Status:
COMPLETED
A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old
Lead Sponsor:
Genzyme, a Sanofi Company
Collaborating Sponsors:
BioMarin/Genzyme LLC
Conditions:
Mucopolysaccharidosis I
Hurler Syndrome
Eligibility:
All Genders
Up to 5 years
Phase:
PHASE2
Brief 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 mucopoly...
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
October 1 2002
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
May 1 2005
Estimated Enrollment :
20 Patients enrolled
Trial Details
Trial ID
NCT00146757
Start Date
October 1 2002
End Date
May 1 2005
Last Update
April 3 2015
Active Locations (4)
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1
Hôpital E. Herriot
Lyon, France
2
Johannes Gutenberg Universität
Kinderklinik, Mainz, Germany
3
Sophia Children's Hospital
Rotterdam, Netherlands
4
Willink Biochemical Genetics Unit Royal Hospital for Children
Manchester, United Kingdom