New hope for kids with aggressive cancer: tailored therapy trial launches

NCT ID NCT02559778

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

First seen Jun 27, 2026 · Last updated Sep 04, 2026 · Updated 1 time

Summary

This study is for children newly diagnosed with high-risk neuroblastoma, a rare and aggressive nerve cancer. It uses genetic testing to choose personalized chemotherapy, then tests whether adding the drug DFMO to standard immunotherapy helps prevent relapse. About 500 children will take part to see if this approach improves 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 500 people

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

Started

Sep 2015

Expected to finish

Sep 2035

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 22 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.

Part A- CLOSED: 1. Diagnosis: Participants must have a diagnosis of neuroblastoma or ganglioneuroblastoma (nodular or intermixed) verified by histology or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites. Participants with the following disease stages at diagnosis are eligible, if they meet the other specified criteria: a) Participants with newly diagnosed neuroblastoma with INSS Stage 4 are eligible with the following: i. Age \> 18 months (\> 547 days) regardless of biologic features or ii. Age 12-18 months (365-547 days) with any of the following 3 unfavorable biologic features (MYCN amplification, unfavorable pathology and/or DNA index = 1) or iii. MYCN amplification (\> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features. b) Participants with newly diagnosed neuroblastoma with INSS Stage 3 are eligible with the following: i. MYCN amplification (\> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features or ii. Age \> 18 months (\> 547 days) with unfavorable pathology, regardless of MYCN status. c) Participants with newly diagnosed neuroblastoma with INSS Stage 2A/2B with MYCN amplification (\> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features. 2. Participants must be age ≤ 21 years at initial diagnosis 3. Participants must not have had prior systemic therapy except for localized emergency radiation to sites of life-threatening or function-threatening disease and/or no more than 1 cycle of chemotherapy per a low or intermediate risk neuroblastoma regimen (as per P9641, A3961, ANBL0531, or similar) prior to determination of MYCN amplification status and histology. 4. Specimens will be obtained only in a non-significant risk manner and not solely for the purpose of investigational testing. 5. Ability to tolerate PBSC collection: No known contraindication to PBSC collection. Examples of contraindications would include a weight or size less than that determined to be feasible at the collecting institution, or a physical condition that would limit the ability of the child to undergo apheresis catheter placement (if necessary) and/or the apheresis procedure. Part A and B both- Part A CLOSED, Part B- OPEN: 6. Adequate Cardiac Function Defined As: 1. Shortening fraction of ≥ 27% by echocardiogram, or 2. Ejection fraction of ≥ 50% by radionuclide evaluation or echocardiogram. 7. Adequate liver function must be demonstrated, defined as: c. Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age AND d. ALT (SGPT) \< 10 x upper limit of normal (ULN) for age 8. ⦁ For participants \< 17 years old: 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 mL/min/1.73 m2. The Bedside Schwartz equation is: \[(0.413) X (Height in cm)\] / SCr ⦁ For participants ≥17 years old: estimated Glomerular Filtration rate (eGFR) as calculated from the Cockcroft and Gault formula (in units of mL/min/1.73 m2) or via radioisotope GFR of ≥ 70 mL/min/1.73 m2. The Cockcroft and Gault formula is: \[(140-age) x (Wt in kg) x (0.85 if female)\] / (72 x SCr) 9. A negative serum pregnancy test is required for female participants of child bearing potential (≥13 years of age or after onset of menses) 10. Post-pubertal study participants must be willing to use a highly effective contraceptive method (i.e., achieves a failure rate of \<1% per year when used consistently and correctly) from the time of informed consent (and assent, as applicable) until 6 months after study treatment discontinuation. Such methods include: combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, transdermal), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable), intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomized partner, sexual abstinence. 11. Informed Consent: All participants and/or legal guardians must sign informed written consent. Assent, when appropriate, will be obtained according to institutional guidelines. Part B- OPEN: 12. All patients must have a pathologically confirmed diagnosis of neuroblastoma, be age ≤ 21 years at initial diagnosis, and classified as high risk by the criteria used by COG or SIOPEN 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. 13. Previous Therapy- participants must fit into one of the strata categories listed in section 10.5 to be eligible to enroll on Part B of this study. 14. Pre-enrollment tumor survey: Prior to enrollment on Part B, a determination of mandatory disease staging must be performed. Tumor imaging studies including CT or MRI, MIBG or PET, and VMA/HVA (PET scan should be done for patients with prior disease that was MIBG non-avid). Bone marrow aspirates and biopsies are required. This disease assessment is required for eligibility and should be done preferably within 2 weeks, but must be done within a maximum of 4 weeks before first dose of study drug. 15. Timing- Enrollment to occur prior to Day + 120 post-transplant, preferably when the participant is within 28 days after completing local radiation therapy (if given). Exclusion Criteria (Part A and B) 1. Participants who are 12-18 months of age with INSS Stage 4 and all stage 3 participants with favorable biologic features (ie, nonamplified MYCN, favorable pathology, and DNA index \> 1) are not eligible. 2. Lactating females are not eligible unless they have agreed not to breastfeed their infants. 3. Participants receiving any investigational drug concurrently. 4. Participants with any other medical condition, including but not limited to malabsorption syndromes, mental illness or substance abuse, deemed by the Investigator to be likely to interfere with the interpretation of the results or which would interfere with a participant's ability to sign or the legal guardian's ability to sign the informed consent, and participant's ability to cooperate and participate in the study

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Conditions

The condition(s) this trial relates to.

ganglioneuroblastoma neuroblastoma

As listed by the trial registrant

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

Neuroblastoma

How to take part

Only the study team decides who joins. These are the ways to reach them.

The places running it

24 sites in 2 countries. The list below names each one and where it is.
The official record

ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

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A doctor treating you

A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

Advocate Aurora Research Institute
COMPLETED

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

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

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

Orlando, Florida, 32806, United States
Augusta University Health
RECRUITING

Augusta, Georgia, 30912, United States
CHU Sainte-Justine
RECRUITING

Montreal, Quebec, QC H3S 2G4, Canada
CHUQ
RECRUITING

Québec, Quebec, QC G1V 4W6, Canada
CIUSSS de l'Estrie-CHUS
RECRUITING

Sherbrooke, Quebec, QC J1H 5H3, Canada
Cardinal Glennon Children's Medical Center
RECRUITING

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

Minneapolis, Minnesota, 55404, United States
Children's Medical Center
RECRUITING

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

Kansas City, Missouri, 64108, United States
Connecticut Children's Hospital
RECRUITING

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

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

Hackensack, New Jersey, 07601, United States
Helen DeVos Children's Hospital
RECRUITING

Grand Rapids, Michigan, 49503, United States
Kapiolani Medical Center for Women and Children
RECRUITING

Honolulu, Hawaii, 96813, United States
Levine Children's Hospital
RECRUITING

Charlotte, North Carolina, 28204, United States
Medical University of South Carolina
RECRUITING

Charleston, South Carolina, 29425, United States
Nicklaus Children's Miami
RECRUITING

Miami, Florida, 33155, United States
Norton Children's Research Institute/Affiliated with University of Louisville School of Medicine
RECRUITING

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

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

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

Portland, Oregon, 97227, United States
St. Joseph's Children's Hospital
RECRUITING

Tampa, Florida, 33614, United States
St. Lukes
COMPLETED

Boise, Idaho, 83712, United States
UCSF Benioff Children's Hospital Oakland
RECRUITING

Oakland, California, 94609, United States
University of Alabama/Children's of Alabama
RECRUITING

Birmingham, Alabama, 35233, United States

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