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

University of Minnesota (UMN)

Status: Terminated

Conditions: MPS I; Mucopolysaccharidosis Type IH; Hurler Syndrome

Treatments: Drug: Laronidase

Study type: Observational

Funder types: Other

Identifiers

NCT01572636

2011OC140

MT2011-21C (Other Identifier)

Details and patient eligibility

About

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.

Full 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.

Enrollment: 20 patients

Sex: All

Volunteers: No Healthy Volunteers

Inclusion criteria

• Diagnosis of mucopolysaccharidosis type IH (MPS I, Hurler syndrome) and being considered as a candidate for first transplant according to a University of Minnesota myeloablative hematopoietic stem cell transplant (HSCT) protocol

Exclusion criteria

• No prior therapy with laronidase enzyme replacement therapy (ERT)

Trial design

20 participants in 1 patient group

Laronidase use in Hurler Syndrome

Description:

Laronidase receiving prior and post transplant

Treatment:

Drug: Laronidase