RecruitingPhase 2NCT06430788
A Study of Emapalumab for Pediatric Aplastic Anemia
Phase 2a/2b Study Emapalumab: A Window of Opportunity in Pediatric Aplastic Anemia
Sponsor
Memorial Sloan Kettering Cancer Center
Enrollment
35 participants
Start Date
May 21, 2024
Study Type
INTERVENTIONAL
Conditions
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
Min Age: 0 YearsMax Age: 25 Years
Inclusion Criteria7
- 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 Criteria6
- 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.
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Interventions
BIOLOGICALEmapalumab
Emapalumab is an interferon gamma (IFNγ) blocking antibody
Locations(6)
View Full Details on ClinicalTrials.gov
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NCT06430788
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