DFMO for Bone Cancer

(DFMO Trial)

Recruiting at 1 trial location

Overseen By Rebecca Zylber, MSN

Age: < 65

Sex: Any

Trial Phase: Phase 1 & 2

Sponsor: Montefiore Medical Center

Disqualifiers: Pregnancy, Uncontrolled infection, Illness, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Compensation: Varies

Number of Visits: Unknown

Duration: Up to 2 years

15 Participants Needed

What You Need to Know Before You Apply

What is the purpose of this trial?

The trial aims to determine if DFMO, an oral medication, is a safe and practical treatment for individuals with Ewing sarcoma or osteosarcoma who have relapsed but currently show no signs of the disease. Participants will take DFMO pills every 12 hours in 28-day cycles. The study seeks individuals under 40 who have completed all planned therapies for their cancer relapse and are now disease-free. As a Phase 1, Phase 2 trial, the research focuses on understanding how DFMO works in people and measuring its effectiveness in an initial, smaller group. Participants have the opportunity to contribute to advancing cancer treatment.

Do I need to stop my current medications to join the trial?

The trial information does not specify whether you need to stop taking your current medications. It's best to discuss this with the trial coordinators or your doctor.

Is there any evidence suggesting that DFMO is likely to be safe for humans?

Research has shown that DFMO has been administered to over 100 children with metastatic cancer, and its dosage and safety are well understood in this group. DFMO is generally well-tolerated. Although DFMO is still under investigation for bone cancer, it is already approved for other uses, suggesting its safety. No major safety issues have been reported. The treatment is being studied to determine if it can help prevent cancer from spreading in conditions like osteosarcoma and Ewing sarcoma. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Researchers are excited about DFMO for bone cancer because it works differently from traditional chemotherapy and radiation treatments. DFMO targets a specific enzyme called ornithine decarboxylase, which is involved in cell growth and cancer progression. This unique mechanism could potentially slow down or stop the growth of cancer cells with fewer side effects compared to conventional treatments. Additionally, DFMO is taken orally, making it a convenient option for patients.

What evidence suggests that DFMO might be an effective treatment for bone cancer?

Research has shown that DFMO, the treatment under study in this trial, may help treat bone cancers such as osteosarcoma and Ewing sarcoma. Studies have found that DFMO can reduce metastasis, the spread of these cancers. In lab tests, DFMO stopped cancer cells from growing and even helped some cells develop normally. Additional research demonstrated that DFMO can significantly slow tumor growth. These findings suggest that DFMO could be a valuable treatment option for patients with these challenging conditions. ^{1 2 4 5 6}

Who Is on the Research Team?

Alice Lee, MD

Principal Investigator

Montefiore Medical Center

Are You a Good Fit for This Trial?

This trial is for patients who have experienced a relapse of Ewing Sarcoma or Osteosarcoma, which are types of bone cancer. Participants should have completed all planned therapy and currently show no signs of the disease.

Inclusion Criteria

I am younger than 40 years old.

I have completed all treatments for my relapsed bone cancer and currently show no signs of the disease.

I can take care of myself but might not be able to do heavy physical work.

Exclusion Criteria

I do not have any infections that aren't responding to treatment.

I have a serious illness besides cancer.

I am not pregnant or breastfeeding.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

DFMO is administered orally every 12 hours in 28-day cycles at FDA approved dosages based on body surface area

Up to 2 years

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• DFMO

Trial Overview The study is testing the feasibility of using DFMO, a drug, as maintenance therapy for those who've had Ewing Sarcoma or Osteosarcoma but are now in remission after completing their initial treatment.

How Is the Trial Designed?

Group I: Treatment with DFMO Experimental Treatment 1 Intervention

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.

Who Is Running the Clinical Trial?

Montefiore Medical Center

Lead Sponsor

Trials

468

Recruited

599,000+

Johns Hopkins University

Collaborator

Trials

2,366

Recruited

15,160,000+

Citations

1. onclive.com onclive.com/view/from-the-lab-to-the-clinic-exploring-dfmo-s-potential-to-prevent-metastatic-disease-in-children-with-osteosarcoma-and-ewing-sarcoma

From the Lab to the Clinic: Exploring DFMO's Potential ... DFMO shows promise in reducing metastasis in osteosarcoma and Ewing sarcoma, with phase 2 trials underway to test its role as maintenance ...

2. clinicaltrials.gov clinicaltrials.gov/study/NCT06892678

Study Details | NCT06892678 | DFMO Maintenance for ... Approximately 30-35% of patients diagnosed with osteosarcoma or Ewing sarcoma will develop relapsed/refractory disease and carry a very poor prognosis. DL-alpha ...

3. pmc.ncbi.nlm.nih.gov pmc.ncbi.nlm.nih.gov/articles/PMC6165283/

Polyamine Biosynthetic Pathway as a Drug Target for ... These results indicate that DFMO treatment results in terminal differentiation of some osteosarcoma cell lines. Remarkably, this occurred ...

4. esmoopen.com esmoopen.com/article/S2059-7029(25)01445-0/fulltext

Phase I dose-escalation trial of AMXT 1501 dicaprate plus ... AMXT 1501 plus DFMO produced an almost complete blockade of tumor growth, with a durable effect after treatment ceased. Evidence from this model ...

5. aacrjournals.org aacrjournals.org/cancerres/article/83/7_Supplement/6725/724423/Abstract-6725-Targeting-urea-cycle-dysfunction-to

Abstract 6725: Targeting urea cycle dysfunction to prevent and ... Conclusion: DFMO inhibits proliferation and clonogenic growth in vitro, and decreases distant metastasis in vivo. Since pediatric dosing and ...

6. fda.gov fda.gov/media/172661/download

eflornithine (dfmo) tablets to reduce the risk of placebo-controlled phase 3 skin cancer prevention study of alpha-difluoromethylornithine in subjects with previous history of skin cancer.