Actively Recruiting
Phase 2a/2b Study of Emapalumab as Initial Treatment in Pediatric Aplastic Anemia Evaluating Immunosuppressive Therapy and Stem Cell Transplant
Led by Memorial Sloan Kettering Cancer Center · Updated on 2025-12-17
35
Participants Needed
6
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
This research aims to evaluate whether starting treatment with emapalumab can improve treatment planning and increase the effectiveness of standard therapies for pediatric patients with severe aplastic anemia (sAA). The study focuses on children and young adults under 25 years old who have been newly diagnosed with sAA characterized by severe cytopenias and hypocellular bone marrow. The trial is sponsored by Memorial Sloan Kettering Cancer Center and funded by the FDA's Office of Orphan Products Development. Participants will initially receive emapalumab, an antibody that blocks interferon gamma, for six weeks. After this initial treatment, they will either receive standard immunosuppressive therapy (IST) with equine anti-thymocyte globulin and cyclosporin along with a reduced dose of emapalumab, or they will proceed to a standard hematopoietic stem cell transplant (HCT). These two treatment paths are both experimental arms within the study. During the study, participants will be closely monitored to assess their response to treatment at six weeks. Evaluations include clinical assessments and laboratory tests to measure blood counts and marrow function. Researchers will track the best response to therapy and monitor safety throughout the process. The total study duration and follow-up extend up to May 2029, allowing for long-term observation of outcomes.
CONDITIONS
Brief Title
A Study of Emapalumab for Pediatric Aplastic Anemia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients undergoing workup for suspected newly diagnosed severe aplastic anemia
- Patients with severe cytopenias and hypocellular bone marrow concerning for severe aplastic anemia
- Patients meeting the Camitta Criteria for suspected severe aplastic anemia: marrow cellularity less than 25% or 25-50% with less than 30% residual hematopoietic cells; at least two of three peripheral cytopenias including ANC less than 500 x10^9/L, platelets less than 20 x10^9/L, and absolute reticulocyte count less than 60 x10^9/L
- Patients without evidence of leukemia or myelodysplastic syndrome
- Patients younger than 25 years at diagnosis
- Able to tolerate emapalumab and immunosuppressive therapy according to standard 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 workup for severe aplastic anemia including telomere testing, DEB, genetics (when applicable), and having a willing and available donor with planned stem cell transplant within 2 weeks of enrollment
- Diagnosis of leukemia or myelodysplastic syndrome
- Patient, parent, or guardian unable to give informed consent or comply with treatment protocol including research tests
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 6 weeks for initial Emapalumab treatment, followed by additional treatment depending on group assignment
Participants first receive Emapalumab for 6 weeks. After this, participants will either receive standard immunosuppressive therapy (hATG and cyclosporin) with a lower dose of Emapalumab or undergo a hematopoietic stem cell transplant.
Visits occur throughout initial 6 weeks of Emapalumab treatment; additional visits depend on subsequent therapy
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
J
Jaap Jan Boelens, MD, PhD
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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