Clinical trial NCT06892678: DFMO Maintenance for Patients With Relapsed/Refractory Ewing Sarcoma or Osteosarcoma
Official title: DFMO Maintenance for Patients With Relapsed/Refractory Ewing Sarcoma or Osteosarcoma
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Osteosarcoma Recurrent
Ewing's Tumor Recurrent
Lead sponsor: Montefiore Medical Center
Sponsor class: OTHER
Collaborators: name: Johns Hopkins University
First posted: 2025-03-25
Enrollment: 15
Sex: ALL
Maximum age (years): 39
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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

Primary outcomes:
measure: Feasibility of Administering DFMO
timeFrame: Up to 2 years
Secondary outcomes:
measure: Event Free Survival
timeFrame: Up to 2 years
Other outcomes:
Locations:
city: Bronx
country: United States
facility: Montefiore Medical Center
state: New York
Study officials:
affiliation: Montefiore Medical Center
name: Alice Lee, MD
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.
citation: 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.