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DFMO Maintenance for Patients With Relapsed/Refractory Ewing Sarcoma or Osteosarcoma

DFMO Maintenance for Patients With Relapsed/Refractory Ewing Sarcoma or Osteosarcoma

Recruiting
39 years and younger
All
Phase 1/2

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Overview

The purpose of this study is to determine the feasibility of administering DFMO to patients with relapsed Ewing sarcoma and osteosarcoma who have completed all planned therapy and have no evidence of disease.

Description

Approximately 30-35% of patients diagnosed with osteosarcoma or Ewing sarcoma will develop relapsed/refractory disease and carry a very poor prognosis. DL-alpha-difluoromethylornithine, commonly known as DFMO or eflornithine, is a synthetic analog of the amino acid ornithine. DFMO has been studied in a number of different cancers as either a therapeutic or a chemopreventative agent and is now FDA approved to reduce the risk of relapse in patients with newly diagnosed high-risk neuroblastoma. As DFMO has now been given to over 100 children with metastatic cancer, dosing and safety in this population is well established. Given the stagnant survival rates for children, adolescents, and young adults with relapsed Ewing sarcoma and osteosarcoma over the past few decades, this study will explore the feasibility of using DFMO in patients with relapsed osteosarcoma and relapsed Ewing sarcoma who are without any evidence of disease at the end of therapy in order to prevent disease recurrence.

Eligibility

Inclusion Criteria:

  • Patients < 40 years of age at the time of enrollment
  • Diagnosis of relapsed osteosarcoma or relapsed Ewing sarcoma who have completed all planned therapy for their relapse, as described in the protocol, and have no evidence of disease
  • Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1, or 2
  • Adequate bone marrow function defined as:
  • Peripheral absolute neutrophil count (ANC) greater or equal to 750/microliters
  • Platelet count greater or equal to 75,000/microliters (transfusion independent)
  • Adequate renal function defined by serum creatinine based on age and gender
  • Adequate liver function defined as:
  • Total bilirubin ≤ 1.5 x the upper limit of normal (ULN) for age AND
  • SGPT (ALT) ≤ 5.0 x ULN for age. For this study the ULN is 45 U/L

Exclusion Criteria:

  • Pregnant or breastfeeding females
  • Patients must not have an uncontrolled infection
  • Patients must not have any significant intercurrent illness

Study details
    Osteosarcoma Recurrent
    Ewing's Tumor Recurrent

NCT06892678

Montefiore Medical Center

16 October 2025

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