Emapalumab for Aplastic Anemia in Children

A Study of Emapalumab for Pediatric Aplastic Anemia

Recruiting Phase 2 Interventional Study
Aplastic Anemia Cytopenia Hypocellular Marrow
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial. Healthy Volunteers Welcome You do not need to have the condition being studied to take part.
Ready to participate?

Review the details below, then apply to join this clinical trial.

At a Glance
Age
0 – 25
Sex
Any
Trial phase
Phase 2
Study type
Interventional
Purpose
Treatment
Participants needed
35 (estimated)
Sponsor
Memorial Sloan Kettering Cancer Center · Other
Who this trial is looking for

This trial is looking for children and young adults under 25 who may have a type of blood disorder called severe Aplastic Anemia. Taking part may involve receiving the treatment emapalumab and undergoing some standard tests to help plan further treatment.

Are You a Good Fit for This Trial?

You may be able to join if

  • I am under 25 years old.
  • I have severe low blood cell counts and a bone marrow biopsy shows a decrease in cells.
  • I may have Aplastic Anemia based on specific medical criteria.
  • I do not have leukemia or myelodysplastic syndromes (MDS).
  • I can tolerate treatment with emapalumab.

You may not be able to join if

  • I have an uncontrolled infection.
  • I have received treatment for Aplastic Anemia before.
  • I have a known inherited bone marrow failure.
  • I have completed all necessary tests for Aplastic Anemia and am ready for a stem cell transplant.
  • I have leukemia or MDS.
  • I or my guardian cannot provide informed consent.

Summarized in plain language from this trial's official eligibility criteria. The full criteria are further down this page — only the research team can confirm whether you qualify.

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About This Trial

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

Trial Locations
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Eligibility 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 \<3…
Contacts

Andromachi Scaradavou, MD

1-833-MSK-KIDS

ScaradaA@mskcc.org

CONTACT

Jaap Jan Boelens, MD, PhD

1-833-MSK-KIDS

boelensj@mskcc.org

CONTACT