Status:
COMPLETED
Administration of IV Laronidase Post Bone Marrow Transplant in Hurler
Lead Sponsor:
Masonic Cancer Center, University of Minnesota
Conditions:
Hurler Syndrome
Eligibility:
All Genders
Up to 14 years
Phase:
PHASE1
Brief Summary
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 pre...
Detailed Description
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 se...
Eligibility Criteria
Inclusion
- 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
Exclusion
- 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
Key Trial Info
Start Date :
May 29 2012
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
March 4 2016
Estimated Enrollment :
11 Patients enrolled
Trial Details
Trial ID
NCT01173016
Start Date
May 29 2012
End Date
March 4 2016
Last Update
March 20 2020
Active Locations (1)
Enter a location and click search to find clinical trials sorted by distance.
1
Masonic Cancer Center, University of Minnesota
Minneapolis, Minnesota, United States, 55455