Phase 2 Trial of Emapalumab or Fludarabine and Dexamethasone to Reduce Inflammation Before Stem Cell Transplant in People with Primary Immune Regulatory Disorders or Autoinflammatory Conditions

What this Trial Is About

Researchers are evaluating whether emapalumab or a combination of fludarabine and dexamethasone can effectively prepare people with primary immune regulatory disorders PIRD andor autoinflammatory conditions for stem cell transplants. The study aims to see if these treatments reduce inflammation and help donor stem cells successfully engraft, enabling the immune system to produce fully functioning cells. This is a phase 2 study supported by the FDA Office of Orphan Products Development. Participants are assigned to one of two groups based on their inflammation type. Group A participants with a high CXCL9 cytokine level receive emapalumab on days -22, -15, -8, and -1 before the transplant. Group B participants with generalized inflammation receive fludarabine and dexamethasone for five consecutive days from days -22 to -18. All participants undergo a standard stem cell transplant on day 0 and may receive an additional emapalumab dose within 30 days post-transplant if inflammation markers rise. During the study, participants remain hospitalized according to usual care and have follow-up visits on days 0, 7, 14, 21, 30, 45, 60, 70, 100, 180, 270, and 365, then quarterly for up to three years. Researchers monitor engraftment success, survival rates, graft-versus-host disease incidence, immune recovery, quality of life, and other health outcomes. Data collection continues long-term to assess transplant effects and safety.

A Study of Treatment of Inflammation Before Stem Cell Transplant in People With a Primary Immune Regulatory Disorder (PIRD) and/or an Autoinflammatory Condition