Emapalumab treatment for pediatric aplastic anemia
Phase 2a/2b Study Emapalumab: A Window of Opportunity in Pediatric Aplastic Anemia
This study is testing if a new treatment called emapalumab can help children with severe aplastic anemia do better with their standard treatments.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 35 (estimated) |
| Ages | 0 Years to 25 Years |
| Sex | All |
| Sponsor | Memorial Sloan Kettering Cancer Center Academic / other |
| Drugs / interventions | emapalumab |
| Locations | 6 sites (New York, New York and 5 other locations) |
| Trial ID | NCT06430788 on ClinicalTrials.gov |
What this trial studies
This study investigates the use of emapalumab as an upfront treatment for pediatric patients diagnosed with severe aplastic anemia (sAA). The goal is to evaluate whether this treatment can enhance the effectiveness of standard treatment options for sAA. Patients will be assessed based on specific criteria related to their bone marrow cellularity and peripheral blood counts. The study aims to provide insights into the potential benefits of emapalumab in managing this serious condition.
Who should consider this trial
Good fit: Ideal candidates are pediatric patients under 25 years of age with suspected newly diagnosed severe aplastic anemia and significant cytopenias.
Not a fit: Patients with uncontrolled infections, previous treatments for sAA, or known inherited bone marrow failure may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could improve outcomes for children with severe aplastic anemia by enhancing the effectiveness of existing therapies.
How similar studies have performed: While this approach is novel in the context of pediatric aplastic anemia, similar studies in other conditions have shown promising results with emapalumab.
Eligibility criteria
Show full inclusion / exclusion 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 \<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.
Where this trial is running
New York, New York and 5 other locations
- Memorial Sloan Kettering Cancer Center (All Protocol Activities) — New York, New York, United States (Recruiting)
- Cincinnati Children's Hospital Medical Center (Data collection only) — Cincinnati, Ohio, United States (Recruiting)
- Children's Hospital of Philadelphia (Data Collection AND Specimen Analysis) — Philadelphia, Pennsylvania, United States (Recruiting)
- Virginia Commonwealth University (Data Collection Only ) — Richmond, Virginia, United States (Recruiting)
- Children's Hospital of Wisconsin (Data Collection Only) — Milwaukee, Wisconsin, United States (Recruiting)
- Medical College of Wisconsin (Data Collection AND Data Analysis) — Milwaukee, Wisconsin, United States (Not_yet_recruiting)
Study contacts
- Principal investigator: Andromachi Scaradavou, MD — Memorial Sloan Kettering Cancer Center
- Study coordinator: Andromachi Scaradavou, MD
- Email: ScaradaA@mskcc.org
- Phone: 1-833-MSK-KIDS
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.