Actively Recruiting
A Study of Emapalumab for Pediatric Aplastic Anemia
Led by Memorial Sloan Kettering Cancer Center · Updated on 2025-12-17
35
Participants Needed
6
Research Sites
260 weeks
Total Duration
On this page
AI-Summary
What this Trial 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
CONDITIONS
Official Title
A Study of Emapalumab for Pediatric Aplastic Anemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients undergoing evaluation for suspected newly diagnosed severe aplastic anemia (sAA)
- Severe cytopenias and a hypocellular marrow concerning for sAA
- Meet suspected sAA definition (Camitta Criteria): marrow cellularity less than 25%, or 25-50% with less than 30% residual hematopoietic cells
- Peripheral cytopenias with at least two of the following: ANC less than 500 x 10^9/L, platelets less than 20 x 10^9/L, absolute reticulocyte count less than 60 x 10^9/L
- No evidence of leukemia or myelodysplastic syndrome (MDS)
- Younger than 25 years of age at diagnosis
- Able to tolerate emapalumab and immunosuppressive therapy according to standard institutional organ function criteria
You will not qualify if you...
- Uncontrolled infection at presentation
- Previous treatment for severe aplastic anemia
- Known inherited bone marrow failure
- Completed full sAA workup including telomere testing, DEB, genetics when applicable, with an available willing donor and planned hematopoietic stem cell transplant within 2 weeks of enrollment
- Diagnosis of leukemia or myelodysplastic syndrome
- Inability of patient or guardian to give informed consent or comply with treatment and research protocol
AI-Screening
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Trial Site Locations
Total: 6 locations
1
Memorial Sloan Kettering Cancer Center (All Protocol Activities)
New York, New York, United States, 10065
Actively Recruiting
2
Cincinnati Children's Hospital Medical Center (Data collection only)
Cincinnati, Ohio, United States, 45229
Actively Recruiting
3
Children's Hospital of Philadelphia (Data Collection AND Specimen Analysis)
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
4
Virginia Commonwealth University (Data Collection Only )
Richmond, Virginia, United States, 23219
Actively Recruiting
5
Children's Hospital of Wisconsin (Data Collection Only)
Milwaukee, Wisconsin, United States, 53226
Actively Recruiting
6
Medical College of Wisconsin (Data Collection AND Data Analysis)
Milwaukee, Wisconsin, United States, 53226
Not Yet Recruiting
Research Team
A
Andromachi Scaradavou, MD
CONTACT
J
Jaap Jan Boelens, MD, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
2
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