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

Genzyme

Status and phase: Completed; Phase 3

Conditions: Hurler-Scheie Syndrome; Mucopolysaccharidosis I; Hurlers Syndrome

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

Study type: Interventional

Funder types: Industry

Identifiers

NCT00912925

ALID-003-99

Details and patient eligibility

About

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

Enrollment: 45 patients

Sex: All

Ages: 5+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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45 participants in 2 patient groups, including a placebo group

Placebo

Placebo Comparator group

Description:

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.

Treatment:

Biological: Placebo

Aldurazyme treatment

Active Comparator group

Description:

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.

Treatment:

Biological: rhIDU (recombinant human-Alpha-L-Iduronidase)