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 EngraftmentIncidence 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
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