A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old
Condition: Mucopolysaccharidosis I; Hurler 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)
Intervention description: 100 U/kg every week; 200 U/kg every week (Week 26 onwards)
Phase: Phase 2
Study type: Interventional
Primary purpose: Treatment
Allocation: Non-Randomized
Intervention model: Single Group Assignment
Masking: None (Open Label)
Enrollment: 20.0
Sex: All
Maximum age: 5 Years
Healthy volunteers: No

Summary:

      The main objectives of this study are to evaluate the safety and pharmacokinetics (PK) of
      enzyme replacement therapy with recombinant human alpha-L-iduronidase [Aldurazyme®
      (laronidase)] in mucopolysaccharidosis I (MPS I) patients less than 5 years old. Efficacy
      measurements will also be evaluated in this study.
    

Eligibility criteria:

        Inclusion Criteria:

          -  Written informed consent is required from the parent(s) or legal guardian(s) prior to
             any protocol-related procedures being performed. (A separate informed consent will be
             requested from the parent(s) for their genotyping, which is independent of the
             inclusion.)

          -  Be less than 5 years of age at the time of enrollment.

          -  Have confirmed iduronidase deficiency with a fibroblast or leukocyte
             alpha-L-iduronidase enzyme activity level of less than 10.0 % of the lower limit of
             the normal range, or below the detection range of the measuring laboratory.

          -  Have a clinical diagnosis of MPS I based on genotyping.

          -  Documentation in his/her medical record that the parent(s) or legal guardian(s) have
             had counseling or a consultation regarding HSCT in order to assure that the parent(s)
             or legal guardian(s) are fully informed regarding the risks and benefits of this
             alternative treatment for patients eligible for the trial and with the severe
             manifestations of MPS I with neurodegeneration.

        Exclusion Criteria:

          -  The patient is under consideration for or has undergone hematopoietic stem cell
             transplantation (HSCT).

          -  The patient has acute hydrocephalus at the time of enrollment.

          -  The patient has a clinically significant organic disease (with the exception of
             symptoms relating to MPS I) including: cardiovascular, hepatic, pulmonary, neurologic,
             or renal disease, other serious intercurrent illness, or extenuating circumstances
             that, in the opinion of the Investigator, would preclude participation in the trial or
             potentially decrease survival.

          -  The patient has received any investigational product within 30 days prior to trial
             enrollment.

          -  The patient has known severe hypersensitivity to Aldurazyme® (laronidase) or
             components of the delivery solution.