Eflornithine (DFMO) for Ewing Sarcoma and Osteosarcoma (NCT07321912) | Clinical Trial Compass
Not Yet RecruitingPhase 2
Eflornithine (DFMO) for Ewing Sarcoma and Osteosarcoma
406 participantsStarted 2026-04-01
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 range0 Years – 50 Years
SexALL
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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:
✓. Adequate liver function defined as:
✓. 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.
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
✕. 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.