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Brief Title: Reduced Intensity Conditioning for Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (BMT CTN 1204)
Official Title: Reduced-Intensity Conditioning for Children and Adults With Hemophagocytic Syndromes or Selected Primary Immune Deficiencies (RICHI) (BMT CTN #1204)
Study ID: NCT01998633
Brief Summary: HLH, HLH-related disorders, Chronic Granulomatous (CGD), HIGM1, Immune dysregulation, polyendocrinopathy, enteropathy, and X-linked inheritance (IPEX) and severe LAD-I represent primary immune disorders that are typically fatal without Hematopoietic Cell Transplant (HCT). However, transplant is often complicated by inflammation, infection and other co-morbidities. In addition, these disorders have been shown to be cured with partial chimerism, making them an ideal target for the use of reduced intensity approaches, where a portion of patients may not achieve full donor chimerism, but instead achieve stable mixed chimerism. Reduced-intensity conditioning strategies have demonstrated improved survival with decreased Treatment Related Mortality (TRM) in institutional series for patients with HLH (Cooper et al., 2006; Marsh et al., 2010; Marsh et al., 2011). However, graft loss and unstable chimerism remain challenges. An institutional case series from Cincinnati Children's Hospital demonstrated full or high-level chimerism and improved durable engraftment using intermediate (Day -14) timing alemtuzumab (Marsh et al., 2013b). This study aims to test the efficacy of the Intermediate RIC strategy in a prospective multi-center study including HLH as well as other primary immunodeficiencies where allogeneic transplant with RIC has been shown to be feasible and stable chimerism is curative.
Detailed Description: The primary goal of this Phase II clinical trial is to determine the one-year overall survival of patients treated for immune deficiencies including HLH, HLH-like disorders, CGD, HIGM1, IPEX syndrome, and severe LAD-I with Matched Related Donor (MRD)/ Matched Unrelated Donor (MUD) bone marrow transplant using a reduced-intensity conditioning strategy including intermediate-timing of alemtuzumab. The donor choice is an unaffected related bone marrow donor who is a 6/6 match at HLA-A, -B (intermediate or higher resolution) and -DRB1 (at high resolution using DNA-based typing) OR a 7/8 or 8/8 match for human leukocyte antigen (HLA)-A, -B, -C and -DRB1 (at high resolution using DNA-based typing), OR an unrelated bone marrow donor who is a 7/8 or 8/8 match at HLA-A, -B, -C and -DRB1 (at high resolution using DNA-based typing). The transplant conditioning regimen will include fludarabine, melphalan, and alemtuzumab starting at Day -14 (Flu/Mel/Alem). Graft Versus Host Disease (GVHD) prophylaxis will consist of cyclosporine and corticosteroids through engraftment. Post-transplant supportive care will include infection surveillance and prophylaxis, and disease-specific supportive care.
Minimum Age: 4 Months
Eligible Ages: CHILD, ADULT
Sex: ALL
Healthy Volunteers: No
University of Alabama at Birmingham, Birmingham, Alabama, United States
Children's National Medical Center, Washington, District of Columbia, United States
Children's Healthcare of Atlanta, Atlanta, Georgia, United States
Ann and Robert H. Lurie Children's Hospital of Chicago, Chicago, Illinois, United States
Johns Hopkins, Baltimore, Maryland, United States
Dana Farber Cancer Institute/Children's Hospital of Boston, Boston, Massachusetts, United States
University of Michigan Medical Center, Ann Arbor, Michigan, United States
Washington University/St. Louis Children's Hospital, Saint Louis, Missouri, United States
Memorial Sloan-Kettering Cancer Center, New York, New York, United States
University of Rochester Medical Center, Rochester, New York, United States
University of North Carolina at Chapel Hill, Chapel Hill, North Carolina, United States
Duke University Medical Center, Durham, North Carolina, United States
Cincinnati Children's Hospital Medical Center, Cincinnati, Ohio, United States
Oregon Health and Science University, Portland, Oregon, United States
Children's Medical Center of Dallas, Dallas, Texas, United States
Cook Children's Medical Center, Fort Worth, Texas, United States
Baylor College of Medicine, Houston, Texas, United States
Fred Hutchinson Cancer Research Center, Seattle, Washington, United States
Midwest Children's Cancer, Milwaukee, Wisconsin, United States
British Columbia Children's Hosp-Vancouver, Vancouver, British Columbia, Canada
Hopital Sainte-Justine, Montreal, Quebec, Canada
McGill University - Montreal, Montreal, Quebec, Canada
Name: Mary Horowitz, MD, MS
Affiliation: Center for International Blood and Marrow Transplant Research (CIBMTR), Medical College of Wisconsin
Role: STUDY_DIRECTOR