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NCT06430788PHASE2RecruitingAccepts Healthy Volunteers

A Study of Emapalumab for Pediatric Aplastic Anemia

Memorial Sloan Kettering Cancer Center

Start Date

5/21/2024

Completion Date

5/21/2029

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 Criteria

Age Range: No minimum to 25 years

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.

Interventions

BIOLOGICAL

Emapalumab

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Conditions

Aplastic AnemiaCytopeniaHypocellular Marrow

Locations

Memorial Sloan Kettering Cancer Center (All Protocol Activities)

New York, New York 10065

United States

Cincinnati Children's Hospital Medical Center (Data collection only)

Cincinnati, Ohio 45229

United States

Children's Hospital of Philadelphia (Data Collection AND Specimen Analysis)

Philadelphia, Pennsylvania 19104

United States

Virginia Commonwealth University (Data Collection Only )

Richmond, Virginia 23219

United States

Children's Hospital of Wisconsin (Data Collection Only)

Milwaukee, Wisconsin 53226

United States

Medical College of Wisconsin (Data Collection AND Data Analysis)

Milwaukee, Wisconsin 53226

United States