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

Genzyme

Status and phase: Completed; Phase 3

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

Treatments: Biological: placebo; Biological: Aldurazyme

Study type: Interventional

Funder types: Industry

Identifiers

NCT00146770

ALID-006-01

Details and patient eligibility

About

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.

Enrollment: 45 patients

Sex: All

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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45 participants in 2 patient groups

Placebo/Aldurazyme

Active Comparator group

Description:

Patients received placebo for 26 weeks in the Double-Blind Study then received 182 weeks of Aldurazyme (0.58 mg/kg every week) in this Extension Study; patients received a total of 182 weeks of Aldurazyme.

Treatment:

Biological: Aldurazyme

Biological: placebo

Biological: Aldurazyme

Aldurazyme/Aldurazyme

Active Comparator group

Description:

Patients received 26 weeks of Aldurazyme in the Double-Blind Study and then received 182 weeks of Aldurazyme in this Extension Study; patients received a total of 208 weeks of Aldurazyme.

Treatment:

Biological: Aldurazyme

Biological: Aldurazyme