Clinical trial · Interventional
A Study of Emapalumab for Pediatric Aplastic Anemia
Phase 2a/2b Study Emapalumab: A Window of Opportunity in Pediatric Aplastic Anemia
NCT06430788CI-TRIAL-00099162recruitingPhase 2ClinicalTrials.gov clinicaltrialsProvenance
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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
Conditions (3)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Aplastic Anemia | — | UNRESOLVED | — |
| Cytopenia | — | UNRESOLVED | — |
| Hypocellular Marrow | — | UNRESOLVED | — |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Emapalumab | Biological | — | UNRESOLVED |
Design
Arms and outcomes
Arms (2)
- type
- EXPERIMENTAL
- label
- Emapalumab, then Standard IST
- description
- Participants will first receive Emapalumab for 6 weeks. After treatment with emapalumab, participants will receive standard IST with drugs called equine anti-thymocyte globulin (hATG) and cyclosporin (CsA) in addition to a lower dose of emapalumab
- interventionNames
- Biological: Emapalumab
- type
- EXPERIMENTAL
- label
- Emapalumab, then HCT
- description
- Participants will first receive Emapalumab for 6 weeks. After treatment with emapalumab, participants will have a standard hematopoietic stem cell transplant (HCT).
- interventionNames
- Biological: Emapalumab
Primary outcomes (1)
- measure
- Best Response
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 0 Years
- Maximum age
- 25 Years
Show eligibility criteria text
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.
References
Publications (0)
Data not yet available
No reference posted for this study.