Eflornithine (DFMO) and Etoposide for Relapsed/Refractory Neuroblastoma
Phase II Trial of Eflornithine (DFMO) and Etoposide for Relapsed/Refractory Neuroblastoma
1 other identifier
interventional
131
2 countries
31
Brief Summary
Difluoromethylornithine (DFMO) will be used in an open label, multicenter, study in combination with etoposide for subjects with relapsed/refractory neuroblastoma.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Sep 2020
Longer than P75 for phase_2
31 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
March 6, 2020
CompletedFirst Posted
Study publicly available on registry
March 10, 2020
CompletedStudy Start
First participant enrolled
September 25, 2020
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 1, 2028
ExpectedStudy Completion
Last participant's last visit for all outcomes
October 1, 2033
July 8, 2026
July 1, 2026
8 years
March 6, 2020
July 7, 2026
Conditions
Outcome Measures
Primary Outcomes (1)
Number of participants with event free survival (EFS) during study
To evaluate the efficacy of difluoromethylornithine (DFMO) in combination with etoposide in patients with relapsed/refractory neuroblastoma, based upon: o Event free survival (EFS) from time of enrollment.
2 years plus 5 years follow up
Secondary Outcomes (3)
Length of time that participants experience Overall Survival (OS)
7 years
Determine the Overall Response Rate (ORR) of Participants using INSS Response Evaluation Criteria.
2 years
Number of Participants with Adverse Events as a Measure of Safety and Tolerability
2 years plus 30 days
Study Arms (1)
Eflornithine (DFMO)
EXPERIMENTALIn this study subjects will receive six 21-day cycles of Etoposide and DFMO followed by an additional 630 days of DFMO alone. Etoposide will be given at 50 mg/m2/dose PO daily for the first 14 days of each 21 days until 6 cycles of etoposide are completed. DFMO (difluoromethylornithine) will be given at a dose of 1000 mg/m2 BID on each day of study.
Interventions
DFMO (difluoromethylornithine) will be given at a dose of 1000 mg/m2 BID on each day of study.
Eligibility Criteria
You may qualify if:
- All patients must have a pathologically confirmed diagnosis of neuroblastoma, ≤ 30.99 years of age with history of relapsed/refractory neuroblastoma.
- All patients must have completed upfront therapy with at least 4 cycles of aggressive multi-drug chemotherapy.
- Specific Criteria by Arm:
- Arms 1 and 2:
- Subjects with no active disease:
- i. No evidence of residual disease by CT/MRI and MIBG scan (or PET for patients who have a history of MIBG non-avid disease).
- o Note: Patients with residual masses detected by CT/MRI may be considered in CR if their MIBG is negative or if MIBG positive and evaluated by PET and found to have negative PET scans; biopsy confirmation may be considered if there is still reasonable concern for persistent disease but is not required.
- ii. No evidence of disease metastatic to bone marrow.
- Arm 3 \[CLOSED TO ENROLLMENT\]:
- Measurable or evaluable disease, including at least one of the following:
- Measurable tumor by CT or MRI; or a positive MIBG and PET; or positive bone marrow biopsy/aspirate in at least one site.
- Timing from prior therapy: Enrollment (first dose of DFMO) no later than 60 days from last dose of the most recent therapy.
- Subjects must have fully recovered from the acute toxic effects of all prior anti- cancer chemotherapy and be within the following timelines:
- Myelosuppressive chemotherapy: Must not have received within 2 weeks of enrollment onto this study (6 weeks if prior nitrosourea).
- Hematopoietic growth factors: At least 5 days since the completion of therapy with a growth factor.
- +18 more criteria
You may not qualify if:
- BSA of \<0.25 m2.
- Subjects that received DFMO at a dose higher than 1000mg/m2 BID prior to this study are not eligible.
- Subjects that received a dose of DFMO in combination with etoposide are not eligible.
- Investigational Drugs: Subjects who are currently receiving another investigational drug are excluded from participation.
- Anti-cancer Agents: Subjects who are currently receiving other anticancer agents are not eligible. Subjects must have fully recovered from hematological and bone marrow suppression effects of prior chemotherapy.
- Infection: Subjects who have an uncontrolled infection are not eligible until the infection is judged to be well controlled in the opinion of the investigator.
- Subjects who, in the opinion of the investigator, may not be able to comply with the safety monitoring requirements of the study, or in whom compliance is likely to be suboptimal, should be excluded.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- Beat NB Cancer Foundationcollaborator
- Giselle Shollerlead
- K C Pharmaceuticals Inc.collaborator
- Team Parker for Lifecollaborator
- USWM, LLCcollaborator
Study Sites (31)
University of Alabama/Children's of Alabama
Birmingham, Alabama, 35201, United States
Arkansas Children's Hospital
Little Rock, Arkansas, 72202, United States
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609, United States
Rady Children's Hospital
San Diego, California, 92123, United States
Connecticut Children's Hospital
Hartford, Connecticut, 06106, United States
Arnold Palmer Hospital for Children
Orlando, Florida, 32806, United States
St. Joseph's Children's Hospital
Tampa, Florida, 33614, United States
Augusta University Health
Augusta, Georgia, 30912, United States
University of Iowa
Iowa City, Iowa, 52242, United States
Norton Children's Research Institute/Affiliated with University of Louisville School of Medicine
Louisville, Kentucky, 40201, United States
Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503, United States
Children's Hospital and Clinics of Minnesota
Minneapolis, Minnesota, 55404, United States
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, 64108, United States
Cardinal Glennon Children's Hospital
St Louis, Missouri, 63104, United States
Hackensack University Medical Center
Hackensack, New Jersey, 07601, United States
Levine Children's Hospital
Charlotte, North Carolina, 28204, United States
Cleveland Clinic Children's
Cleveland, Ohio, 44195, United States
Randall Children's Hospital
Portland, Oregon, 97227, United States
Penn State Milton S. Hershey Medical Center and Children's Hospital
Hershey, Pennsylvania, 17033, United States
Hasbro Children's Hospital
Providence, Rhode Island, 02901, United States
Medical University of South Carolina
Charleston, South Carolina, 29425, United States
Dell Children's Blood and Cancer Center
Austin, Texas, 78723, United States
Children's Medical Center Dallas
Dallas, Texas, 75235, United States
Children's Hospital of The King's Daughters
Norfolk, Virginia, 23507, United States
Medical College of Wisconsin
Milwaukee, Wisconsin, 53226, United States
Alberta Children's Hospital
Calgary, Alberta, AB T3B 6A8, Canada
CancerCare Manitoba
Winnipeg, Manitoba, MB R3E 0V9, Canada
UHC Sainte-Justine
Montreal, Quebec, QC H3S 2G4, Canada
Montreal Children's Hospital
Montreal, Quebec, QC H4A 3H9, Canada
CHUQ
Québec, Quebec, QC G1V 4W6, Canada
CIUSSS de l'Estrie-CHUS
Sherbrooke, Quebec, QC J1H 5H3, Canada
Related Links
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Study Officials
- STUDY CHAIR
Giselle Sholler, MD
Beat Childhood Cancer
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- SPONSOR INVESTIGATOR
- PI Title
- Chair, Beat Childhood Cancer
- Expanded Access
- Yes
Study Record Dates
First Submitted
March 6, 2020
First Posted
March 10, 2020
Study Start
September 25, 2020
Primary Completion (Estimated)
October 1, 2028
Study Completion (Estimated)
October 1, 2033
Last Updated
July 8, 2026
Record last verified: 2026-07
Data Sharing
- IPD Sharing
- Will not share