MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis
Status:
Recruiting
Trial end date:
2022-12-01
Target enrollment:
Participant gender:
Summary
This single-institution, phase II study is designed to test the ability to achieve donor
hematopoietic engraftment while maintaining low rates of transplant-related mortality (TRM)
using busulfan- and fludarabine-based conditioning regimens with busulfan therapeutic drug
monitoring (TDM) for patients with various inherited metabolic disorders (IMD) and severe
osteopetrosis (OP).