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Showing 1-1 of 1 trials for Severe-osteopetrosis
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MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis

Minnesota · Minneapolis, MN

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).