Eflornithine (DFMO) and Etoposide for Relapsed/Refractory Neuroblastoma
NCT ID: NCT04301843
Last Updated: 2025-10-14
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE2
131 participants
INTERVENTIONAL
2020-09-25
2033-10-01
Brief Summary
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Detailed Description
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In this study subjects will receive six 21-day cycles of Etoposide and DFMO followed by an additional 630 days of DFMO alone.
Subjects will be evaluated in 3 arms:
• Arm 1: Subjects who show no active disease after receiving any additional therapy for neuroblastoma that was refractory to standard induction/consolidation therapy.
Refractory: Subjects with progressive disease on upfront therapy OR did not have at least PR on induction OR required additional second line therapy to achieve remission who are now in first remission.
* Arm 2: Subjects who have previously relapsed and currently show no active disease (in CR2 or greater).
* Arm 3: Subjects who are relapsed or refractory with active disease.- CLOSED TO ENROLLMENT
Conditions
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Study Design
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NA
SINGLE_GROUP
TREATMENT
NONE
Study Groups
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Eflornithine (DFMO)
In 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.
Eflornithine
DFMO (difluoromethylornithine) will be given at a dose of 1000 mg/m2 BID on each day of study.
Interventions
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Eflornithine
DFMO (difluoromethylornithine) will be given at a dose of 1000 mg/m2 BID on each day of study.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* 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:
1. Myelosuppressive chemotherapy: Must not have received within 2 weeks of enrollment onto this study (6 weeks if prior nitrosourea).
2. Hematopoietic growth factors: At least 5 days since the completion of therapy with a growth factor.
3. 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.
4. Immunotherapy: At least 6 weeks since the completion of any type of immunotherapy, e.g. tumor vaccines, CAR-T cells.
5. Anti-GD2 Monoclonal antibodies: At least 2 weeks must have elapsed since prior treatment with a monoclonal antibody.
6. XRT: At least 14 days since the last treatment except for radiation delivered with palliative intent to a non-target site.
7. Stem Cell Transplant:
1. Allogeneic: No evidence of active graft vs. host disease
2. Allo/Auto: ≥ 2 months must have elapsed since transplant.
8. 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
* All clinical and laboratory studies for organ functions to determine eligibility must be performed within 7 days prior to first dose of study drug unless otherwise indicated below.
* Subjects must have adequate organ functions at the time of registration:
* Hematological: Total absolute neutrophil count ANC ≥750/μL
* Liver: Subjects must have adequate liver function as defined by AST and ALT \<5x upper limit of normal (Normal=45), Bilirubin \<1.5x upper limit normal (Normal=1.0). Normal PT, PTT, fibrinogen.
* Renal: Estimated Glomerular Filtration rate (eGFR) as calculated from the Bedside Schwartz equation (in units of mL/min/1.73 m2) or via radioisotope GFR of ≥ 70.
The Bedside Schwartz equation is: \[(0.413) X (Height in cm)\] / SCr
* Subjects of childbearing potential must have a negative pregnancy test. Subjects of childbearing potential must agree to use an effective birth control method. Subjects who are lactating must agree to stop breast-feeding.
* Written informed consent in accordance with institutional and FDA guidelines must be obtained from all subjects (or patients' legal representative).
Exclusion Criteria
* 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.
31 Years
ALL
No
Sponsors
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K C Pharmaceuticals Inc.
INDUSTRY
Beat NB Cancer Foundation
OTHER
Team Parker for Life
UNKNOWN
USWM, LLC
UNKNOWN
Giselle Sholler
OTHER
Responsible Party
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Giselle Sholler
Chair, Beat Childhood Cancer
Principal Investigators
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Giselle Sholler, MD
Role: STUDY_CHAIR
Beat Childhood Cancer
Locations
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University of Alabama, Children's 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
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, United States
University of Louisville
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
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
CancerCare Manitoba
Winnipeg, Manitoba, Canada
Montreal Children's Hospital
Montreal, Quebec, Canada
UHC Sainte-Justine
Montreal, Quebec, Canada
CHUQ
Québec, Quebec, Canada
CIUSSS de l'Estrie-CHUS
Sherbrooke, Quebec, Canada
Countries
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Central Contacts
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Facility Contacts
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Related Links
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Beat Childhood Cancer Consortium website
Other Identifiers
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BCC015
Identifier Type: -
Identifier Source: org_study_id
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