIOLOGICAL

0.58 mg/kg Aldurazyme every week for 208 weeks

placeboBIOLOGICAL

Placebo for 26 weeks

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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

Locations (25)

University of South Alabama

Outcomes

Primary Outcomes

Change From Baseline to Week 182 in Percent Predicted Forced Vital Capacity (FVC)

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.