Health TrialsbyHealthPanel

Eflornithine (DFMO) for Ewing Sarcoma and Osteosarcoma

ConditionsOsteosarcomaEwing Sarcoma
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age0-50
SponsorMilton S. Hershey Medical Center

About this trial

Ewing sarcoma (EWS) and osteosarcoma primarily affect adolescents and young adults. Common treatments include chemotherapy, surgery and radiation, however, there have been few recent advancements in the standard of care. By incorporating eflornithine (DFMO) as an additional therapy and/or maintenance therapy we hope to safely observe improved event-free survival and overall survival. There are 5 cohorts covered under this master protocol.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Participants must be ≤50 years of age at enrollment.

Histologically confirmed Ewing sarcoma that is refractory or in first or subsequent relapse. Histological confirmation either at initial diagnosis or disease progression.

Relapsed: Participants that have achieved CR at any point and then relapsed following/during standard of care therapy.

Refractory: Participants that failed to achieve CR after standard of care therapy or having progressed during standard of care therapy.

Disqualifiers

BSA of <0.25 m2

Participants with current CNS disease.

Investigational Drugs: Participants who are currently receiving another investigational drug are excluded from participation.

Anti-cancer Agents: Participants who are currently receiving other anticancer agents are not eligible.

Trial design

Design model

Parallel

Treatments tested in this trial

  • Eflornithine

    Drug

    Oral twice daily

  • Topotecan

    Drug

    IV

  • Cyclophosphamide

    Drug

    IV

  • Vincristine

    Drug

    IV

  • Doxorubicin

    Drug

    IV

  • Ifosfamide

    Drug

    IV

  • Etoposide

    Drug

    IV

  • Cisplatin

    Drug

    IV

  • Methotrexate

    Drug

    IV

Treatment groups

406 Participants
are divided into 5 treatment groups
Group A: Cohort 1: Relapsed or Refractory Ewing Sarcoma Eligible to Receive Local ControlExperimental treatment 3 interventions
Group B: Cohort 2: Ewing Sarcoma Patients who are Metastatic at DiagnosisExperimental treatment 6 interventions
Group C: Cohort 3: Osteosarcoma with relapse in the lung after resection of lung metastasesExperimental treatment 1 intervention
Group D: Cohort 4A: Osteosarcoma with Poor Response to Induction Therapy at Completion of Local ControlExperimental treatment 4 interventions
Group E: Cohort 4B: Osteosarcoma with Metastatic Disease at Diagnosis at Completion of Local ControlExperimental treatment 4 interventions

Trial outcomes

Primary outcomes

1

Number of Cohort 1 participants with relapse free survival (RFS) during study

Cohort 1: To determine if relapse-free survival (RFS) in participants with relapsed or refractory Ewing sarcoma treated with multiagent chemotherapy is improved with the addition of DFMO as compared to historical outcomes of participants treated with the same multiagent chemotherapy without DFMO.

Time frame
2 years plus 5 years follow up
2

Number of Cohort 2 participants with event-free survival (EFS) during study

Cohort 2: To determine if event-free survival (EFS) in participants with newly diagnosed metastatic Ewing sarcoma treated with multiagent chemotherapy is improved with the addition of DFMO as compared to historical outcomes of participants treated with the same multiagent chemotherapy without DFMO.

Time frame
2 years plus 5 years follow up
3

Cohort 3: Number of Cohort 3 participants at 12 months with disease control

To determine the 12-month disease control rate (DCR) in participants with completely resected recurrent osteosarcoma treated with DFMO as compared to historical controls.

Time frame
1 year plus 5 years follow up
4

Cohort 4A: Number of Cohort 4A participants with event-free survival (EFS) during study

Examine whether the addition of DFMO to post-operative chemotherapy with cisplatin, doxorubicin, and methotrexate (MAP) improves the event-free survival (EFS) for participants with osteosarcoma having localized disease with a poor histological response to 10 weeks of pre-operative chemotherapy.

Time frame
2 years plus 5 years follow up

Secondary outcomes

1

Length of time that participants experience Overall Survival (OS)

To compare overall survival in DFMO-treated participants versus historical controls.

Time frame
10 Years
2

Number of Participants with Adverse Events as a Measure of Safety and Tolerability

To characterize safety and tolerability of DFMO plus multiagent chemotherapy.

Time frame
2 years

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Milton S. Hershey Medical Center

Lead sponsor

USWM, LLC (dba US WorldMeds)

Collaborator

Source ClinicalTrials.gov
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