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Clinical Trial Details — Status: Recruiting

Administrative data

NCT number NCT06430788
Other study ID # 23-278
Secondary ID
Status Recruiting
Phase Phase 2
First received
Last updated
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
Health authority
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.

Study Design


Related Conditions & MeSH terms


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
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