CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 20 enrolled
Drug / intervention
Aldurazyme (Recombinant Human Alpha-L-Iduronidase) +1 morebiological
Likely dose
Aldurazyme 100 U/kg intravenously every week, escalating to 200 U/kg every week from week 26 onwardsAI-extracted
Key inclusion· 4
  • ✓Age less than 5 years at enrollment
  • ✓Confirmed iduronidase deficiency: fibroblast or leukocyte alpha-L-iduronidase enzyme activity <10.0% of lower limit of normal range or below detection limit
  • ✓Clinical diagnosis of MPS I confirmed by genotyping
  • ✓Parent(s) or legal guardian(s) must have received counseling/consultation regarding HSCT risks and benefits before enrollment
Key exclusion· 5
  • ✕Patient is under consideration for or has undergone hematopoietic stem cell transplantation (HSCT)
  • ✕Acute hydrocephalus at time of enrollment
  • ✕Clinically significant organic disease (excluding MPS I-related symptoms): cardiovascular, hepatic, pulmonary, neurologic, or renal disease, serious intercurrent illness, or extenuating circumstances that would preclude participation or decrease survival
  • ✕Receipt of any investigational product within 30 days prior to enrollment

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

Search/NCT00146757
NCT00146757Phase 2Completed
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A Phase II Open-Label Clinical Trial of Recombinant Human Alpha-L-iduronidase (Aldurazyme®) to Evaluate the Safety and Pharmacokinetics in Mucopolysaccharidosis I (MPS I) Patients Less Than 5 Years Old

Genzyme, a Sanofi Company·interventional·Posted Sep 7, 2005·Updated Apr 3, 2015

In Brief

A Phase 2 clinical trial evaluating Aldurazyme (Recombinant Human Alpha-L-Iduronidase) for Mucopolysaccharidosis I and 3 related conditions. Completed, enrolled 20 participants across 4 sites in 4 countries.

Detailed 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 mucopolysaccharidosis I (MPS I) patients less than 5 years old. Efficacy measurements will also be evaluated in this study.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsMucopolysaccharidosis I, Hurler Syndrome, Hurler-Scheie Syndrome, Scheie Syndrome
CountriesFrance, Germany, Netherlands, United Kingdom
CollaboratorsBioMarin/Genzyme LLC

Timeline

Phase 2CompletedFinished
2003200420052006200720082009201020112012201320142015201620172018201920202021202220232024202520262027
First PostedSep 7, 2005
Enrollment StartOct 1, 2002
Primary CompletionMay 1, 2005
TodayJul 2, 2026
Enrollment to primary: 2.6 yearsPosted 20.8 years ago

Interventions

Aldurazyme (Recombinant Human Alpha-L-Iduronidase)biological

100 U/kg every week

Aldurazyme (Recombinant Human Alpha-L-Iduronidase)biological

200 U/kg every week (Week 26 onwards)