A Study of Emapalumab for Pediatric Aplastic Anemia

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

FD-R-008175 (Other Grant/Funding Number)

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.

Funding Source- FDA OOPD

Enrollment: 35 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)

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35 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