A Study of Emapalumab for Pediatric Aplastic Anemia

Phase 2

Recruiting

• Conditions: Aplastic Anemia; Hypocellular Marrow; Cytopenia
• Interventions: Biological: Emapalumab

• Registration Number: NCT06430788
• Lead Sponsor: Memorial Sloan Kettering Cancer Center

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

Detailed Description

Not available

Study Timeline

• Study Start: 2024-05-21
• Study First Submitted: 2024-05-21
• Study First Submitted QC: 2024-05-21
• Study First Posted: 2024-05-28
• Status Verified: 2025-04
• Last Update Submitted: 2025-05-14
• Last Update Posted: 2025-05-18
• Primary Completion: 2029-05-21
• Study Completion: 2029-05-21

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 55

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

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

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

Primary Outcome Measures

Name	Time	Method
Best Response	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.

Trial Locations

Locations (5)

Memorial Sloan Kettering Cancer Center (All Protocol Activities); 🇺🇸 New York, New York, United States

Cincinnati Children's Hospital Medical Center (Data collection only); 🇺🇸 Cincinnati, Ohio, United States

Children's Hospital of Philadelphia (Data Collection AND Specimen Analysis); 🇺🇸 Philadelphia, Pennsylvania, United States

Virginia Commonwealth University (Data Collection Only ); 🇺🇸 Richmond, Virginia, United States

Medical College of Wisconsin (Data Collection AND Data Analysis); 🇺🇸 Milwaukee, Wisconsin, United States