A Dose-optimization Study of Aldurazyme® (Laronidase) in Patients With Mucopolysaccharidosis I (MPS I) Disease
Condition: Mucopolysaccharidosis I; Hurler's Syndrome; Hurler-Scheie Syndrome; Scheie Syndrome
MeSH terms: Mucopolysaccharidoses; Mucopolysaccharidosis I; Syndrome
Intervention: Aldurazyme (Recombinant Human Alpha-L-Iduronidase); Aldurazyme (Recombinant Human Alpha-L-Iduronidase); Aldurazyme (Recombinant Human Alpha-L-Iduronidase); Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
Intervention description: 0.58 mg/kg every week; 1.2 mg/kg every week; 1.2 mg/kg every other week; 1.8 mg/kg every other week
Phase: Phase 4
Study type: Interventional
Primary purpose: Treatment
Allocation: Randomized
Intervention model: Parallel Assignment
Masking: None (Open Label)
Enrollment: 34.0
Sex: All
Healthy volunteers: No

Summary:

      The main purpose of this study is to evaluate differences in the pharmacodynamic response of
      4 Aldurazyme® (laronidase) dose regimens in patients with Mucopolysaccharidosis I (MPS I).
    

Eligibility criteria:

        Inclusion Criteria:

          -  Have a documented diagnosis of MPS I, confirmed by measurable clinical signs and
             symptoms of MPS I, and a documented fibroblast or leukocyte α-L-iduronidase enzyme
             activity level of less than 10% of the lower limit of the normal range of the
             measuring laboratory.

          -  Weigh at least 12.5 kg.

          -  Have serum creatinine and blood urea nitrogen (BUN) values within age appropriate
             normal ranges.

          -  Provide signed, written informed consent prior to any protocol-related procedures
             being performed. Consent of a legally authorized guardian(s) is (are) required for
             patients under 18 years. If the patient is under 18 years old and can understand the
             consent, written informed consent will be required from both the patient and the
             authorized guardian(s).

        Exclusion Criteria:

          -  Have previously received Aldurazyme® (laronidase).

          -  Have a suspected hypersensitivity to Aldurazyme® (laronidase) or known
             hypersensitivity to components of the infusion solution.

          -  Have previously undergone hematopoietic stem cell transplantation (HSCT; i.e., from
             bone marrow [BMT], peripheral blood, or umbilical cord blood) or other major organ
             transplantation.

          -  Have a medical condition, serious inter-current illness, or other extenuating
             circumstance that may interfere with study compliance including all prescribed
             evaluations and follow-up activities, except the 6MWT. (Note: All patients may not be
             capable of performing the 6MWT due to age and/or maturity level. Exemption from
             performing the 6MWT must be obtained in writing by the investigator from the sponsor's
             medical monitor prior to enrollment).

          -  Have an acute illness that requires surgical intervention, and/or anticipates surgery
             during study participation, and/or has had surgery within 30 days prior to study
             enrollment.

          -  Have received an investigational drug within 30 days prior to study enrollment.

          -  Is pregnant or lactating. Female patients of childbearing potential must have a
             negative pregnancy test [urine β-human chronic gonadotropin (hCG)] at entry (prior to
             the first infusion). Note: All female patients of childbearing potential and sexually
             mature males must be advised to use a medically accepted method of contraception
             throughout the study.