Eflornithine (DFMO) for Ewing Sarcoma and Osteosarcoma

This study is testing a drug called eflornithine (DFMO) for people with Ewing sarcoma and osteosarcoma. These cancers often affect young people, and while current treatments like chemotherapy, surgery, and radiation are used, there haven't been many new advancements. Researchers hope that adding eflornithine, either as an extra treatment or a maintenance therapy, will help improve how long people live without their cancer returning or getting worse. The study will enroll up to 406 participants aged 0 to 50 years old. It will look at how long participants live without their cancer returning (relapse-free survival) or without certain events happening (event-free survival), and how well the disease is controlled. Some participants will receive eflornithine along with standard chemotherapy drugs like Topotecan, Cyclophosphamide, Vincristine, and Doxorubicin. You may be eligible if you have Ewing sarcoma that has come back or is not responding to treatment.

Study design
This interventional study plans to enroll 406 participants. It is testing eflornithine in combination with standard chemotherapy drugs.
What's involved
Not specified in the trial record.
Compensation
Not stated in the trial record.
Follow-up
Participants will be followed for 2 years plus an additional 5 years for relapse-free survival and event-free survival, and for 1 year plus an additional 5 years for disease control.

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NCT07321912

Eflornithine (DFMO) for Ewing Sarcoma and Osteosarcoma

Recruiting
PHASE2Ages 0–50InterventionalTreatment
Milton S. Hershey Medical Center
~406 participants
Updated 2026-07-09 on ClinicalTrials.gov
What's tested:EflornithineTopotecanCyclophosphamideVincristineDoxorubicinIfosfamide

At a glance

Recruiting sites
2 of 2 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Number of Cohort 1 participants with relapse free survival (RFS) during study
Measured over 2 years plus 5 years follow up
+4 more outcomes measured
Osteosarcoma
Ewing Sarcoma
2 sites across 2 states
Florida1
Pennsylvania1
  • Giselle SaulnierSholler, MD · STUDY_CHAIR · Penn State Health Children's Hospital

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Eligibility criteria

Inclusion

Relapsed: Participants that have achieved CR at any point and then relapsed following/during standard of care therapy.
Refractory: Participants that failed to achieve CR after standard of care therapy or having progressed during standard of care therapy.
Note: Standard of care therapy for Ewing sarcoma includes multi-agent chemotherapy with local control consisting of either surgery and/or radiation therapy. 3. 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). 4. 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. 5. Relapsed or refractory disease, including at least one of the following:
Tumor by CT or MRI
FDG-PET that is positive for disease
Bone Marrow biopsy/aspirate that is positive for disease
For participants \< 17 years old: estimated Glomerular Filtration rate (eGFR) as calculated from the Bedside Schwartz equation (in units of mL/min/1.73 m2) or via radioisotope GFR of ≥ 70 mL/min/1.73 m2. The Bedside Schwartz equation is: \[(0.413) X (Height in cm)\] / SCr
For participants ≥17 years old: estimated Glomerular Filtration rate (eGFR) as calculated from the Cockcroft and Gault formula (in units of mL/min/1.73 m2) or via radioisotope GFR of ≥ 70 mL/min/1.73 m2. The Cockcroft and Gault formula is: \[(140-age) x (Wt in kg) x (0.85 if female)\] / (72 x SCr)
OR a 24 hour urine Creatinine clearance ≥ 70 mL/min/1.73 m2 7. Adequate liver function defined as:
Age
Lesions which are discontinuous from the primary tumor, are not regional lymph nodes, and do not share a bone or body cavity with the primary tumor. Skip lesions in the same bone as the primary tumor do not constitute metastatic disease. Skip lesions in an adjacent bone are considered bone metastases. If there is any doubt whether lesions are metastatic, a biopsy of those lesions should be performed.
Contralateral pleural effusion and/or contralateral pleural nodules.
Distant lymph node involvement.
Participants with pulmonary nodules are considered to have metastatic disease if the participant has:
Solitary nodule ≥0.5 cm or multiple nodules of ≥0.3 cm unless lesion is biopsied and negative for tumor;
Participants with solitary nodule \<0.5 cm or multiple nodules \<0.3 cm are not considered to have lung metastasis unless biopsy documents tumor.
Bone marrow metastatic disease is based on morphologic evidence of Ewing sarcoma based on H\&E stains. In the absence of morphologic evidence of marrow involvement on H\&E, participants with bone marrow involvement detected ONLY by flow cytometry, RT PCR, FISH, or immunohistochemistry will NOT be considered to have clinical bone marrow involvement for the purposes of this study.
For participants \< 17 years old: estimated Glomerular Filtration rate (eGFR) as calculated from the Bedside Schwartz equation (in units of mL/min/1.73 m2) or via radioisotope GFR of ≥ 70 mL/min/1.73 m2. The Bedside Schwartz equation is: \[(0.413) X (Height in cm)\] / SCr
For participants ≥17 years old: estimated Glomerular Filtration rate (eGFR) as calculated from the Cockcroft and Gault formula (in units of mL/min/1.73 m2) or via radioisotope GFR of ≥ 70 mL/min/1.73 m2. The Cockcroft and Gault formula is: \[(140-age) x (Wt in kg) x (0.85 if female)\] / (72 x SCr)
OR a 24 hour urine Creatinine clearance ≥ 70 mL/min/1.73 m2 5. Adequate liver function defined as:
Total bilirubin ≤1.5 x upper limit of normal (ULN) for age, and
SGPT (ALT \<3 x upper limit of normal (ULN) for age (except for participants with liver metastasis who may enroll if ALT \< 5 times ULN for age). 6. Adequate cardiac function defined as:
Shortening fraction of ≥27% or
Ejection fraction of ≥50% 7. Participants must have fully recovered from the hematological and bone marrow suppression effects of prior chemotherapy. 8. Participants must have a Lansky Play Scale or Karnofsky Performance Scale score of ≥ 60. 9. Participants of childbearing potential must have a negative pregnancy test and agree to use an effective birth control method. Participants who are lactating must agree to stop breast-feeding. 10. Written informed consent in accordance with institutional and FDA guidelines must be obtained from all participants (or participants' legal representative).
Age
Surgical resection of all possible sites of suspected pulmonary metastases in order to achieve a complete remission within 4 weeks prior to study enrollment\*
Pathologic confirmation of metastases from at least one of the resected sites.
No local recurrence or metastatic disease elsewhere. \*For participants with bilateral pulmonary metastases, resection must be performed from both lungs and the study enrollment must be within 4 weeks from date of the last lung surgery. No evidence of pulmonary metastatic disease; participants may have no visible lung nodules greater than 3 mm, and not considered to be disease.
Myelosuppressive anti-cancer therapy: Must not have been received within 2 weeks of study entry (4 weeks if prior nitrosourea).
Biologic (anti-neoplastic agent): At least 7 days since the completion of therapy with a biologic agent.
Radiation therapy (RT): ≥2 weeks for local palliative RT (small port); ≥6 weeks must have elapsed if prior craniospinal RT or if ≥50% radiation of pelvis; ≥6 weeks must have elapsed if other substantial BM radiation.
Surgery: ≥2 weeks from last major surgery, including pulmonary metastasectomy, with the exclusion of a central line placement and core needle or small open biopsies.
Platelet count ≥50,000/μL without transfusion in last 7 days
Hgb ≥8.5 without transfusion in last 7 days 7. Adequate renal function defined as:
For participants \< 17 years old: estimated Glomerular Filtration rate (eGFR) as calculated from the Bedside Schwartz equation (in units of mL/min/1.73 m2) or via radioisotope GFR of ≥ 70 mL/min/1.73 m2. The Bedside Schwartz equation is: \[(0.413) X (Height in cm)\] / SCr
For participants ≥17 years old: estimated Glomerular Filtration rate (eGFR) as calculated from the Cockcroft and Gault formula (in units of mL/min/1.73 m2) or via radioisotope GFR of ≥ 70 mL/min/1.73 m2. The Cockcroft and Gault formula is: \[(140-age) x (Wt in kg) x (0.85 if female)\] / (72 x SCr)
OR a 24 hour urine Creatinine clearance ≥ 70 mL/min/1.73 m2 8. Adequate liver function defined as:
Total bilirubin ≤1.5 x upper limit of normal (ULN) for age.
SGPT (ALT \<3 x upper limit of normal (ULN) for age. 9. Adequate cardiac function defined as:
Shortening fraction of ≥27%, or
Ejection fraction of ≥50%. 10. Adequate pulmonary function defined as:
For participants \< 17 years old: estimated Glomerular Filtration rate (eGFR) as calculated from the Bedside Schwartz equation (in units of mL/min/1.73 m2) or via radioisotope GFR of ≥ 70 mL/min/1.73 m2. The Bedside Schwartz equation is: \[(0.413) X (Height in cm)\] / SCr
For participants ≥17 years old: estimated Glomerular Filtration rate (eGFR) as calculated from the Cockcroft and Gault formula (in units of mL/min/1.73 m2) or via radioisotope GFR of ≥ 70 mL/min/1.73 m2. The Cockcroft and Gault formula is: \[(140-age) x (Wt in kg) x (0.85 if female)\] / (72 x SCr)
OR a 24 hour urine Creatinine clearance ≥ 70 mL/min/1.73 m2 12. Adequate cardiac function defined as:
Shortening fraction of ≥28%, or
Ejection fraction of ≥50% 13. Adequate liver function defined as:
Total bilirubin ≤1.5 x upper limit of normal (ULN) for age
SGPT (ALT \<3 x upper limit of normal (ULN) for age. 14. Participants must have fully recovered from the hematological and bone marrow suppression effects of prior chemotherapy. 15. Participants of childbearing potential must have a negative pregnancy test and agree to use an effective birth control method. Participants who are lactating must agree to stop breast-feeding. 16. Written informed consent in accordance with institutional and FDA guidelines must be obtained from all participants (or participants' legal representative).
  • Number of Cohort 1 participants with relapse free survival (RFS) during study2 years plus 5 years follow up

    Cohort 1: To determine if relapse-free survival (RFS) in participants with relapsed or refractory Ewing sarcoma treated with multiagent chemotherapy is improved with the addition of DFMO as compared to historical outcomes of participants treated with the same multiagent chemotherapy without DFMO.

  • Number of Cohort 2 participants with event-free survival (EFS) during study2 years plus 5 years follow up

    Cohort 2: To determine if event-free survival (EFS) in participants with newly diagnosed metastatic Ewing sarcoma treated with multiagent chemotherapy is improved with the addition of DFMO as compared to historical outcomes of participants treated with the same multiagent chemotherapy without DFMO.

  • Cohort 3: Number of Cohort 3 participants at 12 months with disease control1 year plus 5 years follow up

    To determine the 12-month disease control rate (DCR) in participants with completely resected recurrent osteosarcoma treated with DFMO as compared to historical controls.

  • Cohort 4A: Number of Cohort 4A participants with event-free survival (EFS) during study2 years plus 5 years follow up

    Examine whether the addition of DFMO to post-operative chemotherapy with cisplatin, doxorubicin, and methotrexate (MAP) improves the event-free survival (EFS) for participants with osteosarcoma having localized disease with a poor histological response to 10 weeks of pre-operative chemotherapy.

  • Cohort 4B: Number of Cohort 4B participants with event-free survival (EFS) during study2 years plus 5 years follow up

    Examine whether the addition of DFMO to post-operative chemotherapy with cisplatin, doxorubicin, and methotrexate (MAP) improves the event-free survival (EFS) for participants with osteosarcoma with metastatic disease at diagnosis (Cohort 4B).