Eflornithine (DFMO) for Ewing Sarcoma and Osteosarcoma (NCT07321912) | Clinical Trial Compass
RecruitingPhase 2
Eflornithine (DFMO) for Ewing Sarcoma and Osteosarcoma
United States406 participantsStarted 2026-06-05
Plain-language summary
Ewing sarcoma (EWS) and osteosarcoma primarily affect adolescents and young adults. Common treatments include chemotherapy, surgery and radiation, however, there have been few recent advancements in the standard of care. By incorporating eflornithine (DFMO) as an additional therapy and/or maintenance therapy we hope to safely observe improved event-free survival and overall survival. There are 5 cohorts covered under this master protocol.
Who can participate
Age range
0 Years – 50 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Participants must be ≤50 years of age at enrollment.
. Histologically confirmed Ewing sarcoma that is refractory or in first or subsequent relapse. Histological confirmation either at initial diagnosis or disease progression.
. Extent of disease is judged by treating team to be amenable to the delivery of definitive local control (either definitive radiation, surgery, or a combination of these) at the time of study enrollment (to be completed after protocol defined Cycle 2).
. Participants may enroll anytime during Cycle 1 or 2, prior to local control, as long as they received the same treatment during Cycle 1 and 2 as prescribed in this protocol.
. Relapsed or refractory disease, including at least one of the following:
. Participants must have adequate renal function as defined as:
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Number of Cohort 1 participants with relapse free survival (RFS) during study
Timeframe: 2 years plus 5 years follow up
2
Number of Cohort 2 participants with event-free survival (EFS) during study
Timeframe: 2 years plus 5 years follow up
3
Cohort 3: Number of Cohort 3 participants at 12 months with disease control
Timeframe: 1 year plus 5 years follow up
4
Cohort 4A: Number of Cohort 4A participants with event-free survival (EFS) during study
Timeframe: 2 years plus 5 years follow up
5
Cohort 4B: Number of Cohort 4B participants with event-free survival (EFS) during study
. Total bilirubin ≤1.5 x upper limit of normal (ULN) for age, and
Exclusion criteria
. BSA of \<0.25 m2
. Participants with current CNS disease.
. Investigational Drugs: Participants who are currently receiving another investigational drug are excluded from participation.
. Anti-cancer Agents: Participants who are currently receiving other anticancer agents are not eligible.
. Infection: Participants who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator.
. Participants who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study, or in whom compliance is likely to be suboptimal, should be excluded.
. Participants must be ≤50 years of age at enrollment.
. Participants with histologic diagnosis (by institutional pathologist) of newly diagnosed Ewing sarcoma or peripheral primitive neuroectodermal tumor (PNET) arising from bone or soft tissue and with metastatic disease involving lung, bone, bone marrow, or other metastatic site.