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Protocol No. 2013LS104 Principal Investigator Orchard, Paul
Phase Phase II
Age Group Both Scope Local
Secondary Protocol No. MT2013-31
Title MT2013-31:Allogeneic Hematopoietic Cell Transplantation for Inherited Metabolic Disorders and Severe Osteopetrosis following Conditioning with Busulfan (Therapeutic Drug Monitoring), Fludarabine +/- ATG
Objective To evaluate the ability to achieve high-level donor hematopoietic engraftment (defined as neutrophil recovery by Day +42 post-transplant and ≥ 80% donor cells on the myeloid fraction of peripheral blood at Day +100 post-transplant) using related and unrelated BM, PBSC, or UCB grafts following a reduced-intensity conditioning regimen consisting of targeted-exposure busulfan, fludarabine +/- rabbit ATG in patients with inherited metabolic disorders, osteopetrosis and males with Rett syndrome.
Key Eligibility 0 through 55 years of age Adequate graft available Adequate organ function
Applicable Disease Sites Non Cancer
Status Open
Participating Institutions Masonic Cancer Center
Treatment Type Non-Cancer Therapeutic
Contact Paul Orchard Phone:612-626-2313
Timothy Krepski Phone:612-273-2800
Kim Nelson Phone: