A Study of Emapalumab for Pediatric Aplastic Anemia
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Emapalumab.
- Who it may be relevant to
- Registry conditions: Aplastic Anemia, Cytopenia, Hypocellular Marrow. Basic parameters: 0 years — 25 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official title
Phase 2a/2b Study Emapalumab: A Window of Opportunity in Pediatric Aplastic Anemia
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
Interventions
- Biological Emapalumab
Emapalumab is an interferon gamma (IFNγ) blocking antibody
Primary outcome measures
- Best Response [Time frame: 6 weeks]
Eligibility criteria
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.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: Yes
Study design
- Allocation
- Non-randomized
- Model
- Sequential
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
United States · 6 centers
- Memorial Sloan Kettering Cancer Center (All Protocol Activities) — New York
- Cincinnati Children's Hospital Medical Center (Data collection only) — Cincinnati
- Children's Hospital of Philadelphia (Data Collection AND Specimen Analysis) — Philadelphia
- Virginia Commonwealth University (Data Collection Only ) — Richmond
- Children's Hospital of Wisconsin (Data Collection Only) — Milwaukee
- Medical College of Wisconsin (Data Collection AND Data Analysis) — Milwaukee
Identifiers
NCT: NCT06430788 · 23-278 · FD-R-008175