Phase 2a/2b Study Emapalumab: A Window of Opportunity in Pediatric Aplastic Anemia
Memorial Sloan Kettering Cancer Center
Summary
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
Eligibility
- Age range
- 0–25 years
- Sex
- All
- Healthy volunteers
- Yes
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 *…
Interventions
- BiologicalEmapalumab
Emapalumab is an interferon gamma (IFNγ) blocking antibody
Locations (6)
- Memorial Sloan Kettering Cancer Center (All Protocol Activities)New York, New York
- Cincinnati Children's Hospital Medical Center (Data collection only)Cincinnati, Ohio
- Children's Hospital of Philadelphia (Data Collection AND Specimen Analysis)Philadelphia, Pennsylvania
- Virginia Commonwealth University (Data Collection Only )Richmond, Virginia
- Children's Hospital of Wisconsin (Data Collection Only)Milwaukee, Wisconsin
- Medical College of Wisconsin (Data Collection AND Data Analysis)Milwaukee, Wisconsin