CI

At a glance

ClinicalIndex Comparison Record
Phase 2Completed· 25 enrolled
Drug / intervention
Laronidase ERT +1 moredrug
Likely dose
Laronidase ERT administered 10-12 weeks prior to HSCT and 8 weeks following HSCTAI-extracted
Key inclusion· 2
  • ✓Diagnosis of mucopolysaccharidosis type IH (MPS I, Hurler syndrome)
  • ✓Candidate for first hematopoietic stem cell transplant (HSCT) according to University of Minnesota myeloablative HSCT protocol
Key exclusion· 2
  • ✕Previous administration of laronidase enzyme
  • ✕Second or subsequent HSCT

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

Search/NCT00176891
NCT00176891Phase 2Completed
CompareCT.gov

Phase II Study of Combined Laronidase (AldurazymeTM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH)

Masonic Cancer Center, University of Minnesota·interventional·Posted Sep 15, 2005·Updated Jul 11, 2019

In Brief

A Phase 2 clinical trial evaluating Stem Cell Transplant and Laronidase ERT for Mucopolysaccharidosis I and Hurler Syndrome. Completed, enrolled 25 participants across 1 site.

Detailed Summary

The investigators hypothesize that weekly infusions of Laronidase ERT for 10-12 weeks prior to transplant and 8 weeks following transplant will result in a reduction of glycosaminoglycans (GAG) burden that is associated with decreased complications following transplant.

Study Details

Study Typeinterventional
Allocation--
Masking--
Primary Purpose--
ConditionsMucopolysaccharidosis I, Hurler Syndrome
CountriesUnited States
Collaborators--

Timeline

Phase 2CompletedFinished
200420052006200720082009201020112012201320142015201620172018201920202021202220232024202520262027
First PostedSep 15, 2005
Enrollment StartMar 1, 2004
Primary CompletionNov 1, 2012
TodayJul 2, 2026
Enrollment to primary: 8.7 yearsPosted 20.8 years ago

Interventions

Stem Cell Transplantprocedure

enzyme replacement 10-12 weeks prior to hematopoietic stem cell transplant (HSCT), and 8 weeks following

Laronidase ERTdrug

Laronidase ERT will be administered 10-12 weeks prior to HSCT, and 8 weeks following.