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"
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Enrollment
39
Emapalumab on Days -22 (22 days before the day of the stem cell transplant), -15, -8, and -1.
Fludarabine and dexamethasone for 5 days in a row on Days -22 through -18.
Participants in both groups will receive their standard-of-care stem cell transplant on Day 0.
University of California, San Francisco (Data collection only)
San Francisco, California, United States
RECRUITINGChildren's Healthcare of Atlanta (Data Collection Only)
Atlanta, Georgia, United States
RECRUITINGMemorial Sloan Kettering Basking Ridge (Limited Protocol Activities)
Basking Ridge, New Jersey, United States
RECRUITINGMemorial Sloan Kettering Monmouth (Limited Protocol Activities)
Middletown, New Jersey, United States
RECRUITINGMemorial Sloan Kettering Bergen (Limited Protocol Activities)
Montvale, New Jersey, United States
RECRUITINGMemorial Sloan Kettering Suffolk - Commack (Limited Protocol Activities)
Commack, New York, United States
RECRUITINGMemorial Sloan Kettering Westchester (Limited Protocol Activities)
Harrison, New York, United States
RECRUITINGMemorial Sloan Kettering Cancer Center
New York, New York, United States
RECRUITINGMemorial Sloan Kettering Nassau (Limited Protocol Activities)
Rockville Centre, New York, United States
RECRUITINGChildren's Hospital of Philadelphia (Data Collection Only)
Philadelphia, Pennsylvania, United States
RECRUITING...and 2 more locations
Engraftment
is defined as the first of three days of absolute neutrophil count \>500k/µL and the first of seven days of platelets \>20,000/µL in the absence of transfusional support.
Time frame: 1 year
Determine Overall Survival (OS)
the duration of time between HCT and death due to any cause
Time frame: 5 years
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