Eflornithine (DFMO) and Etoposide for Relapsed/Refractory Neuroblastoma

Running, not enrolling · Phase 2

Conditions studied: Neuroblastoma

In brief

Difluoromethylornithine (DFMO) will be used in an open label, multicenter, study in combination with etoposide for subjects with relapsed/refractory neuroblastoma.

Key facts

Study ID: NCT04301843
Run by: Giselle Sholler
People needed: 131
Starts: 2020-09-25
Expected to finish: 2033-10-01
Last updated by the study team: 2026-07-08

Who can join

Age: any, up to 31. Sex: any. Healthy volunteers: not accepted.

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.
Biologic (anti-neoplastic agent): At least 7 days since the completion of therapy with a biologic agent. For agents that have known adverse events occurring beyond 7 days after administration, this period must be extended beyond the time during which adverse events are known to occur. The duration of this interval must be discussed with the Study Chair.
Immunotherapy: At least 6 weeks since the completion of any type of immunotherapy, e.g. tumor vaccines, CAR-T cells.
Anti-GD2 Monoclonal antibodies: At least 2 weeks must have elapsed since prior treatment with a monoclonal antibody.
XRT: At least 14 days since the last treatment except for radiation delivered with palliative intent to a non-target site.
Stem Cell Transplant:
Allogeneic: No evidence of active graft vs. host disease
Allo/Auto: ≥ 2 months must have elapsed since transplant.
MIBG Therapy: At least 8 weeks since treatment with MIBG therapy
Subjects must have a Lansky or Karnofsky Performance Scale score of 60% or higher.
Life expectancy > 2 months

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.

Where it is running

University of Alabama/Children's of Alabama — Birmingham, Alabama, United States
Arkansas Children's Hospital — Little Rock, Arkansas, United States
UCSF Benioff Children's Hospital Oakland — Oakland, California, United States
Rady Children's Hospital — San Diego, California, United States
Connecticut Children's Hospital — Hartford, Connecticut, United States
Arnold Palmer Hospital for Children — Orlando, Florida, United States
St. Joseph's Children's Hospital — Tampa, Florida, United States
Augusta University Health — Augusta, Georgia, United States
University of Iowa — Iowa City, Iowa, United States
Norton Children's Research Institute/Affiliated with University of Louisville School of Medicine — Louisville, Kentucky, United States
Helen DeVos Children's Hospital — Grand Rapids, Michigan, United States
Children's Hospital and Clinics of Minnesota — Minneapolis, Minnesota, United States
Children's Mercy Hospitals and Clinics — Kansas City, Missouri, United States
Cardinal Glennon Children's Hospital — St Louis, Missouri, United States
Hackensack University Medical Center — Hackensack, New Jersey, United States
Levine Children's Hospital — Charlotte, North Carolina, United States
Cleveland Clinic Children's — Cleveland, Ohio, United States
Randall Children's Hospital — Portland, Oregon, United States
Penn State Milton S. Hershey Medical Center and Children's Hospital — Hershey, Pennsylvania, United States
Hasbro Children's Hospital — Providence, Rhode Island, United States
Medical University of South Carolina — Charleston, South Carolina, United States
Dell Children's Blood and Cancer Center — Austin, Texas, United States
Children's Medical Center Dallas — Dallas, Texas, United States
Children's Hospital of The King's Daughters — Norfolk, Virginia, United States
Medical College of Wisconsin — Milwaukee, Wisconsin, United States

Full record on ClinicalTrials.gov

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