Promising drug may stop deadly childhood cancer's return

NCT ID NCT02679144

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

First seen Jun 27, 2026 · Last updated Sep 04, 2026 · Updated 2 times

Summary

This study tests an oral drug called DFMO in children and young adults with high-risk neuroblastoma who are currently in remission. The goal is to see if taking DFMO for two years can prevent the cancer from coming back. The trial involves 441 participants and focuses on safety and how long they stay cancer-free.

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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 441 people

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

Started

Feb 2016

Expected to finish

Feb 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

1 year to 30 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 and classified as high risk at the time of diagnosis. Exception: patients who are initially diagnosed as non-high-risk neuroblastoma, but later converted (and/or relapsed) to high risk neuroblastoma are also eligible. * All patients must be in complete remission (CR): 1. No evidence of residual disease on scan 2. No evidence of disease metastatic to bone marrow. * Specific Criteria by Stratum: Stratum 1/1B: All patients must have completed standard upfront therapy that replicates treatment which patients who were enrolled on ANBL0032 received, including: intensive induction chemotherapy and (if feasible) resection of primary tumor, followed by: consolidation with high-dose chemotherapy with stem cell transplant and radiotherapy, followed by: immunotherapy with Ch14.18/IL-2/GM-CSF (dinutuximab) and retinoic acid;. All subjects on Stratum 1/B must have also met the following criteria: • A pre-transplant disease status evaluation that met International Neuroblastoma Response Criteria (INRC) for CR (complete response), VGPR (very good partial response), or PR (partial response) for primary site, soft tissue metastases and bone metastases. Patients who meet those criteria must also meet the protocol-specified criteria for bone marrow response prior to transplant as outlined below: No more than 10% tumor involvement (based on total nucleated cellular content) seen on any specimen from a bilateral bone marrow aspirate/biopsy. Stratum 2: Neuroblastoma that is in first complete remission following standard upfront therapy different from that described for Stratum 1. Stratum 3: Neuroblastoma that failed to have a response of at least PR following induction chemotherapy and surgical resection of the primary tumor, but that has achieved CR following additional therapy. Stratum 4: Patients who have achieved a second or subsequent CR following relapse(s). * Pre-enrollment tumor survey: Prior to enrollment on this study, a determination of mandatory disease staging must be performed: * Tumor imaging studies including * Bilateral bone marrow aspirates and biopsy * This disease assessment is required for eligibility and preferably should be done within 2 weeks prior to enrollment, but must be done within a maximum of 4 weeks before enrollment. * Timing from prior therapy: Stratum 1/1B: Enrollment no later than 60 days after completion of upfront therapy, (last dose of cis-retinoic acid) with a maximum of 6 cycles of cis-retinoic acid maintenance therapy. Stratum 2, 3 and 4: Enrollment no later than 60 days from last dose of the most recent therapy. * Patients must have a Lansky or Karnofsky Performance Scale score of \> 50% and patients must have a life expectancy of ≥ 2 months. * All clinical and laboratory studies for organ functions to determine eligibility must be performed within 7 days prior to enrollment unless otherwise indicated below. * Patients must have adequate organ functions at the time of registration: * Hematological: Total absolute phagocyte count ≥1000/μL * Liver: Subjects must have adequate liver function * Renal: Adequate renal function * Females of childbearing potential must have a negative pregnancy test. Patients of childbearing potential must agree to use an effective birth control method. Female patients who are lactating must agree to stop breast-feeding. * Written informed consent in accordance with institutional and FDA (food and drug administration) guidelines must be obtained from all subjects (or patients' legal representative). Exclusion Criteria: * BSA (Body Surface Area) of \<0.25 m2. * 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

Advocate Aurora Research Institute

Chicago, Illinois, 60453, United States
Alberta Children's Hospital

Calgary, Alberta, AB T3B 6A8, Canada
All Children's Hospital Johns Hopkins Medicine

St. Petersburg, Florida, 33701, United States
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 Medical Center

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

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

Cleveland, Ohio, 44106, United States
Connecticut Children's Hospital

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

Austin, Texas, 78723, United States
Duke University

Durham, North Carolina, 27708, United States
Hackensack University Medical Center

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

Providence, Rhode Island, 02903, United States
Helen DeVos Children's Hospital

Grand Rapids, Michigan, 49503, United States
Janesway Children's Health and Rehabilitation Centre

St. John's, Newfoundland and Labrador, NL A1B 3V6, Canada
Kapiolani Medical Center for Women and Children

Honolulu, Hawaii, 96813, United States
Kentucky Children's Hospital

Lexington, Kentucky, 40536, 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
Monroe Carrell Jr. Children's Hospital at Vanderbilt

Nashville, Tennessee, 37232, United States
Montreal Children's Hospital

Montreal, Quebec, QC H4A 3H9, Canada
NYU Langone Health Hassenfeld Children's Hospital

New York, New York, 10016, United States
Nationwide Children's Hospital

Columbus, Ohio, 43205, United States
Norton Children's Research Institute/Affiliated with University of Louisville School of Medicine

Louisville, Kentucky, 40202, United States
Penn State Milton S. Hershey Medical Center and Children's Hospital

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

Salt Lake City, Utah, 84113, United States
Rady Children's Hospital

San Diego, California, 92123, United States
Randall Children's Hospital

Portland, Oregon, 97227, United States
Rocky Mountain Pediatric Hematology

Denver, Colorado, 80205, United States
St. Joseph's Children's Hospital

Tampa, Florida, 33614, United States
St. Lukes

Boise, Idaho, 83712, United States
Texas Children's Cancer and Hematology Centers

Houston, Texas, 77030, United States
The Children's Hospital at Montefiore

The Bronx, New York, 10467, United States
Tufts Medical Center

Boston, Massachusetts, 02111, 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, 35233, United States
University of Florida

Gainesville, Florida, 32611, United States
University of Iowa

Iowa City, Iowa, 52242, United States
Virginia Commonwealth University

Richmond, Virginia, 23284, United States

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