NCT00258011
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.
This is a multi-center, open label, study conducted to evaluate the safety of laronidase administered by intravenous drip infusion in Japanese patients with MPS I disease.
Following baseline evaluation, patients will receive weekly infusions of JC0498 at an intravenous dose of 100 units/kg. Patient safety will be monitored continuously throughout the trial. In addition, the effects of JC0498 treatment in this patient population will be assessed by periodically evaluating aspects of MPS I disease in patients at scheduled intervals over the duration of the trial.
Since patients may be eligible for the trial if they have received JC0498, a portion of the data may be captured retrospectively and recorded onto the case report forms (CRFs).
This study represents the first good clinical practice (GCP) effort to characterize MPS I in the Japanese population and evaluate the effects of JC0498 on disease manifestations.
All
Any
No
Aldurazyme (Recombinant Human Alpha-L-Iduronidase)
Phase 3
Interventional
3
2005-12
2014-03-07
Osaka, , Japan
Tokyo, , Japan
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