CI

At a glance

ClinicalIndex Comparison Record
Phase 3Completed· 3 enrolled
Drug / intervention
Aldurazyme (Recombinant Human Alpha-L-Iduronidase)biological
Likely dose
0.58 mg/kg intravenously every weekAI-extracted
Key inclusion· 2
  • ✓Clinical diagnosis of MPS I confirmed by measurable clinical signs and symptoms
  • ✓Confirmed iduronidase deficiency with leukocyte alpha-L-iduronidase enzyme activity <10% of lower limit of normal range
Key exclusion· 5
  • ✕Under consideration for or has previously undergone hematopoietic stem cell transplantation (HSCT)
  • ✕Acute hydrocephalus at time of enrollment
  • ✕Clinically significant organic disease (except MPS I symptoms) including cardiovascular, hepatic, pulmonary, neurologic, or renal disease, or other serious intercurrent illness
  • ✕Receipt of any investigational product within 30 days prior to enrollment (except JC0498)

Standardized by ClinicalIndex from the ClinicalTrials.gov record · verify against the source.

Search/NCT00258011
NCT00258011Phase 3Completed
CompareCT.gov

A Safety Confirmatory Study of JC0498 (Laronidase) in Mucopolysaccharidosis I (MPS I) Patients

Genzyme, a Sanofi Company·interventional·Posted Nov 24, 2005·Updated Mar 7, 2014

In Brief

A Phase 3 clinical trial evaluating Aldurazyme (Recombinant Human Alpha-L-Iduronidase) for Mucopolysaccharidosis I and 3 related conditions. Completed, enrolled 3 participants across 2 sites.

Detailed Summary

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.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsMucopolysaccharidosis I, Hurler Syndrome, Hurler-Scheie Syndrome, Scheie Syndrome
CountriesJapan
CollaboratorsBioMarin/Genzyme LLC

Timeline

Phase 3CompletedFinished
2006200720082009201020112012201320142015201620172018201920202021202220232024202520262027
First PostedNov 24, 2005
Enrollment StartDec 1, 2005
Primary CompletionOct 1, 2006
TodayJul 2, 2026
Enrollment to primary: 10 monthsPosted 20.6 years ago

Interventions

Aldurazyme (Recombinant Human Alpha-L-Iduronidase)biological

0.58 mg/kg every week