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A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old

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Trial Status Completed

Trial Identifier

NCT00146757

Condition

MPS 1, Mucopolysaccharidosis I, Hurler-Scheie Syndrome, Scheie Syndrome, Hurler Syndrome

The below information is taken directly from public registry websites such as ClinicalTrials.gov, EuClinicalTrials.eu, ISRCTN.com, etc. and has not been edited.

Trial 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

Birth Sex

All

Age

Up to 5 Years

Healthy Volunteers

No

Drug/Treatment:

Aldurazyme (Recombinant Human Alpha-L-Iduronidase), Aldurazyme (Recombinant Human Alpha-L-Iduronidase)

Phase:

Phase 2

Study Type:

Interventional

Number of Participants:

20

Study Started:

2002-10

Study Updated:

2015-04-03

Trial Locations

  • Hôpital E. Herriot

    Lyon, , France

  • Johannes Gutenberg Universität

    Kinderklinik, Mainz, Germany

  • Sophia Children's Hospital

    Rotterdam, , Netherlands

  • Willink Biochemical Genetics Unit Royal Hospital for Children

    Manchester, , United Kingdom

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

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