A Study of Emapalumab for Pediatric Aplastic Anemia

Description

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

Conditions

Aplastic Anemia, Cytopenia, Hypocellular Marrow

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Study Overview

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Study Details

Study overview

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

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

A Study of Emapalumab for Pediatric Aplastic Anemia

Condition
Aplastic Anemia
Intervention / Treatment

-

Contacts and Locations

New York

Memorial Sloan Kettering Cancer Center (All Protocol Activities), New York, New York, United States, 10065

Cincinnati

Cincinnati Children's Hospital Medical Center (Data collection only), Cincinnati, Ohio, United States, 45229

Philadelphia

Children's Hospital of Philadelphia (Data Collection AND Specimen Analysis), Philadelphia, Pennsylvania, United States, 19104

Richmond

Virginia Commonwealth University (Data Collection Only ), Richmond, Virginia, United States, 23219

Milwaukee

Medical College of Wisconsin (Data Collection AND Data Analysis), Milwaukee, Wisconsin, United States, 53226

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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:
  • * 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)
  • * 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.

Ages Eligible for Study

0 Years to 25 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

Yes

Collaborators and Investigators

Memorial Sloan Kettering Cancer Center,

Joseph Oved, MD, PRINCIPAL_INVESTIGATOR, Memorial Sloan Kettering Cancer Center

Study Record Dates

2029-05-21