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

Information source: Masonic Cancer Center, University of Minnesota
ClinicalTrials.gov processed this data on August 20, 2015
Link to the current ClinicalTrials.gov record.

Condition(s) targeted: Mucopolysaccharidosis Type IH; MPS I; Hurler Syndrome

Intervention: Laronidase (Drug)

Phase: N/A

Status: Recruiting

Sponsored by: Masonic Cancer Center, University of Minnesota

Official(s) and/or principal investigator(s):
Paul Orchard, M.D., Principal Investigator, Affiliation: Masonic Cancer Center, University of Minnesota

Overall contact:
Paul Orchard, M.D., Phone: 612-626-2313, Email: orcha001@umn.edu

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 Details

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

Study design: Observational Model: Cohort, Time Perspective: Prospective

Primary outcome: Overall Survival

Secondary outcome:

Incidence of Engraftment

Incidence of Grade III-IV Acute Graft Versus Host Disease

Proportion of patients in need of ventilator support

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.

Eligibility

Minimum age: N/A. Maximum age: N/A. Gender(s): Both.

Criteria:

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)

Locations and Contacts

Paul Orchard, M.D., Phone: 612-626-2313, Email: orcha001@umn.edu

Masonic Cancer Center, University of Minnesota, Minneapolis, Minnesota 55455, United States; Recruiting
Paul Orchard, M.D., Phone: 612-626-2313, Email: orcha001@umn.edu
Paul Orchard, M.D., Principal Investigator
Additional Information

Starting date: April 2012
Last updated: April 22, 2015

Page last updated: August 20, 2015

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