Osteosarcoma RecurrentPhase 1/2DFMONCT06892678
DFMO Maintenance for Patients With Relapsed/Refractory Ewing Sarcoma or Osteosarcoma
A Phase 1/2 study testing DFMO in people with Osteosarcoma Recurrent and Ewing's Tumor Recurrent, run by Montefiore Medical Center at 1 site in United States.
Trial summary
The purpose of this study is to determine the feasibility of administering DL-alpha-difluoromethylornithine (DFMO) to patients with relapsed Ewing sarcoma and osteosarcoma who have completed all planned therapy and have no evidence of disease. Read more about this study
- Who is it for?
- Ages Up to 39 years
- Conditions the study is for
- Osteosarcoma Recurrent, Ewing's Tumor Recurrent
- What is being tested?
- DFMO
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 15 participants
- Where
- 1 siteUnited States
- Study dates
- 7 Apr 2025to Apr 2030
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 39 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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
- Myelosuppressive chemotherapy: At least 14 days must have elapsed since completion of myelosuppressive therapy
- Monoclonal antibodies: At least 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to Grade < 2
- Biologic therapy (defined as anti-cancer agents not known to be myelosuppressive): At least 7 days after the last dose of agent
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- Radiation therapy: At least 14 days must have elapsed after local External Beam Radiation Therapy (XRT), at least 90 days after Total Body Irradiation (TBI), craniospinal XRT or if radiation to greater than 50% of the pelvis, and at least 42 days if other substantial bone marrow radiation
- Adequate bone marrow function defined as:
- Peripheral absolute neutrophil count (ANC) greater or equal to 750/microliter
- Platelet count greater or equal to 75,000/microliter (transfusion independent)
- Adequate renal function defined by serum creatinine based on age and gender (see protocol)
- 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
Who cannot join
- Pregnant or breastfeeding females. Men and women of childbearing potential and their partners must agree to use adequate contraception while enrolled on this study. Based on the teratogenic potential of the agent, pregnant women will be excluded from this study. Because of potential risks to breastfed infants due to drug metabolites that could be excreted in breast milk, female patients who are lactating must agree to stop breastfeeding or will otherwise be excluded from this study. Females of childbearing potential must have a negative pregnancy test to be eligible for this study
- Patients must not have an uncontrolled infection
- Patients with a significant intercurrent illness (any ongoing serious medical problem unrelated to cancer or its treatment) that is not covered by the detailed exclusion criteria and that is expected to interfere with the action of study agents or to significantly increase the severity of the toxicities experienced from study treatment are not eligible
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Montefiore Medical Center
The Bronx, New York, 10467
Rebecca Zylber, 718-741-2356, rzylber@montefiore.org
Lara Fabish, lfabish@montefiore.org
About this study
The purpose of this study is to determine the feasibility of administering DL-alpha-difluoromethylornithine (DFMO) to patients with relapsed Ewing sarcoma and osteosarcoma who have completed all planned therapy and have no evidence of disease.
Read the 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
- Being tested
- DFMO
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
DFMODrug
Also called Difluoromethylornithine, iWilfin
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.
| Study group | Role | What they receive |
|---|---|---|
| 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 m^2 will take 768 mg (four tablets) orally twice a day.
* Patients with a BSA 0.75 to 1.5 m^2 will take 576 mg (three tablets) orally twice a day.
* Patients with a BSA of 0.5 to < 0.75 m^2 will take 384 mg (two tablets) orally twice a day.
* Patients with a BSA of 0.25 to < 0.5 m^2 will take 192 mg (one tablet) orally twice a day. Receives: Drug: DFMO |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Up to 2 years
Secondary outcomes (1)
Event Free Survival
The number/percentage of participants with event-free survival (EFS) at 2 years 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.
Measured: Up to 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Mar 2025
- Study start7 Apr 2025
- Primary completionApr 2030 (estimated)
- Study completionApr 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (2)Papers the sponsor has linked to this study.
- Other trials for Osteosarcoma RecurrentStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 15 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Montefiore Medical Center
- Sponsor type
- University, hospital or other
- Collaborators
- Johns Hopkins University
- Responsible party
- Sponsor
- Investigators
- Alice Lee, MD, Principal investigator, Montefiore Medical Center
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06892678
- Sponsor's protocol ID
- 2024-16461
- Acronym
- DFMO
- Status last verified
- May 2026
- First submitted
- 19 Mar 2025
- Last updated
- 14 May 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
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.
PubMed 34496122Lagmay 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.
PubMed 27400942
Study at a glance
From the registry record, last updated by the sponsor on 14 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- DFMO Maintenance for Patients With Relapsed/Refractory Ewing Sarcoma or Osteosarcoma
- ClinicalTrials.gov ID
- NCT06892678
- Acronym / sponsor ID
- DFMO / 2024-16461
- Conditions
- Osteosarcoma Recurrent, Ewing's Tumor Recurrent
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 15 participants (estimated)
- Lead sponsor
- Montefiore Medical CenterUniversity, hospital or other
- Collaborators
- Johns Hopkins University
- Study start
- 7 Apr 2025 (actual)
- Primary completion
- Apr 2030 (estimated)
- Study completion
- Apr 2030 (estimated)
- First posted
- 25 Mar 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The purpose of this study is to determine the feasibility of administering DL-alpha-difluoromethylornithine (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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Treatment with DFMO | Experimental | Drug: DFMODFMO 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 m^2 will take 768 mg (four tablets) orally twice a day. * Patients with a BSA 0.75 to 1.5 m^2 will take 576 mg (three tablets) orally twice a day. * Patients with a BSA of 0.5 to < 0.75 m^2 will take 384 mg (two tablets) orally twice a day. * Patients with a BSA of 0.25 to < 0.5 m^2 will take 192 mg (one tablet) orally twice a day. |
Who may qualify
- Ages
- Up to 39 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (14 listed)
- 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
- Myelosuppressive chemotherapy: At least 14 days must have elapsed since completion of myelosuppressive therapy
- Monoclonal antibodies: At least 21 days must have elapsed from infusion of last dose of antibody, and toxicity related to prior antibody therapy must be recovered to Grade < 2
Key exclusion criteria (3 listed)
- Pregnant or breastfeeding females. Men and women of childbearing potential and their partners must agree to use adequate contraception while enrolled on this study. Based on the teratogenic potential of the agent, pregnant women will be excluded from this study. Because of potential risks to breastfed infants due to drug metabolites that could be excreted in breast milk, female patients who are lactating must agree to stop breastfeeding or will otherwise be excluded from this study. Females of childbearing potential must have a negative pregnancy test to be eligible for this study
- Patients must not have an uncontrolled infection
- Patients with a significant intercurrent illness (any ongoing serious medical problem unrelated to cancer or its treatment) that is not covered by the detailed exclusion criteria and that is expected to interfere with the action of study agents or to significantly increase the severity of the toxicities experienced from study treatment are not eligible
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| DFMOAlso: Difluoromethylornithine, iWilfinDFMO 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. | Drug | Treatment with DFMO |
Primary outcome
Feasibility of Administering DFMO
Time frame: 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.
1 secondary outcome
Event Free Survival
Time frame: Up to 2 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 May 2026.
- Background: PubMed 34496122Collier 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.
- Background: PubMed 27400942Lagmay 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Alice Lee, MD principal investigator
Montefiore Medical Center
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing DFMO in people with Osteosarcoma Recurrent and Ewing's Tumor Recurrent, run by Montefiore Medical Center at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 14 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 14 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Rebecca Zylber, MSN (718-741-2356, rzylber@montefiore.org), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Feasibility of Administering DFMO (time frame: Up to 2 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06892678, which also lists 2 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 May 2026; AzTrials last refreshed its copy on 30 Oct 2026.