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Laronidase ERT with HSCT for Hurler Syndrome
NCT00176891Phase 2Completed
- Sponsor
- Masonic Cancer Center
- Start
- Mar 2004
- Primary completion
- Nov 2012
- Modality
- Replacement Enzymes
- Countries
- 1
01
Overview
Phase 2, open-label, single-arm trial of laronidase in mucopolysaccharidosis type I, sponsored by Masonic Cancer Center. Primary endpoints: Number of Patients Alive at One Year Post Transplant at 1 year and Number of Patients Requiring Ventilator Support at One Year Post Transplant at 1 year. Started Mar 2004; status: completed.
02
Drugs
laronidase
03
Conditions
mucopolysaccharidosis type I
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