A Study of Emapalumab for Pediatric Aplastic Anemia

Launched by MEMORIAL SLOAN KETTERING CANCER CENTER · May 21, 2024

Keywords

ClinConnect Summary

This clinical trial is studying a medication called emapalumab to see if it can help children and young adults with a condition known as severe Aplastic Anemia (sAA). Aplastic Anemia is a serious condition where the bone marrow doesn’t produce enough blood cells, leading to problems like severe fatigue and increased risk of infections. The goal of the study is to find out if starting treatment with emapalumab can improve the overall effectiveness of standard treatments for this condition.

The trial is open to children and young adults under 25 years old who are being evaluated for newly diagnosed sAA. To participate, they should have low blood cell counts and specific signs of the disease, but they should not have other blood disorders like leukemia. Participants will receive emapalumab along with standard treatments and will be monitored closely for their response. It’s important for families to know that this trial is still recruiting participants, meaning there may be opportunities for eligible patients to join and potentially benefit from this new treatment approach.

Gender

ALL

Eligibility criteria

• Inclusion Criteria:
• * Patients undergoing workup for suspected newly diagnosed sAA:
• • Patients with severe cytopenias and a hypocellular marrow concerning for sAA
• * Patients that meet the definition for suspected sAA (Camitta Criteria) as follows:
• • Marrow Cellularity: \<25%, or 25-50% with \<30% residual hematopoietic cells Peripheral cytopenias (at least 2 of 3) Absolute neutrophil count (ANC): \<500 x 10\^9/L Platelets: \<20 x 10\^9/L Absolute Reticulocyte Count: \<60 x 10\^9/L
• • Patients that do not have evidence of leukemia or MDS
• • Patients \< 25 years of age at time of diagnosis
• • Able to tolerate emapalumab and IST (with standard institutional organ function criteria)
• Exclusion Criteria:
• • Uncontrolled infection at presentation.
• • Patients who have undergone previous treatment for sAA.
• • Patients with known inherited bone marrow failure
• • Patient who has completed a full workup for sAA including having results back from telomere testing, DEB and genetics (when applicable), as well as having an appropriate willing and available donor and would otherwise be admitted for HSCT within 2 weeks of enrolling on the trial
• • Patients with leukemia or MDS
• • Patient or parent or guardian unable to give informed consent or unable to comply with the treatment protocol including research tests.

Trial Officials