RecruitingPhase 2NCT06430788

A Study of Emapalumab for Pediatric Aplastic Anemia

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

Sponsor

Memorial Sloan Kettering Cancer Center

Enrollment

35 participants

Start Date

May 21, 2024

Study Type

INTERVENTIONAL

Conditions

Aplastic Anemia Cytopenia Hypocellular Marrow

Summary

The purpose of this study is to find out whether upfront emapalumab treatment can help in sAA (Aplastic Anemia) treatment planning and increase the effectiveness of standard treatment options. Funding Source- FDA OOPD

Eligibility

Min Age: 0 YearsMax Age: 25 Years

Inclusion Criteria7

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 Criteria6

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.