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Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome

Hurler Syndrome Clinical Trial

Official title:
MT2011-21C Laronidase (Aldurazyme TM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH).

Clinical Trial Summary

This is a standard of care treatment guideline for patients with the diagnosis of mucopolysaccharidosis type IH (MPS I, Hurler syndrome) who are being considered as candidates for first hematopoietic stem cell transplantation (HSCT) according to a University of Minnesota myeloablative HSCT protocol.

Clinical Trial Description

Laronidase Enzyme Replacement Therapy will be performed using laronidase once a week for 12 weeks prior to hematopoietic stem cell transplantation and for 8 weeks post-transplant to reduce pulmonary complications. ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT01572636
Study type Observational
Source Masonic Cancer Center, University of Minnesota
Contact
Status Terminated
Phase
Start date March 28, 2012
Completion date May 1, 2018
View Details
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
Completed NCT01173016 - Administration of IV Laronidase Post Bone Marrow Transplant in Hurler Phase 1
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Withdrawn NCT00286689 - Effects of Growth Hormone in Chronically Ill Children N/A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