RECRUITING

A Study of Emapalumab for Pediatric Aplastic Anemia

Conditions

Aplastic Anemia Cytopenia Hypocellular Marrow pediatric aplastic anemia Emapalumab Memorial Sloan Kettering Cancer Center 23-278

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

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

Official Title

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

Quick Facts

Study Start:2024-05-21

Study Completion:2029-05-21

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06430788

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.

Ages Eligible for Study:0 Years to 25 Years

Sexes Eligible for Study:ALL

Accepts Healthy Volunteers:Yes

Standard Ages:CHILD, ADULT

Inclusion Criteria	Exclusion 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.

Inclusion Criteria

Exclusion 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.

Contacts and Locations

Study Contact

Joseph Oved, MD

CONTACT

1-833-MSK-KIDS

ovedj@mskcc.org

Jaap Jan Boelens, MD, PhD

CONTACT

1-833-MSK-KIDS

boelensj@mskcc.org

Principal Investigator

Joseph Oved, MD

PRINCIPAL_INVESTIGATOR

Memorial Sloan Kettering Cancer Center

Study Locations (Sites)

Memorial Sloan Kettering Cancer Center (All Protocol Activities)

New York, New York, 10065

United States

Cincinnati Children's Hospital Medical Center (Data collection only)

Cincinnati, Ohio, 45229

United States

Children's Hospital of Philadelphia (Data Collection AND Specimen Analysis)

Philadelphia, Pennsylvania, 19104

United States

Virginia Commonwealth University (Data Collection Only )

Richmond, Virginia, 23219

United States

Medical College of Wisconsin (Data Collection AND Data Analysis)

Milwaukee, Wisconsin, 53226

United States

Collaborators and Investigators

Sponsor: Memorial Sloan Kettering Cancer Center

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

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-05-21

Study Completion Date2029-05-21

Study Record Updates

Study Start Date2024-05-21

Study Completion Date2029-05-21

Terms related to this study

Keywords Provided by Researchers

pediatric aplastic anemia
aplastic anemia
cytopenia
hypocellular marrow
Emapalumab
Memorial Sloan Kettering Cancer Center
23-278

Additional Relevant MeSH Terms

Aplastic Anemia
Cytopenia
Hypocellular Marrow