NCT06892678

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.

Trial Health

77
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
15

participants targeted

Target at below P25 for phase_1

Timeline
48mo left

Started Apr 2025

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress22%
Apr 2025Apr 2030

First Submitted

Initial submission to the registry

March 19, 2025

Completed
6 days until next milestone

First Posted

Study publicly available on registry

March 25, 2025

Completed
13 days until next milestone

Study Start

First participant enrolled

April 7, 2025

Completed
5 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2030

Expected
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2030

Last Updated

May 11, 2025

Status Verified

May 1, 2025

Enrollment Period

5 years

First QC Date

March 19, 2025

Last Update Submit

May 6, 2025

Conditions

Osteosarcoma RecurrentEwing's Tumor Recurrent

Outcome Measures

Primary Outcomes (1)

  • Feasibility of Administering DFMO

    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.

    Up to 2 years

Secondary Outcomes (1)

  • Event Free Survival

    Up to 2 years

Study Arms (1)

Treatment with DFMO

EXPERIMENTAL

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.

Drug: DFMO

Interventions

DFMODRUG

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.

Also known as: Difluoromethylornithine, iWilfin
Treatment with DFMO

Eligibility Criteria

AgeUp to 39 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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

You may not qualify if:

  • Pregnant or breastfeeding females
  • Patients must not have an uncontrolled infection
  • Patients must not have any significant intercurrent illness

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Montefiore Medical Center

The Bronx, New York, 10467, United States

RECRUITING

Related Publications (2)

  • 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.

    PMID: 34496122BACKGROUND

  • 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.

    PMID: 27400942BACKGROUND

MeSH Terms

Interventions

Eflornithine

Intervention Hierarchy (Ancestors)

OrnithineAmino Acids, BasicAmino AcidsAmino Acids, Peptides, and ProteinsAmino Acids, Diamino

Study Officials

  • Alice Lee, MD

    Montefiore Medical Center

    PRINCIPAL INVESTIGATOR

Central Study Contacts

Rebecca Zylber, MSN

CONTACT

718-741-2356rzylber@montefiore.org

Lara Fabish, MSN

CONTACT

718-741-2356lfabish@montefiore.org

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 19, 2025

First Posted

March 25, 2025

Study Start

April 7, 2025

Primary Completion (Estimated)

April 1, 2030

Study Completion (Estimated)

April 1, 2030

Last Updated

May 11, 2025

Record last verified: 2025-05

Data Sharing

IPD Sharing
Will not share

Locations

US(1)