NCT01572636

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

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

• Status: Terminated
• Phase: —
• Study type: Observational
• Enrollment: 20 (actual)
• Sponsor: Masonic Cancer Center, University of Minnesota · Academic / Other
• Sex: All
• Age: —
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Mucopolysaccharidosis Type IH
• MPS I
• Hurler Syndrome

Interventions

Type	Name	Description
DRUG	Laronidase	Administered 0.58 mg/kg/dose intravenously (IV) once a week beginning 12 weeks before planned hematopoietic stem cell transplant (HSCT) and resume same dosing regimen for 8 weeks after HSCT.

Timeline

• Start date: 2012-03-28
• Primary completion: 2018-05-01
• Completion: 2018-05-01
• First posted: 2012-04-06
• Last updated: 2018-09-27

Locations

1 site across 1 country: United States