MT2011-21C Laronidase (Aldurazyme TM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH).
N/A
N/A
N/A
N/A
N/A
Open (Enrolling)
Both
Mucopolysaccharidosis Type IH, MPS I, Hurler Syndrome
Both
Mucopolysaccharidosis Type IH, MPS I, Hurler Syndrome
Trial Information
MT2011-21C Laronidase (Aldurazyme TM) Enzyme Replacement Therapy (ERT) With Hematopoietic Stem Cell Transplantation (HSCT) for Hurler Syndrome (MPS IH).
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)
Type of Study:
Observational
Study Design:
Observational Model: Cohort, Time Perspective: Prospective
Outcome Measure:
Overall Survival
Outcome Description:
Patients alive at 1 year post transplantation.
Outcome Time Frame:
At 1 Year
Safety Issue:
No
Principal Investigator
Paul Orchard, M.D.
Investigator Role:
Principal Investigator
Investigator Affiliation:
Masonic Cancer Center, University of Minnesota
Authority:
United States: Institutional Review Board
Study ID:
2011OC140
NCT ID:
NCT01572636
Start Date:
April 2012
Completion Date:
April 2022
Related Keywords:
- Mucopolysaccharidosis Type IH
- MPS I
- Hurler Syndrome
- Hurler Syndrome
- Mucopolysaccharidoses
- Mucopolysaccharidosis I
Name | Location |
|---|---|
| Masonic Cancer Center, University of Minnesota | Minneapolis, Minnesota 55455 |
