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โ† Trials
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A Study of Emapalumab for Pediatric Aplastic Anemia

RecruitingPhase 2Ages 0 Years - 25 YearsAllMemorial Sloan Kettering Cancer Center
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In plain words

What the trial is, and whether it could suit you

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

Sign in to get the eligibility rules as simple questions to take to your doctor.

๐Ÿ“‹ Eligibility rules (as published)
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.
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What it tests

  • Emapalumab
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Where

6 sites
Memorial Sloan Kettering Cancer Center (All Protocol Activities)
New York, United States
Cincinnati Children's Hospital Medical Center (Data collection only)
Cincinnati, United States
Children's Hospital of Philadelphia (Data Collection AND Specimen Analysis)
Philadelphia, United States
Virginia Commonwealth University (Data Collection Only )
Richmond, United States
Children's Hospital of Wisconsin (Data Collection Only)
Milwaukee, United States
Medical College of Wisconsin (Data Collection AND Data Analysis)
Milwaukee, United States
๐Ÿค Taking part is your choice, and you can leave at any time. Only the trial team can confirm you are eligible โ€” always decide together with your own doctor.
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