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Clinical Trial Summary

This is a single center pilot study in which Laronidase will be given weekly for two years in patients with Hurler syndrome, also known as mucopolysaccharide IH (MPS I, Hurler syndrome), that have previously been treated with an allogeneic transplant.


Clinical Trial Description

This 2-year open-label pilot study of laronidase includes patients (age 5-13 years) who are at least 2 years post-hematopoietic cell transplantation (HCT) and donor engrafted. Outcomes are assessed semi-annually and compared to historic controls. Eligible patients will receive Laronidase as an infusion over several hours once a week at a local site. The dosing of enzyme will be the standard doses recommended by Genzyme.

The findings of this Pilot Study will be used to assess whether a subsequent larger study can be conducted. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT01173016
Study type Interventional
Source Masonic Cancer Center, University of Minnesota
Contact
Status Completed
Phase Phase 1
Start date May 29, 2012
Completion date March 4, 2016

See also
  Status Clinical Trial Phase
Completed NCT00146757 - A Study Evaluating the Safety and Pharmacokinetics of Aldurazyme® (Laronidase) in MPS I Patients Less Than 5 Years Old Phase 2
Terminated NCT01572636 - Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome
Completed NCT00176891 - Stem Cell Transplant w/Laronidase for Hurler Phase 2
Completed NCT03513328 - Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation Phase 1/Phase 2
Recruiting NCT02171104 - MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis Phase 2
Completed NCT01917708 - Bone Marrow Transplant With Abatacept for Non-Malignant Diseases Phase 1
Completed NCT00638547 - Intrathecal Enzyme Replacement for Hurler Syndrome Phase 1
Withdrawn NCT00286689 - Effects of Growth Hormone in Chronically Ill Children N/A
Completed NCT01873911 - Neurobehavioral Phenotypes in MPS III
Completed NCT01043640 - Allogeneic Bone Marrow Transplant for Inherited Metabolic Disorders Phase 2
Completed NCT00258011 - Study of Aldurazyme® Replacement Therapy in Patients With Mucopolysaccharidosis I (MPS I) Disease Phase 3
Active, not recruiting NCT03580083 - RGX-111 Gene Therapy in Patients With MPS I Phase 1/Phase 2
No longer available NCT03639844 - BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study