NCT06430788PHASE2RecruitingAccepts Healthy Volunteers
A Study of Emapalumab for Pediatric Aplastic Anemia
Memorial Sloan Kettering Cancer Center
Start Date
5/21/2024
Completion Date
5/21/2029
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 Criteria
Age Range: No minimum to 25 years
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.
Interventions
BIOLOGICAL
Emapalumab
Conditions
Aplastic AnemiaCytopeniaHypocellular Marrow
Locations
Memorial Sloan Kettering Cancer Center (All Protocol Activities)
New York, New York 10065
United States
Cincinnati Children's Hospital Medical Center (Data collection only)
Cincinnati, Ohio 45229
United States
Children's Hospital of Philadelphia (Data Collection AND Specimen Analysis)
Philadelphia, Pennsylvania 19104
United States
Virginia Commonwealth University (Data Collection Only )
Richmond, Virginia 23219
United States
Children's Hospital of Wisconsin (Data Collection Only)
Milwaukee, Wisconsin 53226
United States
Medical College of Wisconsin (Data Collection AND Data Analysis)
Milwaukee, Wisconsin 53226
United States