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

Last updated: May 6, 2025
Sponsor: Montefiore Medical Center
Overall Status: Active - Recruiting

Phase

1/2

Condition

Osteosarcoma

Treatment

DFMO

Clinical Study ID

NCT06892678
2024-16461
  • Ages < 39
  • All Genders

Study Summary

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.

Eligibility Criteria

Inclusion

Inclusion Criteria:

  • Patients < 40 years of age at the time of enrollment

  • Diagnosis of relapsed osteosarcoma or relapsed Ewing sarcoma who have completed allplanned therapy for their relapse, as described in the protocol, and have noevidence of disease

  • Patients must have a performance status corresponding to Eastern CooperativeOncology 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

Exclusion Criteria:

  • Pregnant or breastfeeding females

  • Patients must not have an uncontrolled infection

  • Patients must not have any significant intercurrent illness

Study Design

Total Participants: 15
Treatment Group(s): 1
Primary Treatment: DFMO
Phase: 1/2
Study Start date:
April 07, 2025
Estimated Completion Date:
April 30, 2030

Study 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.

Connect with a study center

  • Montefiore Medical Center

    Bronx, New York 10467
    United States

    Active - Recruiting

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