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

Montefiore Medicine Academic Health System

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Ewing's Tumor Recurrent; Osteosarcoma Recurrent

Treatments: Drug: DFMO

Study type: Interventional

Funder types: Other

Identifiers

NCT06892678

2024-16461

Details and patient eligibility

About

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.

Full 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.

Enrollment: 15 estimated patients

Sex: All

Ages: Under 39 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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15 participants in 1 patient group

Treatment with DFMO

Experimental group

Description:

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.

Treatment:

Drug: DFMO