Hurler Syndrome Clinical Trial
Official title:
Pilot Study of Administration of Intravenous Laronidase Following Allogeneic Transplantation for Hurler Syndrome
This is a single center pilot study in which Laronidase will be given weekly for two years in patients with Hurler syndrome, also known as mucopolysaccharide IH (MPS I, Hurler syndrome), that have previously been treated with an allogeneic transplant.
This 2-year open-label pilot study of laronidase includes patients (age 5-13 years) who are
at least 2 years post-hematopoietic cell transplantation (HCT) and donor engrafted. Outcomes
are assessed semi-annually and compared to historic controls. Eligible patients will receive
Laronidase as an infusion over several hours once a week at a local site. The dosing of
enzyme will be the standard doses recommended by Genzyme.
The findings of this Pilot Study will be used to assess whether a subsequent larger study can
be conducted.
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Status | Clinical Trial | Phase | |
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