NCT06430788 · Memorial Sloan Kettering Cancer Center
A Study of Emapalumab for Pediatric Aplastic Anemia
What this study is about
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.
View original scientific 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.
Interventions
BIOLOGICAL
Emapalumab
Emapalumab is an interferon gamma (IFNγ) blocking antibody
Primary outcome measures
Best Response
Time frame: 6 weeks
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.
Who can participate
This study lists these criteria on ClinicalTrials.gov. A study coordinator reviews eligibility during screening — this page does not determine whether you qualify.
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 applicabl
Where
- New York, New York
- Cincinnati, Ohio
- Philadelphia, Pennsylvania
- Richmond, Virginia
- Milwaukee, Wisconsin
Related conditions & keywords
Frequently asked questions
What is a clinical trial?
A clinical trial is a research study that tests new medical treatments, drugs, devices, or procedures to determine their safety and effectiveness. Trials are carefully designed and monitored to protect participants while advancing medical knowledge.
Is it safe to participate?
Clinical trials follow strict safety guidelines and ethical standards. Trials must be reviewed and approved, and participants are closely monitored by medical professionals throughout the study. You can withdraw at any time if you choose.
Will I be compensated?
Many clinical trials offer compensation for your time, travel expenses, and inconvenience. The specific compensation varies by study and will be discussed during the screening process. All study-related medical care is typically provided at no cost to participants.
Will I receive a placebo instead of treatment?
When effective treatment exists, participants typically receive either the standard treatment plus the study intervention, or the standard treatment plus placebo. You would not be denied effective care. Placebos are primarily used when no proven treatment is available, or in addition to standard care. Your trial consent form will clearly explain what treatments you may receive.
Can I leave a trial if I change my mind?
Absolutely. Participation in clinical trials is completely voluntary. You have the right to withdraw from the study at any time, for any reason, without penalty or loss of benefits to which you are otherwise entitled.
How long does a clinical trial last?
Trial duration varies widely depending on the study design and purpose. Some trials last just a few weeks, while others may continue for months or years. The study coordinator will provide specific timeline information during your screening call.
Data: ClinicalTrials.gov · synced Dec 17, 2025 · Source of record for eligibility and locations