Clinical Study of Aldurazyme in Patients With Mucopolysaccharidosis (MPS) I
Condition: Mucopolysaccharidosis I; Hurlers Syndrome; Hurler-Scheie Syndrome
MeSH terms: Mucopolysaccharidoses; Mucopolysaccharidosis I; Syndrome
Intervention: rhIDU (recombinant human-Alpha-L-Iduronidase); Placebo
Intervention description: 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.; 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.
Phase: Phase 3
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Enrollment: 45.0
Sex: All
Minimum age: 5 Years
Healthy volunteers: No

Summary:

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

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.