NCT06430788RecruitingPhase 2
A Study of Emapalumab for Pediatric Aplastic Anemia
Phase 2a/2b Study Emapalumab: A Window of Opportunity in Pediatric Aplastic Anemia
Source: ClinicalTrials.gov · Last updated 17 Dec 2025
Brief summary (registry, in English)
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
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 35 (estimated)
- First submitted
- 21 May 2024
- Start date
- 21 May 2024
- Primary completion date
- 21 May 2029
- Lead sponsor
- Memorial Sloan Kettering Cancer Center
- Diseases
- Childhood cancers
- NCT number
- NCT06430788
What you commit to
- Study type: Interventional · Phase 2
- Planned participants: 35
- From 21 May 2024 to the primary completion date, 21 May 2029
- 6 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- Patients who have undergone previous treatment for sAA.
Other illness
- Uncontrolled infection at presentation.
- Patients with known inherited bone marrow failure
Other
- 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.
Who the study is looking for
- 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)
Age in the registry: 0 years to 25 years · Sex: All · Accepts healthy volunteers
Sites (6)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Cincinnati Children's Hospital Medical Center (Data Collection Only) Coordinator of this site? Confirm its status | Cincinnati | United States | Recruiting |
| Children's Hospital of Wisconsin (Data Collection Only) Coordinator of this site? Confirm its status | Milwaukee | United States | Recruiting |
| Medical College of Wisconsin (Data Collection AND Data Analysis) Coordinator of this site? Confirm its status | Milwaukee | United States | Not yet recruiting |
| Memorial Sloan Kettering Cancer Center (All Protocol Activities) Coordinator of this site? Confirm its status | New York | United States | Recruiting |
| Children's Hospital of Philadelphia (Data Collection AND Specimen Analysis) Coordinator of this site? Confirm its status | Philadelphia | United States | Recruiting |
| Virginia Commonwealth University (Data Collection Only ) Coordinator of this site? Confirm its status | Richmond | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.