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New treatment option for Aplastic Anemia
Official title A Study of Emapalumab for Pediatric Aplastic Anemia
ClinicalTrials.gov ID: NCT06430788
What this study is testing
What is Emapalumab?
Emapalumab is an investigational medicine, being studied as a potential treatment for aplastic anemia.
Also referred to as Gamifant.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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 2: a mid-size study of how well it works
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 0 to 25. Healthy volunteers may be eligible.
You may be able to join if
- 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...
- Patients that do not have evidence of leukemia or MDS
- Patients \< 25 years of age at time of diagnosis
You likely can't join if
- 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...
- 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.
See the full 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: Marrow Cellularity: \<25%, or 25-50% with \<30% residual hematopoietic cells Peripheral cytopenias (at least 2 of 3) Absolute neutrophil...
- 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...
- 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.
The study team makes the final eligibility decision.
Where it's taking place
- New York, New York, United States
- Cincinnati, Ohio, United States
- Philadelphia, Pennsylvania, United States
- Richmond, Virginia, United States
- Milwaukee, Wisconsin, United States
Compensation & support
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 0 years to 25 years. Healthy volunteers may be eligible. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include New York, New York, United States; Cincinnati, Ohio, United States; Philadelphia, Pennsylvania, United States; Richmond, Virginia, United States; Milwaukee, Wisconsin, United States. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.