Saves the questions and what to expect into your notes, next to the visit they belong to.
Keep this study
Lead
Masonic Cancer Center, University of Minnesota
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.
Age
0–14
Sex
ALL
Healthy volunteers
Not accepted
You may be eligible if
Mucopolysaccharidosis type IH (MPS I, Hurler syndrome) treated with a prior allogeneic transplant \>2 years previously
Age \<14 years old
\>10% engrafted based on recent testing (\<4 months prior to enrollment)
Willing to commit to traveling to the University of Minnesota every 6 months
Written informed consent prior to the performance of any study related procedures
You may not be if
Previous administration of Laronidase enzyme \> 3 months post transplantation
Anticipated survival less than 2 years
History of cardiac or pulmonary insufficiency, including an ejection fraction (EF) \< 40% or those requiring continuous supplemental oxygen
Clareo Health | Administration of IV Laronidase Post Bone Marrow Transplant in Hurler
