Mucopolysaccharidosis I Clinical Trial
Official title:
A Safety Confirmatory Study of JC0498 (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients
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.
n/a
Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
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