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

Phase 1

Recruiting

• Conditions: Osteosarcoma Recurrent; Ewing's Tumor Recurrent
• Interventions: Drug: DFMO

• Registration Number: NCT06892678
• Lead Sponsor: Montefiore Medical Center

Brief 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.

Detailed 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.

Study Timeline

• Study First Submitted: 2025-03-19
• Study First Submitted QC: 2025-03-19
• Study First Posted: 2025-03-25
• Study Start: 2025-04-07
• Status Verified: 2025-05
• Last Update Submitted: 2025-05-06
• Last Update Posted: 2025-05-11
• Primary Completion: 2030-04
• Study Completion: 2030-04

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 15

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Treatment with DFMO	DFMO	DFMO will be administered orally every 12 hours in 28-day cycles at the FDA approved dosages based on the patient's body surface area (BSA). DFMO tablets are 192 mg. * Patients with a BSA \< 1.5 m2 will take 768 mg (four tablets) orally twice a day. * Patients with a BSA 0.75 to 1.5 m2 will take 576 mg (three tablets) orally twice a day. * Patients with a BSA of 0.5 to \< 0.75 m2 will take 384 mg (two tablets) orally twice a day. * Patients with a BSA of 0.25 to \< 0.5 m2 will take 192 mg (one tablet) orally twice a day.

Primary Outcome Measures

Name	Time	Method
Feasibility of Administering DFMO	Up to 2 years	Feasibility will be defined as the ability to successfully administer DFMO to at least 80% of subjects who initiate therapy until either disease recurrence or completion of the maximally allowed duration of therapy. Results will be summarized using basic descriptive statistics.

Secondary Outcome Measures

Name	Time	Method
Event Free Survival	Up to 2 years	The number/percentage of participants with event-free survival (EFS) will be determined. Event-free survival will be defined as the time from diagnosis until drug discontinuation, disease progression, recurrence at any site, secondary malignancy, death from any cause, or last follow-up, whichever is observed first.

Trial Locations

Locations (1)

Montefiore Medical Center; 🇺🇸 Bronx, New York, United States