NCT00146757
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.
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.
All
Up to 5 Years
No
Aldurazyme (Recombinant Human Alpha-L-Iduronidase), Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
Phase 2
Interventional
20
2002-10
2015-04-03
Lyon, , France
Kinderklinik, Mainz, Germany
Rotterdam, , Netherlands
Manchester, , United Kingdom
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