A Study of Emapalumab for Pediatric Aplastic Anemia

Aplastic Anemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06430788
• Other study ID #: 23-278
• Status: Recruiting
• Phase: Phase 2
• Start date: May 21, 2024
• Est. completion date: May 21, 2029

Study information

• Verified date: May 2024
• Source: Memorial Sloan Kettering Cancer Center
• Contact: Joseph Oved, MD
• Phone: 1-833-MSK-KIDS
• Email: ovedj[@]mskcc.org
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 55
• Est. completion date: May 21, 2029
• Est. primary completion date: May 21, 2029
• Accepts healthy volunteers: Accepts Healthy Volunteers
• Gender: All
• Age group: 0 Years to 25 Years

Eligibility

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.

Related Conditions & MeSH terms

• Anemia
• Anemia, Aplastic
• Aplastic Anemia
• Cytopenia

Intervention

Biological:
• Emapalumab
Emapalumab is an interferon gamma (IFN?) blocking antibody

Locations

Country	Name	City	State
United States	Cincinnati Children's Hospital Medical Center (Data collection only)	Cincinnati	Ohio
United States	Medical College of Wisconsin (Data Collection AND Data Analysis)	Milwaukee	Wisconsin
United States	Memorial Sloan Kettering Cancer Center (All Protocol Activities)	New York	New York
United States	Children's Hospital of Philadelphia (Data Collection AND Specimen Analysis)	Philadelphia	Pennsylvania
United States	Virginia Commonwealth Univeristy (Data Collection Only )	Richmond	Virginia

Sponsors (1)

Lead Sponsor	Collaborator
Memorial Sloan Kettering Cancer Center

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Best Response	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

External Links

•   Memorial Sloan Kettering Cancer Center