Emapalumab treatment for pediatric aplastic anemia

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This study investigates the use of emapalumab as an upfront treatment for pediatric patients diagnosed with severe aplastic anemia (sAA). The goal is to evaluate whether this treatment can enhance the effectiveness of standard treatment options for sAA. Patients will be assessed based on specific criteria related to their bone marrow cellularity and peripheral blood counts. The study aims to provide insights into the potential benefits of emapalumab in managing this serious condition.

Why it matters

Who can join: the full rules

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.

Where it's running

• Memorial Sloan Kettering Cancer Center (All Protocol Activities)
  New York, New York, United States Recruiting
• Cincinnati Children's Hospital Medical Center (Data collection only)
  Cincinnati, Ohio, United States Recruiting
• Children's Hospital of Philadelphia (Data Collection AND Specimen Analysis)
  Philadelphia, Pennsylvania, United States Recruiting
• Virginia Commonwealth University (Data Collection Only )
  Richmond, Virginia, United States Recruiting
• Children's Hospital of Wisconsin (Data Collection Only)
  Milwaukee, Wisconsin, United States Recruiting
• Medical College of Wisconsin (Data Collection AND Data Analysis)
  Milwaukee, Wisconsin, United States Not yet recruiting

Study contacts

• Principal investigator: Andromachi Scaradavou, MD — Memorial Sloan Kettering Cancer Center
• Study coordinator: Andromachi Scaradavou, MD
• Email: ScaradaA@mskcc.org
• Phone: 1-833-MSK-KIDS

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

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• Preventing graft rejection after allogeneic stem cell transplant

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How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.