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

NCT ID: NCT06892678

Last Updated: 2025-05-11

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-04-07
• Study Completion Date: 2030-04-30

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.

Conditions

Osteosarcoma Recurrent; Ewing's Tumor Recurrent

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Treatment with 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.

Group Type: EXPERIMENTAL

DFMO

Intervention Type: DRUG

DFMO dose will be calculated based on the BSA measured within 14 days prior to the beginning of each cycle. Tablets may be swallowed whole, chewed, or crushed and mixed with soft food or liquid.

Interventions

DFMO

DFMO dose will be calculated based on the BSA measured within 14 days prior to the beginning of each cycle. Tablets may be swallowed whole, chewed, or crushed and mixed with soft food or liquid.

Intervention Type: DRUG

Other Intervention Names

Difluoromethylornithine; iWilfin

Eligibility Criteria

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

• Maximum Eligible Age: 39 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Johns Hopkins University

OTHER

Sponsor Role: collaborator

Montefiore Medical Center

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Alice Lee, MD

Role: PRINCIPAL_INVESTIGATOR

Montefiore Medical Center

Locations

Montefiore Medical Center

The Bronx, New York, United States

Site Status: RECRUITING

Countries

United States

Central Contacts

Rebecca Zylber, MSN

Role: CONTACT

rzylber@montefiore.org

718-741-2356

Lara Fabish, MSN

Role: CONTACT

lfabish@montefiore.org

718-741-2356

Facility Contacts

Rebecca Zylber

Role: primary

rzylber@montefiore.org

718-741-2356

Lara Fabish

Role: backup

lfabish@montefiore.org

References

Collier AB 3rd, Krailo MD, Dang HM, DuBois SG, Hawkins DS, Bernstein ML, Bomgaars LR, Reed DR, Gorlick RG, Janeway KA. Outcome of patients with relapsed or progressive Ewing sarcoma enrolled on cooperative group phase 2 clinical trials: A report from the Children's Oncology Group. Pediatr Blood Cancer. 2021 Dec;68(12):e29333. doi: 10.1002/pbc.29333. Epub 2021 Sep 8.

Reference Type: BACKGROUND

PMID: 34496122 (View on PubMed)

Lagmay JP, Krailo MD, Dang H, Kim A, Hawkins DS, Beaty O 3rd, Widemann BC, Zwerdling T, Bomgaars L, Langevin AM, Grier HE, Weigel B, Blaney SM, Gorlick R, Janeway KA. Outcome of Patients With Recurrent Osteosarcoma Enrolled in Seven Phase II Trials Through Children's Cancer Group, Pediatric Oncology Group, and Children's Oncology Group: Learning From the Past to Move Forward. J Clin Oncol. 2016 Sep 1;34(25):3031-8. doi: 10.1200/JCO.2015.65.5381. Epub 2016 Jul 11.

Reference Type: BACKGROUND

PMID: 27400942 (View on PubMed)

Other Identifiers

2024-16461

Identifier Type: -

Identifier Source: org_study_id