Laronidase (Aldurazyme TM) Enzyme Replacement Therapy With Hematopoietic Stem Cell Transplant for Hurler Syndrome
| Status: | Terminated |
|---|---|
| Conditions: | Metabolic |
| Therapuetic Areas: | Pharmacology / Toxicology |
| Healthy: | No |
| Age Range: | Any |
| Updated: | 9/29/2018 |
| Start Date: | March 28, 2012 |
| End Date: | May 1, 2018 |
MT2011-21C Laronidase (Aldurazyme TM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH).
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.
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.
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.
weeks prior to hematopoietic stem cell transplantation and for 8 weeks post-transplant to
reduce pulmonary complications.
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)
We found this trial at
1
site
425 E River Pkwy # 754
Minneapolis, Minnesota 55455
Minneapolis, Minnesota 55455
612-624-2620
Principal Investigator: Paul Orchard, M.D.
Phone: 612-626-2313
Masonic Cancer Center at University of Minnesota The Masonic Cancer Center was founded in 1991....
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