Promising combo targets hard-to-treat childhood cancer

NCT ID NCT04301843

Disease control Sponsor: Giselle Sholler Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Sep 04, 2026 · Updated 4 times

Summary

This study tests whether combining two drugs, DFMO and etoposide, can help children and young adults (up to age 30) whose neuroblastoma has come back or not responded to standard treatment. About 131 participants will receive the drug combination to see if it delays cancer growth and improves survival. The goal is to find a better option for this aggressive childhood cancer.

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Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 131 people

The number the study aims to enrol. It can still change while the study runs.

Started

Sep 2020

Expected to finish

Oct 2033

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

Up to 31 years

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * 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: 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: * 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.

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Conditions

The condition(s) this trial relates to.

neuroblastoma

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Neuroblastoma

Contacts and locations

Locations

Alberta Children's Hospital

Calgary, Alberta, AB T3B 6A8, Canada
Arkansas Children's Hospital

Little Rock, Arkansas, 72202, United States
Arnold Palmer Hospital for Children

Orlando, Florida, 32806, United States
Augusta University Health

Augusta, Georgia, 30912, United States
CHUQ

Québec, Quebec, QC G1V 4W6, Canada
CancerCare Manitoba

Winnipeg, Manitoba, MB R3E 0V9, Canada
Cardinal Glennon Children's Hospital

St Louis, Missouri, 63104, United States
Children's Hospital and Clinics of Minnesota

Minneapolis, Minnesota, 55404, United States
Children's Hospital of The King's Daughters

Norfolk, Virginia, 23507, United States
Children's Medical Center Dallas

Dallas, Texas, 75235, United States
Children's Mercy Hospitals and Clinics

Kansas City, Missouri, 64108, United States
Cleveland Clinic Children's

Cleveland, Ohio, 44195, United States
Connecticut Children's Hospital

Hartford, Connecticut, 06106, United States
Dell Children's Blood and Cancer Center

Austin, Texas, 78723, United States
Hackensack University Medical Center

Hackensack, New Jersey, 07601, United States
Hasbro Children's Hospital

Providence, Rhode Island, 02901, United States
Helen DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States
Levine Children's Hospital

Charlotte, North Carolina, 28204, United States
Medical College of Wisconsin

Milwaukee, Wisconsin, 53226, United States
Medical University of South Carolina

Charleston, South Carolina, 29425, United States
Montreal Children's Hospital

Montreal, Quebec, QC H4A 3H9, Canada
Norton Children's Research Institute/Affiliated with University of Louisville School of Medicine

Louisville, Kentucky, 40201, United States
Penn State Milton S. Hershey Medical Center and Children's Hospital

Hershey, Pennsylvania, 17033, United States
Rady Children's Hospital

San Diego, California, 92123, United States
St. Joseph's Children's Hospital

Tampa, Florida, 33614, United States
UCSF Benioff Children's Hospital Oakland

Oakland, California, 94609, United States
UHC Sainte-Justine

Montreal, Quebec, QC H3S 2G4, Canada
University of Alabama/Children's of Alabama

Birmingham, Alabama, 35201, United States
University of Iowa

Iowa City, Iowa, 52242, United States

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