Intrathecal Enzyme Replacement for Hurler Syndrome

Status Completed

Clinical Trial Summary

This protocol will examine whether the enzyme alpha-L-iduronidase (Laronidase), delivered into the spinal fluid of patients with Hurler syndrome at intervals before and after bone marrow transplant, is a safe and effective approach to slow the neurologic degeneration seen in Hurler patients undergoing transplantation.

Clinical Trial Description

Subjects will receive an infusion of Laronidase into his/her spinal fluid approximately 12 weeks before, 2 weeks before, 100 days after and 6 months after transplant. This procedure is done by lumbar puncture (also called a "spinal tap"). ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details

NCT number	NCT00638547
Study type	Interventional
Source	Masonic Cancer Center, University of Minnesota
Contact
Status	Completed
Phase	Phase 1
Start date	January 2, 2008
Completion date	November 18, 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
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
Withdrawn	`NCT00286689` - Effects of Growth Hormone in Chronically Ill Children	N/A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