A Phase 2 Study of Emapalumab for Pediatric Aplastic Anemia


Full Title

Phase 2a/2b Study Emapalumab: A Window of Opportunity in Pediatric Aplastic Anemia


Doctors want to know if giving emapalumab as upfront pre-treatment works well for people with severe acquired aplastic anemia (sAA). This medication is given after diagnosis, but before standard sAA treatment. In people with sAA, stem cells are destroyed and the body cannot make new blood cells.

Emapalumab blocks a protein called interferon-gamma, which activates the immune system and increases inflammation. By blocking this protein, emapalumab may help reduce inflammation, decrease sAA symptoms, preserve stem cells, and help standard treatments work better. Emapalumab is given intravenously (by vein). Emapalumab is approved by the FDA to treat other conditions with overactive inflammation.

Standard treatments for sAA include immune suppression therapy (IST) or hematopoietic stem cell transplantation (HCT). However, neither treatment can be started right away after diagnosis. In addition, the inflammation caused by sAA can affect how well these treatments work.

If you join this study, you will get emapalumab soon after your diagnosis. Depending on how you respond to the drug, you may get either:

  • Standard IST in addition to a lower dose of emapalumab, or
  • HCT

Who Can Join

To join this study, there are a few conditions. You or your child must:

  • Have newly diagnosed sAA that has not yet been treated.
  • Be age 25 or younger.


For more information or to see if you or your child can join this study, please contact Dr. Joseph Oved’s office at 646-888-3314.




Phase II (phase 2)

Disease Status

Newly Diagnosed



ClinicalTrials.gov ID