A Study of Emapalumab for Pediatric Aplastic Anemia

Memorial Sloan Kettering Cancer Center (MSK) logo

Memorial Sloan Kettering Cancer Center (MSK)

Status and phase

Enrolling
Phase 2

Conditions

Cytopenia
Hypocellular Marrow
Aplastic Anemia

Treatments

Biological: Emapalumab

Study type

Interventional

Funder types

Other

Identifiers

NCT06430788
23-278

Details and patient eligibility

About

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.

Enrollment

55 estimated patients

Sex

All

Ages

Under 25 years old

Volunteers

Accepts Healthy Volunteers

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.

Trial design

Primary purpose

Treatment

Allocation

Non-Randomized

Interventional model

Sequential Assignment

Masking

None (Open label)

55 participants in 2 patient groups

Emapalumab, then Standard IST
Experimental group
Description:
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
Treatment:
Biological: Emapalumab
Emapalumab, then HCT
Experimental group
Description:
Participants will first receive Emapalumab for 6 weeks. After treatment with emapalumab, participants will have a standard hematopoietic stem cell transplant (HCT).
Treatment:
Biological: Emapalumab

Trial contacts and locations

5

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Central trial contact

Jaap Jan Boelens, MD, PhD; Joseph Oved, MD

Data sourced from clinicaltrials.gov

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