A Study of Treatment of Inflammation Before Stem Cell Transplant in People With a Primary Immune Regulatory Disorder (PIRD) and/or an Autoinflammatory Condition
Start Date
3/15/2023
Completion Date
3/1/2027
Summary
The researchers are doing this study to find out whether emapalumab or a combination of fludarabine and dexamethasone are effective in preparing people with a primary immune regulatory disorder (PIRD) and/or an autoinflammatory condition to receive a stem cell transplant. The researchers will look at how well the study treatments reduce inflammation and aid in the engraftment process (the process of donated stem cells traveling to the bone marrow, where they begin to make new immune cells. "Funding Source - FDA OOPD"
Detailed Description
Defects in the immune cells can hinder the immune system's ability to fight infection. A stem cell transplant can replace non-working immune system cells with a donor's fully functioning immune cells, but if inflammation is present, the immune cells from the donor may not graft successfully. The proposed trial tests whether emapalumab or fludarabine and dexamethasone can help prepare the body to receive a stem cell transplant by reducing inflammation, so that the immune system will be able to produce fully functioning immune cells. Participants will receive emapalumab or a combination of fludarabine and dexamethasone over the course of four days in the hospital prior to a planned transplant procedure. This study has two study groups: 1. Participants in Group A have a high CXCL9, a specific cytokine, level and will receive emapalumab on Days -22, -15, -8, and -1. 2. Participants in Group B have a generalized inflammation and will receive fludarabine and dexamethasone from Days -22 to -18 (5 days). All study participants are eligible to receive one additional emapalumab 3mg/kg in the first 30 days post-transplant if they begin to have CXCL9 levels that are trending up. Participants will receive personalized rATG dosing. Participants will remain in the hospital according to the usual standard of care guidelines for stem cell transplant. Subject status and follow-up examination/data collection will occur on days 0, 7, 14, 21,30, 45, 60, 7, 100, 180, 270, and 365, then quarterly until 3 years post-transplant.
Eligibility Criteria
Age Range: No minimum to No maximum
Interventions
Emapalumab
Fludarabine and Dexamethasone
Allogeneic hematopoietic stem cell transplant (allo-HCT)
Conditions
Locations
University of California, San Francisco
San Francisco, California 94143
United States
Children's Healthcare of Atlanta
Atlanta, Georgia 30322
United States
Memorial Sloan Kettering Basking Ridge
Basking Ridge, New Jersey 07920
United States
Memorial Sloan Kettering Monmouth
Middletown, New Jersey 07748
United States
Memorial Sloan Kettering Bergen
Montvale, New Jersey 07645
United States
Memorial Sloan Kettering Suffolk - Commack
Commack, New York 11725
United States
Memorial Sloan Kettering Westchester
Harrison, New York 10604
United States
Columbia University Irving Medical Center
New York, New York 10032
United States
Memorial Sloan Kettering Cancer Center
New York, New York 10065
United States
Memorial Sloan Kettering Nassau
Rockville Centre, New York 11553
United States
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania 19104
United States
Texas Children's Hospital
Houston, Texas 77030
United States
Children's Hospital of Wisconsin
Milwaukee, Wisconsin 53226
United States