A Study of Emapalumab for Pediatric Aplastic Anemia
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age0-25
SponsorMemorial Sloan Kettering Cancer Center
About this trial
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
This trial accepts healthy volunteersQualifiers
Patients with severe cytopenias and a hypocellular marrow concerning for sAA
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)
Disqualifiers
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
Trial design
Design model
Sequential
Treatments tested in this trial
Emapalumab
Biological/VaccineEmapalumab is an interferon gamma (IFNγ) blocking antibody
Treatment groups
35 Participants
are divided into 2 treatment groupsGroup A: Emapalumab, then Standard ISTExperimental treatment 1 intervention
Group B: Emapalumab, then HCTExperimental treatment 1 intervention
Trial outcomes
Primary outcomes
1
Best Response
The primary objective of the study is to assess the efficacy of early upfront emapalumab on hematologic recovery within 6 weeks of starting therapy after a new diagnosis of Aplastic Anemia. Response will be determined by blood count.
Time frame
6 weeksSponsors and contacts
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Source ClinicalTrials.gov