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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
Open (Enrolling)
Both
Mucopolysaccharidosis Type IH, MPS I, Hurler Syndrome

Thank you

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 MinnesotaMinneapolis, Minnesota  55455