A Study of Emapalumab for Pediatric Aplastic Anemia

December 11, 2025 updated by: Memorial Sloan Kettering Cancer Center

Phase 2a/2b Study Emapalumab: A Window of Opportunity in Pediatric Aplastic Anemia

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

35

Phase

  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

Study Locations

  • United States
    • New York
      • New York, New York, United States, 10065
        • Recruiting
        • Memorial Sloan Kettering Cancer Center (All protocol activities)
        • Contact:
          • Andromachi Scaradavou, MD
          • Phone Number: 1-833-MSK-KIDS
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Recruiting
        • Cincinnati Children's Hospital Medical Center (Data Collection Only)
        • Contact:
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Children's Hospital of Philadelphia (Data Collection AND Specimen Analysis)
        • Contact:
          • Tim Olson, MD, PhD
          • Phone Number: 800-879-2467
    • Virginia
      • Richmond, Virginia, United States, 23219
        • Recruiting
        • Virginia Commonwealth University (Data Collection Only )
        • Contact:
          • Joe Laver, MD, MHA
          • Phone Number: 804-828-9213
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Not yet recruiting
        • Medical College of Wisconsin (Data Collection AND Data Analysis)
        • Contact:
          • Larisa Broglie, MD
          • Phone Number: 414-266-2420
      • Milwaukee, Wisconsin, United States, 53226
        • Recruiting
        • Children's Hospital of Wisconsin (Data Collection Only)
        • Contact:
          • Larisa Broglie, MD
          • Phone Number: 414-266-2420

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult

Accepts Healthy Volunteers

Yes

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Sequential Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Emapalumab, then Standard IST
Participants 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
Biological: Emapalumab
Emapalumab is an interferon gamma (IFNγ) blocking antibody
Other Names:
  • Gamifant
Experimental: Emapalumab, then HCT
Participants will first receive Emapalumab for 6 weeks. After treatment with emapalumab, participants will have a standard hematopoietic stem cell transplant (HCT).
Biological: Emapalumab
Emapalumab is an interferon gamma (IFNγ) blocking antibody
Other Names:
  • Gamifant

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Best Response
Time Frame: 6 weeks
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Memorial Sloan Kettering Cancer Center

Investigators

  • Principal Investigator: Andromachi Scaradavou, MD, Memorial Sloan Kettering Cancer Center

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

May 21, 2024

Primary Completion (Estimated)

May 21, 2029

Study Completion (Estimated)

May 21, 2029

Study Registration Dates

First Submitted

May 21, 2024

First Submitted That Met QC Criteria

May 21, 2024

First Posted (Actual)

May 28, 2024

Study Record Updates

Last Update Posted (Actual)

December 17, 2025

Last Update Submitted That Met QC Criteria

December 11, 2025

Last Verified

December 1, 2025

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 23-278
  • FD-R-008175 (Other Grant/Funding Number: FDA OOPD)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan Description

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.

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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