A Study of Emapalumab for Pediatric Aplastic Anemia
Phase 2a/2b Study Emapalumab: A Window of Opportunity in Pediatric Aplastic Anemia
2 other identifiers
interventional
35
1 country
6
Brief 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
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started May 2024
Longer than P75 for phase_2
6 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
May 21, 2024
CompletedStudy Start
First participant enrolled
May 21, 2024
CompletedFirst Posted
Study publicly available on registry
May 28, 2024
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 21, 2029
ExpectedStudy Completion
Last participant's last visit for all outcomes
May 21, 2029
December 17, 2025
December 1, 2025
5 years
May 21, 2024
December 11, 2025
Conditions
Keywords
Outcome Measures
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.
6 weeks
Study Arms (2)
Emapalumab, then Standard IST
EXPERIMENTALParticipants will first receive Emapalumab for 6 weeks. After treatment with emapalumab, participants will receive standard IST with drugs called equine anti-thymocyte globulin (hATG) and cyclosporin (CsA) in addition to a lower dose of emapalumab
Emapalumab, then HCT
EXPERIMENTALParticipants will first receive Emapalumab for 6 weeks. After treatment with emapalumab, participants will have a standard hematopoietic stem cell transplant (HCT).
Interventions
Emapalumab is an interferon gamma (IFNγ) blocking antibody
Eligibility Criteria
You may qualify if:
- 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)
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (6)
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
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- PRINCIPAL INVESTIGATOR
Andromachi Scaradavou, MD
Memorial Sloan Kettering Cancer Center
Central Study Contacts
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SEQUENTIAL
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
May 21, 2024
First Posted
May 28, 2024
Study Start
May 21, 2024
Primary Completion (Estimated)
May 21, 2029
Study Completion (Estimated)
May 21, 2029
Last Updated
December 17, 2025
Record last verified: 2025-12
Data Sharing
- IPD Sharing
- Will share
Memorial Sloan Kettering Cancer Center supports the international committee of medical journal editors (ICMJE) and the ethical obligation of responsible sharing of data from clinical trials. The protocol summary, a statistical summary, and informed consent form will be made available on clinicaltrials.gov when required as a condition of Federal awards, other agreements supporting the research and/or as otherwise required. Requests for deidentified individual participant data can be made beginning 12 months after publication and for up to 36 months post publication. Deidentified individual participant data reported in the manuscript will be shared under the terms of a Data Use Agreement and may only be used for approved proposals. Requests may be made to: crdatashare@mskcc.org.