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RecruitingAccepts healthy volunteersPhase 2

A Study of Emapalumab for Pediatric Aplastic Anemia

Memorial Sloan Kettering Cancer Center · NCT06430788

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Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 0 mo–25 · All sexes
Healthy volunteers
Accepted
Study type
Interventional · Phase 2
Target enrollment
35
US sites recruiting
5
Study start
May 21, 2024
Record last updated
December 17, 2025

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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

Being studied: Emapalumab

Conditions: Aplastic Anemia; Cytopenia; Hypocellular Marrow

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full 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.

Recruiting US locations

  • Memorial Sloan Kettering Cancer Center (All Protocol Activities)

    New York, NY 10065

    Site contact: Andromachi Scaradavou, MD · 1-833-MSK-KIDS

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

    Cincinnati, OH 45229

    Site contact: Anthony Sabulski, MD · 513-636-3200 · anthony.sabulski@cchmc.org

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

    Philadelphia, PA 19104

    Site contact: Tim Olson, MD, PhD · 800-879-2467

  • Virginia Commonwealth University (Data Collection Only )

    Richmond, VA 23219

    Site contact: Joe Laver, MD, MHA · 804-828-9213

  • Children's Hospital of Wisconsin (Data Collection Only)

    Milwaukee, WI 53226

    Site contact: Larisa Broglie, MD · 414-266-2420

Study contacts (from the official record)

  • Andromachi Scaradavou, MD · 1-833-MSK-KIDS · ScaradaA@mskcc.org
  • Jaap Jan Boelens, MD, PhD · 1-833-MSK-KIDS · boelensj@mskcc.org
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A Study of Emapalumab for Pediatric Aplastic Anemia (NCT06430788) | Bored Studies