A Study of Emapalumab for Pediatric Aplastic Anemia
Recruiting
Phase 2Interventional Study
Aplastic AnemiaCytopeniaHypocellular 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.
Think this trial could be right for you?
Answer a few quick questions to see if you may meet the eligibility requirements.
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…
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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