Recruiting
Phase 2

Emapalumab

Sponsor:

Memorial Sloan Kettering Cancer Center

Code:

NCT06430788

Conditions

Aplastic Anemia

Cytopenia

Hypocellular Marrow

Eligibility Criteria

Sex: All

Age: 0 - 25

Healthy Volunteers: Accepted

Interventions

Emapalumab

Study Details

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

Conditions

Aplastic Anemia

Cytopenia

Hypocellular Marrow

Study ID

NCT06430788

Start date

May 21, 2024

Status verified date

Dec, 2025

Completion date

May 21, 2029

Anticipated

Primary completion date

May 21, 2029

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 0 - 25

Healthy Volunteers: Accepted

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 Design

Enrollment

35 participants

Anticipated

Allocation

Non randomized

Intervention Model

Sequential

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

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

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).

Interventions

Emapalumab

Emapalumab is an interferon gamma (IFNγ) blocking antibody

Primary outcome measure

  • Best Response [ Time Frame: 6 weeks ]

Central Contacts and Locations

Central contacts

Andromachi Scaradavou, MD

1-833-MSK-KIDSScaradaA@mskcc.org

Jaap Jan Boelens, MD, PhD

1-833-MSK-KIDSboelensj@mskcc.org

Locations

Memorial Sloan Kettering Cancer Center (All Protocol Activities)

Recruiting

New York, New York, United States, 10065

Contacts

Andromachi Scaradavou, MD

1-833-MSK-KIDS

Cincinnati Children's Hospital Medical Center (Data collection only)

Recruiting

Cincinnati, Ohio, United States, 45229

Contacts

Anthony Sabulski, MD

513-636-3200anthony.sabulski@cchmc.org

Children's Hospital of Philadelphia (Data Collection AND Specimen Analysis)

Recruiting

Philadelphia, Pennsylvania, United States, 19104

Contacts

Tim Olson, MD, PhD

800-879-2467

Virginia Commonwealth University (Data Collection Only )

Recruiting

Richmond, Virginia, United States, 23219

Contacts

Joe Laver, MD, MHA

804-828-9213

Children's Hospital of Wisconsin (Data Collection Only)

Recruiting

Milwaukee, Wisconsin, United States, 53226

Contacts

Larisa Broglie, MD

414-266-2420

More Information

Sponsor

Memorial Sloan Kettering Cancer Center

Last update posted

Dec 17, 2025

Last verified

Dec, 2025

Keywords

  • pediatric aplastic anemia
  • aplastic anemia
  • cytopenia
  • hypocellular marrow
  • Emapalumab
  • Memorial Sloan Kettering Cancer Center
  • 23-278

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-08. This information was provided to ClinicalTrials.gov by Memorial Sloan Kettering Cancer Center on 2025-12-17.