Aplastic Anemia study: Emapalumab
Official title A Study of Emapalumab for Pediatric Aplastic Anemia
ClinicalTrials.gov ID: NCT06430788
What this study is testing
What is Emapalumab?
This is an investigational medicine being studied to see if it can help treat aplastic anemia. It is designed to block a specific protein in the body called interferon gamma.
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
- This study aims to see if using emapalumab early can improve how well standard treatments work for children with aplastic anemia.
- Who it's for
- The study is looking for children with aplastic anemia, a condition where the bone marrow does not make enough blood cells.
- What's involved
- Participants will receive emapalumab along with their standard care. The team will monitor their blood counts and health to see how they respond.
- Time commitment
- The source text does not specify the total duration or the number of required visits.
- 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 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
- Able to tolerate emapalumab and IST (with standard institutional organ function criteria)
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.
The study team makes the final eligibility decision.
Where it is enrolling
- New York, New York, United States
- Cincinnati, Ohio, United States
- Philadelphia, Pennsylvania, United States
- Richmond, Virginia, United States
- Milwaukee, Wisconsin, United States
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
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, but 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?
This study is enrolling now at 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 enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.