Ganglioneuroblastoma, NodularPhase 3NCT06172296
Dinutuximab With Chemotherapy, Surgery and Stem Cell Transplantation for the Treatment of Children With Newly Diagnosed High Risk Neuroblastoma
A Phase 3 study testing Biospecimen Collection, Bone Marrow Aspiration and Bone Marrow Biopsy in people with Ganglioneuroblastoma, Nodular and Neuroblastoma, run by National Cancer Institute (NCI) at 180 sites in 5 countries.
Trial summary
This phase III trial tests how well the addition of dinutuximab to Induction chemotherapy along with standard of care surgical resection of the primary tumor, radiation, stem cell transplantation, and immunotherapy works for treating children with newly diagnosed high-risk neuroblastoma. Read more about this study
- Who is it for?
- Ages Up to 30 years
- Conditions the study is for
- Ganglioneuroblastoma, Nodular, Neuroblastoma
- What is being tested?
- Biospecimen Collection, Bone Marrow Aspiration and Bone Marrow Biopsy
- Type of treatment
- Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 478 participants
- Where
- 180 sitesin 5 countries
- Study dates
- 19 Apr 2024to 31 Dec 2029
- Phase
- Phase 3
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Patients must be enrolled on APEC14B1 and have consented to testing through the Molecular Characterization Initiative (MCI), prior to enrollment on ANBL2131
- ≤ 30 years at the time of initial diagnosis with high-risk disease
- * Must have a diagnosis of neuroblastoma (NBL) or ganglioneuroblastoma (nodular) verified by tumor pathology analysis or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamines
- Newly diagnosed, high risk neuroblastoma (HRNBL) defined as one of the following:
- Any age with International Neuroblastoma Risk Group (INRG) Stage L2, MS, or M and MYCN amplification
- Age ≥ 547 days and INRG stage M regardless of biologic features (clinical MYCN testing not required prior to enrollment)
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- Any age initially diagnosed with INRG Stage L1 MYCN amplified NBL who have progressed to stage M without systemic chemotherapy
- Age ≥ 547 days of age initially diagnosed with INRG Stage L1, L2, or MS who have progressed to stage M without systemic chemotherapy (clinical MYCN testing not required prior to enrollment)
- Patients must have a body surface area (BSA) ≥ 0.25 m^2
- No prior anti-cancer therapy except as outlined below:
- Patients initially recognized to have high-risk disease treated with topotecan/cyclophosphamide initiated on an emergent basis and within allowed timing, and with consent
- Patients observed or treated with a single cycle of chemotherapy per a low or intermediate risk neuroblastoma regimen (e.g., as per ANBL0531, ANBL1232 or similar) for what initially appeared to be non-high-risk disease but subsequently found to meet the criteria
- Patients who received localized emergency radiation to sites of life threatening or function-threatening disease prior to or immediately after establishment of the definitive diagnosis
- Human immunodeficiency virus (HIV) -infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial
- A serum creatinine based on age/sex as follows:
- 1 month to < 6 months: Male 0.4 mg/dL and female 0.4mg/dL
- 6 months to < 1 year: Male 0.5 mg/dL and female 0.5 mg/dL
- 1 to < 2 years: Male 0.6 mg/dL and female 0.6 mg/dL
- 2 to < 6 years: Male 0.8 mg/dL and female 0.8 mg/dL
- 6 to < 10 years: Male 1 mg/dL and female 1 mg/dL
- 10 to < 13 years: Male 1.2 mg/dL and female 1.2 mg/dL
- 13 to < 16 years: Male 1.5 mg/dL and female 1.4 mg/dL
- ≥ 16 years: Male 1.7 mg/dL and female 1.4 mg/dL
- The threshold creatinine values were derived from the Schwartz formula for estimating glomerular filtration rate (GFR) utilizing child length and stature data published by the Centers for Disease Control (CDC)
- or a 24-hour urine creatinine clearance ≥ 70 mL/min/1.73 m^2 or
- or a GFR ≥ 70 mL/min/1.73 m^2. GFR must be performed using direct measurement with a nuclear blood sampling method or direct small molecule clearance method (iothalamate or other molecule per institutional standard)
- Note: Estimated GFR (eGFR) from serum creatinine, cystatin C or other estimates are not acceptable for determining eligibility
- Total bilirubin ≤ 1.5 x upper limit of normal (ULN) for age
- Serum glutamic pyruvic transaminase (SGPT) (Alanine aminotransferase [ALT]) ≤ 10 x ULN*
- Note: For the purpose of this study, the ULN for SGPT (ALT) has been set to the value of 45 U/L
- * Shortening fraction of ≥ 27% by echocardiogram, or
- Ejection fraction of ≥ 50% by echocardiogram or radionuclide angiogram
- Ability to tolerate Peripheral Blood Stem Cell (PBSC) collection:
- No known contraindication to PBSC collection. Examples of contraindications might be a weight or size less than the collecting institution finds feasible, or a physical condition that would limit the ability of the child to undergo apheresis catheter placement (if necessary) and/or the apheresis procedure
Who cannot join
- Patients who are 365-546 days of age with INRG Stage M and MYCN non-amplified NBL, irrespective of additional biologic features
- Patients ≥ 547 days of age with INRG Stage L2, MYCN non-amplified NBL, regardless of additional biologic features
- Patients with known bone marrow failure syndromes
- Patients on chronic immunosuppressive medications (e.g., tacrolimus, cyclosporine, corticosteroids) for reasons other than prevention/treatment of allergic reactions and adrenal replacement therapy are not eligible. Topical and inhaled corticosteroids are acceptable
- Patients with a primary immunodeficiency syndrome who require ongoing immune globulin replacement therapy
- Female patients who are pregnant since fetal toxicities and teratogenic effects have been noted for several of the study drugs. A pregnancy test is required prior to enrollment for female patients of childbearing potential
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- Lactating females who plan to breastfeed their infants
- Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation
- All patients and/or their parents or legal guardians must sign a written informed consent
- All institutional, food and drug administration (FDA), and National Cancer Institute (NCI) requirements for human studies must be met
Trial locations and contacts
180 sites in 5 countries. Contact the study team or a site near you to ask about taking part.
180 sites in 5 countries
United States158 sites, 155 recruiting
- Recruiting
Children's Hospital Medical Center of Akron
Akron, Ohio, 44308
Site Public Contact, 330-543-3193
Erin Wright
- Recruiting
Albany Medical Center
Albany, New York, 12208
Site Public Contact, 518-262-5513
Lauren R. Weintraub
- Suspended
University of New Mexico Cancer Center
Albuquerque, New Mexico, 87106
- Recruiting
Lehigh Valley Hospital-Cedar Crest
Allentown, Pennsylvania, 18103
Site Public Contact, 610-402-9543, Morgan_M.Horton@lvhn.org
Jacob A. Troutman
- Recruiting
C S Mott Children's Hospital
Ann Arbor, Michigan, 48109
Site Public Contact, 800-865-1125
Rajen Mody
- Recruiting
Mission Hospital
Asheville, North Carolina, 28801
Site Public Contact, 828-213-7055, NCDV.ResearchRegulatory@HCAHealthcare.com
Douglas J. Scothorn
- Recruiting
Children's Healthcare of Atlanta - Arthur M Blank Hospital
Atlanta, Georgia, 30329
Site Public Contact, 404-785-0232, Olivia.Floyd@choa.org
William T. Cash
- Recruiting
Children's Hospital Colorado
Aurora, Colorado, 80045
Site Public Contact, 303-764-5056, josh.b.gordon@nsmtp.kp.org
Navin R. Pinto
- Recruiting
Dell Children's Medical Center of Central Texas
Austin, Texas, 78723
Site Public Contact, 512-628-1902, TXAUS-DL-SFCHemonc.research@ascension.org
Shannon M. Cohn
- Recruiting
University of Maryland/Greenebaum Cancer Center
Baltimore, Maryland, 21201
Site Public Contact, 800-888-8823
Teresa A. York
- Recruiting
Sinai Hospital of Baltimore
Baltimore, Maryland, 21215
Site Public Contact, 410-601-9083
Jason M. Fixler
- Recruiting
Johns Hopkins University/Sidney Kimmel Cancer Center
Baltimore, Maryland, 21287
Site Public Contact, 410-955-8804, jhcccro@jhmi.edu
Allen R. Chen
- Recruiting
Eastern Maine Medical Center
Bangor, Maine, 04401
Site Public Contact, 207-973-4274
Daniel L. Callaway
- Recruiting
Walter Reed National Military Medical Center
Bethesda, Maryland, 20889-5600
Site Public Contact, 301-319-2100
Kip R. Hartman
- Recruiting
Children's Hospital of Alabama
Birmingham, Alabama, 35233
Site Public Contact, 205-638-9285, oncologyresearch@peds.uab.edu
Elizabeth D. Alva
- Recruiting
Massachusetts General Hospital Cancer Center
Boston, Massachusetts, 02114
Site Public Contact, 877-726-5130
Lauren H. Boal
- Recruiting
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Site Public Contact, 877-442-3324
Suzanne Shusterman
- Recruiting
Roswell Park Cancer Institute
Buffalo, New York, 14263
Site Public Contact, 800-767-9355, askroswell@roswellpark.org
Clare J. Twist
- Recruiting
University of Vermont and State Agricultural College
Burlington, Vermont, 05405
Site Public Contact, 802-656-8990, rpo@uvm.edu
Jessica L. Heath
- Recruiting
UNC Lineberger Comprehensive Cancer Center
Chapel Hill, North Carolina, 27599
Site Public Contact, 877-668-0683, cancerclinicaltrials@med.unc.edu
Thomas B. Alexander
- Recruiting
Medical University of South Carolina
Charleston, South Carolina, 29425
Site Public Contact, 843-792-9321, hcc-clinical-trials@musc.edu
Jacqueline M. Kraveka
- Recruiting
Carolinas Medical Center/Levine Cancer Institute
Charlotte, North Carolina, 28203
Site Public Contact, 800-804-9376
Joel A. Kaplan
- Recruiting
Novant Health Presbyterian Medical Center
Charlotte, North Carolina, 28204
Site Public Contact, 980-201-6360, kashah@novanthealth.org
Holly Edington
- Recruiting
University of Virginia Cancer Center
Charlottesville, Virginia, 22908
Site Public Contact, 434-243-6303, uvacancertrials@hscmail.mcc.virginia.edu
Brian C. Belyea
- Recruiting
Lurie Children's Hospital-Chicago
Chicago, Illinois, 60611
Site Public Contact, 773-880-4562
Elizabeth A. Sokol
- Recruiting
University of Illinois
Chicago, Illinois, 60612
Site Public Contact, 312-355-3046
Dipti S. Dighe
- Recruiting
University of Chicago Comprehensive Cancer Center
Chicago, Illinois, 60637
Site Public Contact, 773-702-8222, cancerclinicaltrials@bsd.uchicago.edu
Ami V. Desai
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Site Public Contact, 513-636-2799, cancer@cchmc.org
Katherine M. Somers
- Recruiting
Rainbow Babies and Childrens Hospital
Cleveland, Ohio, 44106
Site Public Contact, 216-844-5437
Duncan S. Stearns
- Recruiting
Cleveland Clinic Foundation
Cleveland, Ohio, 44195
Site Public Contact, 866-223-8100, TaussigResearch@ccf.org
Matteo M. Trucco
- Recruiting
University of Missouri Children's Hospital
Columbia, Missouri, 65212
Site Public Contact, 573-882-1960, snwq62@health.missouri.edu
Barbara A. Gruner
- Recruiting
Prisma Health Richland Hospital
Columbia, South Carolina, 29203
Site Public Contact, 864-522-4317, Kim.Williams3@prismahealth.org
Stuart L. Cramer
- Recruiting
Nationwide Children's Hospital
Columbus, Ohio, 43205
Site Public Contact, 614-722-6039, Melinda.Triplet@nationwidechildrens.org
Mark A. Ranalli
- Recruiting
Driscoll Children's Hospital
Corpus Christi, Texas, 78411
Site Public Contact, 361-694-5311, Crystal.DeLosSantos@dchstx.org
Nkechi I. Mba
- Recruiting
Medical City Dallas Hospital
Dallas, Texas, 75230
Site Public Contact, 972-566-5588
Maurizio L. Ghisoli
- Recruiting
UT Southwestern/Simmons Cancer Center-Dallas
Dallas, Texas, 75390
Site Public Contact, 214-648-7097, canceranswerline@UTSouthwestern.edu
Tanya C. Watt
- Recruiting
Geisinger Medical Center
Danville, Pennsylvania, 17822
Site Public Contact, 570-271-5251, cancerresearch@geisinger.edu
Jagadeesh Ramdas
- Recruiting
Dayton Children's Hospital
Dayton, Ohio, 45404
Site Public Contact, 800-228-4055
Jordan M. Wright
- Recruiting
Blank Children's Hospital
Des Moines, Iowa, 50309
Site Public Contact, 515-241-8912, samantha.mallory@unitypoint.org
Samantha L. Mallory
- Suspended
Children's Hospital of Michigan
Detroit, Michigan, 48201
- Recruiting
Kaiser Permanente Downey Medical Center
Downey, California, 90242
Site Public Contact, 626-564-3455
Robert M. Cooper
- Recruiting
City of Hope Comprehensive Cancer Center
Duarte, California, 91010
Site Public Contact, 800-826-4673, becomingapatient@coh.org
Hung C. Tran
- Recruiting
Duke University Medical Center
Durham, North Carolina, 27710
Site Public Contact, 888-275-3853
Jessica M. Sun
- Recruiting
El Paso Children's Hospital
El Paso, Texas, 79905
Site Public Contact, 915-298-5444, ranjan.bista@ttuhsc.edu
Benjamin Carcamo
- Recruiting
Sanford Broadway Medical Center
Fargo, North Dakota, 58122
Site Public Contact, 701-323-5760, OncologyClinicalTrialsFargo@sanfordhealth.org
Samuel J. Milanovich
- Recruiting
Broward Health Medical Center
Fort Lauderdale, Florida, 33316
Site Public Contact, 302-651-5572, Allison.bruce@nemours.org
Hector M. Rodriguez-Cortes
- Recruiting
Golisano Children's Hospital of Southwest Florida
Fort Myers, Florida, 33908
Site Public Contact, 239-343-5333, molly.arnstrom@leehealth.org
Emad K. Salman
- Recruiting
Cook Children's Medical Center
Fort Worth, Texas, 76104
Site Public Contact, 682-885-2103, CookChildrensResearch@cookchildrens.org
Chelsee Greer
- Recruiting
UF Health Cancer Institute - Gainesville
Gainesville, Florida, 32610
Site Public Contact, 352-273-8010, cancer-center@ufl.edu
Brian Stover
- Recruiting
Corewell Health Grand Rapids Hospitals - Helen DeVos Children's Hospital
Grand Rapids, Michigan, 49503
Site Public Contact, 616-391-1230, crcwm-regulatory@crcwm.org
Kathleen Y. Butler
- Suspended
Saint Vincent Hospital Cancer Center Green Bay
Green Bay, Wisconsin, 54301
- Recruiting
East Carolina University
Greenville, North Carolina, 27834
Site Public Contact, 252-744-1015, eubankss@ecu.edu
Andrea R. Whitfield
- Recruiting
BI-LO Charities Children's Cancer Center
Greenville, South Carolina, 29605
Site Public Contact, 864-522-4317, Kim.Williams3@prismahealth.org
Aniket Saha
- Recruiting
Hackensack University Medical Center
Hackensack, New Jersey, 07601
Site Public Contact, 551-996-2897
Katharine Offer
- Recruiting
Connecticut Children's Medical Center
Hartford, Connecticut, 06106
Site Public Contact, 860-545-9981
Michael S. Isakoff
- Recruiting
Memorial Regional Hospital/Joe DiMaggio Children's Hospital
Hollywood, Florida, 33021
Site Public Contact, 954-265-1847, OHR@mhs.net
Iftikhar Hanif
- Recruiting
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, 96826
Site Public Contact, 808-983-6090
Wade T. Kyono
- Recruiting
Baylor College of Medicine/Dan L Duncan Comprehensive Cancer Center
Houston, Texas, 77030
Site Public Contact, 713-798-1354, burton@bcm.edu
Jennifer H. Foster
- Recruiting
UT MD Anderson Cancer Center
Houston, Texas, 77030
Site Public Contact, 866-632-6789, askmdanderson@mdanderson.org
Najat C. Daw
- Recruiting
Edwards Comprehensive Cancer Center
Huntington, West Virginia, 25701
Site Public Contact, 304-399-6566, Christina.Cole@chhi.org
Mark A. Ranalli
- Recruiting
Riley Hospital for Children
Indianapolis, Indiana, 46202
Site Public Contact, 800-248-1199
Marissa Just
- Recruiting
Ascension Saint Vincent Indianapolis Hospital
Indianapolis, Indiana, 46260
Site Public Contact, 317-338-2194, research@stvincent.org
Bassem I. Razzouk
- Recruiting
University of Iowa/Holden Comprehensive Cancer Center
Iowa City, Iowa, 52242
Site Public Contact, 800-237-1225
Andrew P. Groves
- Recruiting
University of Mississippi Medical Center
Jackson, Mississippi, 39216
Site Public Contact, 601-815-6700
Amanda Strobel
- Recruiting
Nemours Children's Clinic-Jacksonville
Jacksonville, Florida, 32207
Site Public Contact, 302-651-5572, Allison.bruce@nemours.org
Sridhi Patel
- Recruiting
Bronson Methodist Hospital
Kalamazoo, Michigan, 49007
Site Public Contact, 616-391-1230, crcwm-regulatory@crcwm.org
Kathleen Y. Butler
- Recruiting
Children's Mercy Hospitals and Clinics
Kansas City, Missouri, 64108
Site Public Contact, 816-302-6808, COGResearchGroup@cmh.edu
Keith J. August
- Recruiting
East Tennessee Childrens Hospital
Knoxville, Tennessee, 37916
Site Public Contact, 865-541-8266
Susan E. Spiller
- Recruiting
Alliance for Childhood Diseases/Cure 4 the Kids Foundation
Las Vegas, Nevada, 89135
Site Public Contact, 702-384-0013, research@sncrf.org
Joseph L. Lasky
- Recruiting
Dartmouth Hitchcock Medical Center/Dartmouth Cancer Center
Lebanon, New Hampshire, 03756
Site Public Contact, 800-639-6918, cancer.research.nurse@dartmouth.edu
Angela Ricci
- Recruiting
University of Kentucky/Markey Cancer Center
Lexington, Kentucky, 40536
Site Public Contact, 859-257-3379
James T. Badgett
- Recruiting
Arkansas Children's Hospital
Little Rock, Arkansas, 72202-3591
Site Public Contact, 501-364-7373
Michael W. Bishop
- Recruiting
Loma Linda University Medical Center
Loma Linda, California, 92354
Site Public Contact, 909-558-4050
Albert Kheradpour
- Recruiting
Miller Children's and Women's Hospital Long Beach
Long Beach, California, 90806
Site Public Contact, 562-933-5600
Jacqueline N. Casillas
- Recruiting
Children's Hospital Los Angeles
Los Angeles, California, 90027
Site Public Contact, 323-361-4110
Araz Marachelian
- Recruiting
Cedars-Sinai Medical Center
Los Angeles, California, 90048
Site Public Contact, 310-423-2133, Cancer.trial.info@cshs.org
Fataneh (Fae) Majlessipour
- Recruiting
Mattel Children's Hospital UCLA
Los Angeles, California, 90095
Site Public Contact, 310-825-6708
Satiro N. De Oliveira
- Recruiting
Norton Children's Hospital
Louisville, Kentucky, 40202
Site Public Contact, 502-629-5500, CancerResource@nortonhealthcare.org
Michael J. Ferguson
- Recruiting
Covenant Children's Hospital
Lubbock, Texas, 79410
Site Public Contact, 806-725-8657, mbisbee@providence.org
Kishor M. Bhende
- Recruiting
UMC Cancer Center / UMC Health System
Lubbock, Texas, 79415
Site Public Contact, 806-775-8590
Erin K. Barr
- Recruiting
Valley Children's Hospital
Madera, California, 93636
Site Public Contact, 559-353-3000, Research@valleychildrens.org
Ruetima Titapiwatanakun
- Recruiting
University of Wisconsin Carbone Cancer Center - University Hospital
Madison, Wisconsin, 53792
Site Public Contact, 800-622-8922, clinicaltrials@cancer.wisc.edu
Margo L. Hoover-Regan
- Recruiting
Marshfield Medical Center-Marshfield
Marshfield, Wisconsin, 54449
Site Public Contact, 800-782-8581, oncology.clinical.trials@marshfieldresearch.org
Michelle A. Manalang
- Recruiting
Saint Jude Children's Research Hospital
Memphis, Tennessee, 38105
Site Public Contact, 888-226-4343, referralinfo@stjude.org
Sara M. Federico
- Recruiting
Banner Children's at Desert
Mesa, Arizona, 85202
Site Public Contact, 480-412-3100
Joseph C. Torkildson
- Recruiting
University of Miami Miller School of Medicine-Sylvester Cancer Center
Miami, Florida, 33136
Site Public Contact, 305-243-2647
Meghan McCormick
- Recruiting
Nicklaus Children's Hospital
Miami, Florida, 33155
Site Public Contact, 888-624-2778
Ziad A. Khatib
- Recruiting
Children's Hospital of Wisconsin
Milwaukee, Wisconsin, 53226
Site Public Contact, 414-955-4727, MACCCTO@mcw.edu
Angela Steineck
- Recruiting
NYU Langone Hospital - Long Island
Mineola, New York, 11501
Site Public Contact, 212-263-4432, cancertrials@nyulangone.org
Chana L. Glasser
- Recruiting
Children's Hospitals and Clinics of Minnesota - Minneapolis
Minneapolis, Minnesota, 55404
Site Public Contact, 612-813-5913, pauline.mitby@childrensmn.org
Michael K. Richards
- Recruiting
University of Minnesota/Masonic Cancer Center
Minneapolis, Minnesota, 55455
Site Public Contact, 612-624-2620
Robin L. Williams
- Recruiting
USA Health Strada Patient Care Center
Mobile, Alabama, 36604
Site Public Contact, 800-388-8721
Hamayun Imran
- Recruiting
West Virginia University Healthcare
Morgantown, West Virginia, 26506
Site Public Contact, 304-293-7374, cancertrialsinfo@hsc.wvu.edu
Ashley E. Meyer
- Recruiting
Morristown Medical Center
Morristown, New Jersey, 07960
Site Public Contact, 973-971-5900
Kathryn L. Laurie
- Recruiting
The Children's Hospital at TriStar Centennial
Nashville, Tennessee, 37203
Site Public Contact, 615-342-1919
Clinton M. Carroll
- Recruiting
Vanderbilt University/Ingram Cancer Center
Nashville, Tennessee, 37232
Site Public Contact, 800-811-8480
Daniel J. Benedetti
- Recruiting
Saint Peter's University Hospital
New Brunswick, New Jersey, 08901
Site Public Contact, 732-745-8600, officeofresearch@saintpetersuh.com
Nibal A. Zaghloul
- Recruiting
Rutgers Cancer Institute of New Jersey-Robert Wood Johnson University Hospital
New Brunswick, New Jersey, 08903
Site Public Contact, 732-235-8675
Nehal S. Parikh
- Recruiting
Yale University
New Haven, Connecticut, 06520
Site Public Contact, 203-785-5702, canceranswers@yale.edu
Farzana Pashankar
- Recruiting
The Steven and Alexandra Cohen Children's Medical Center of New York
New Hyde Park, New York, 11040
Site Public Contact, 718-470-3460
Julie I. Krystal
- Recruiting
Children's Hospital New Orleans
New Orleans, Louisiana, 70118
Site Public Contact, 504-894-5377
Maria C. Velez-Yanguas
- Recruiting
Laura and Isaac Perlmutter Cancer Center at NYU Langone
New York, New York, 10016
Site Public Contact, CancerTrials@nyulangone.org
Elizabeth A. Raetz
- Recruiting
NYP/Columbia University Medical Center/Herbert Irving Comprehensive Cancer Center
New York, New York, 10032
Site Public Contact, 212-342-5162, cancerclinicaltrials@cumc.columbia.edu
Nobuko Hijiya
- Recruiting
Newark Beth Israel Medical Center
Newark, New Jersey, 07112
Site Public Contact, 973-926-7230, Christine.Kosmides@rwjbh.org
Teena Bhatla
- Recruiting
Advocate Children's Hospital-Oak Lawn
Oak Lawn, Illinois, 60453
Site Public Contact, 847-723-7570
Rebecca E. McFall
- Recruiting
UCSF Benioff Children's Hospital Oakland
Oakland, California, 94609
Site Public Contact, 510-428-3264, PedOncRschOAK@ucsf.edu
Jennifer G. Michlitsch
- Recruiting
Kaiser Permanente-Oakland
Oakland, California, 94611
Site Public Contact, 877-642-4691, Kpoct@kp.org
Aarati V. Rao
- Recruiting
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104
Site Public Contact, 405-271-8777, ou-clinical-trials@ouhsc.edu
Rene Y. McNall-Knapp
- Recruiting
Children's Hospital and Medical Center of Omaha
Omaha, Nebraska, 68114
Site Public Contact, 402-955-3949
Jill C. Beck
- Recruiting
University of Nebraska Medical Center
Omaha, Nebraska, 68198
Site Public Contact, 402-559-6941, unmcrsa@unmc.edu
Jill C. Beck
- Recruiting
Children's Hospital of Orange County
Orange, California, 92868
Site Public Contact, 714-509-8646, oncresearch@choc.org
Elyssa M. Rubin
- Recruiting
AdventHealth Orlando
Orlando, Florida, 32803
Site Public Contact, 407-303-2090, FH.Cancer.Research@flhosp.org
Fouad M. Hajjar
- Recruiting
Arnold Palmer Hospital for Children
Orlando, Florida, 32806
Site Public Contact, 321-841-5357, Jennifer.spinelli@orlandohealth.com
Zhongbo Hu
- Recruiting
Nemours Children's Hospital
Orlando, Florida, 32827
Site Public Contact, 302-651-5572, Allison.bruce@nemours.org
Sridhi Patel
- Recruiting
Lucile Packard Children's Hospital Stanford University
Palo Alto, California, 94304
Site Public Contact, 800-694-0012, ccto-office@stanford.edu
Jay Michael S. Balagtas
- Recruiting
Advocate Children's Hospital-Park Ridge
Park Ridge, Illinois, 60068
Site Public Contact, helpdesk@childrensoncologygroup.org
Rebecca E. McFall
- Recruiting
Saint Joseph's Regional Medical Center
Paterson, New Jersey, 07503
Site Public Contact, 973-754-2207, HallL@sjhmc.org
Alissa Kahn
- Recruiting
OSF Children's Hospital of Illinois
Peoria, Illinois, 61637
Site Public Contact, 309-624-4945, ChildrensHospitalofIllinois@osfhealthcare.org
Prerna Kumar
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Site Public Contact, 267-425-5544, CancerTrials@email.chop.edu
Rochelle Bagatell
- Recruiting
Saint Christopher's Hospital for Children
Philadelphia, Pennsylvania, 19134
Site Public Contact, 215-427-8991
Gregory E. Halligan
- Recruiting
Phoenix Childrens Hospital
Phoenix, Arizona, 85016
Site Public Contact, 602-546-0920
Alok K. Kothari
- Recruiting
Children's Hospital of Pittsburgh of UPMC
Pittsburgh, Pennsylvania, 15224
Site Public Contact, 412-692-8570, jean.tersak@chp.edu
Jean M. Tersak
- Recruiting
Legacy Emanuel Children's Hospital
Portland, Oregon, 97227
Site Public Contact, 503-413-2560
Jason M. Glover
- Recruiting
Oregon Health and Science University
Portland, Oregon, 97239
Site Public Contact, 503-494-1080, trials@ohsu.edu
Susan J. Lindemulder
- Recruiting
Naval Medical Center - Portsmouth
Portsmouth, Virginia, 23708-2197
Site Public Contact, 757-953-5939
Karin Brockman
- Recruiting
Rhode Island Hospital
Providence, Rhode Island, 02903
Site Public Contact, 401-444-1488
Bradley DeNardo
- Recruiting
Mayo Clinic in Rochester
Rochester, Minnesota, 55905
Site Public Contact, 855-776-0015
Peter Schoettler
- Recruiting
University of Rochester
Rochester, New York, 14642
Site Public Contact, 585-275-5830
Rafi R. Kazi
- Recruiting
Corewell Health Children's
Royal Oak, Michigan, 48073
Site Public Contact, 248-551-7695
Marie V. Nelson
- Recruiting
Sutter Medical Center Sacramento
Sacramento, California, 95816
Site Public Contact, clinicalresearch@sutterhealth.org
Sandra Baril
- Recruiting
University of California Davis Comprehensive Cancer Center
Sacramento, California, 95817
Site Public Contact, 916-734-3089
Marcio H. Malogolowkin
- Recruiting
Primary Children's Hospital
Salt Lake City, Utah, 84113
Site Public Contact, 801-585-5270
Leonora R. Slatnick
- Recruiting
Children's Hospital of San Antonio
San Antonio, Texas, 78207
Site Public Contact, 210-704-2894, bridget.medina@christushealth.org
Julie Voeller
- Recruiting
Methodist Children's Hospital of South Texas
San Antonio, Texas, 78229
Site Public Contact, 210-575-6240, Vinod.GidvaniDiaz@hcahealthcare.com
Jose M. Esquilin
- Recruiting
University of Texas Health Science Center at San Antonio
San Antonio, Texas, 78229
Site Public Contact, 210-450-3800, phoresearchoffice@uthscsa.edu
Aaron J. Sugalski
- Recruiting
Rady Children's Hospital - San Diego
San Diego, California, 92123
Site Public Contact, 858-966-5934
William D. Roberts
- Recruiting
UCSF Medical Center-Mission Bay
San Francisco, California, 94158
Site Public Contact, 877-827-3222, cancertrials@ucsf.edu
Arun A. Rangaswami
- Recruiting
Memorial Health University Medical Center
Savannah, Georgia, 31404
Site Public Contact, 912-350-7887, Lorraine.OHara@hcahealthcare.com
James N. Yarnall
- Recruiting
Maine Children's Cancer Program
Scarborough, Maine, 04074
Site Public Contact, 207-396-8670, clinicalresearch@mainehealth.org
Sei-Gyung K. Sze
- Recruiting
Seattle Children's Hospital
Seattle, Washington, 98105
Site Public Contact, 866-987-2000
Sarah E. Leary
- Recruiting
Sanford USD Medical Center - Sioux Falls
Sioux Falls, South Dakota, 57117-5134
Site Public Contact, 605-312-3320, OncologyClinicalTrialsSF@SanfordHealth.org
Jordan Fritch-Hanson
- Recruiting
Providence Sacred Heart Medical Center and Children's Hospital
Spokane, Washington, 99204
Site Public Contact, 800-228-6618, HopeBeginsHere@providence.org
Judy L. Felgenhauer
- Recruiting
Southern Illinois University School of Medicine
Springfield, Illinois, 62702
Site Public Contact, 217-545-7929
Gregory P. Brandt
- Recruiting
Washington University School of Medicine
St Louis, Missouri, 63110
Site Public Contact, 800-600-3606, info@siteman.wustl.edu
Frederick S. Huang
- Recruiting
Mercy Hospital Saint Louis
St Louis, Missouri, 63141
Site Public Contact, 314-251-7066
Robin D. Hanson
- Recruiting
Johns Hopkins All Children's Hospital
St. Petersburg, Florida, 33701
Site Public Contact, 727-767-4784, Ashley.Repp@jhmi.edu
Jennifer B. Dean
- Recruiting
State University of New York Upstate Medical University
Syracuse, New York, 13210
Site Public Contact, 315-464-5476
Melanie A. Comito
- Recruiting
Mary Bridge Children's Hospital and Health Center
Tacoma, Washington, 98405
Site Public Contact, 253-403-1461, research@multicare.org
Robert G. Irwin
- Recruiting
Montefiore Medical Center - Moses Campus
The Bronx, New York, 10467
Site Public Contact, 718-379-6866, eskwak@montefiore.org
Alice Lee
- Recruiting
ProMedica Toledo Hospital/Russell J Ebeid Children's Hospital
Toledo, Ohio, 43606
Site Public Contact, 419-824-1842, PCIOncResearch@promedica.org
Jamie L. Dargart
- Recruiting
Banner University Medical Center - Tucson
Tucson, Arizona, 85719
Site Public Contact, UACC-IIT@uacc.arizona.edu
Monica M. Davini
- Recruiting
Saint Francis Children's Hospital
Tulsa, Oklahoma, 74136
Site Public Contact, 918-502-6720
Jill A. Salo
- Recruiting
New York Medical College
Valhalla, New York, 10595
Site Public Contact, 914-594-3794
Andrew J. Bellantoni
- Recruiting
Children's National Medical Center
Washington D.C., District of Columbia, 20010
Site Public Contact, 202-476-2800, OncCRC_OnCall@childrensnational.org
Jeffrey S. Dome
- Recruiting
Saint Mary's Medical Center
West Palm Beach, Florida, 33407
Site Public Contact, 561-822-4745
Matthew D. Ramirez
- Recruiting
Wesley Medical Center
Wichita, Kansas, 67214
Site Public Contact, 316-962-7802, WesleyResearch@wesleymc.com
Nathan S. Hall
- Recruiting
Alfred I duPont Hospital for Children
Wilmington, Delaware, 19803
Site Public Contact, 302-651-5572, Allison.bruce@nemours.org
Sridhi Patel
- Recruiting
Wake Forest University Health Sciences
Winston-Salem, North Carolina, 27157
Site Public Contact, 336-713-6771
Sarah Supples
Canada15 sites, 15 recruiting
- Recruiting
Alberta Children's Hospital
Calgary, Alberta, T3B 6A8
Site Public Contact, 403-220-6898, research4kids@ucalgary.ca
Victor A. Lewis
- Recruiting
University of Alberta Hospital
Edmonton, Alberta, T6G 2B7
Site Public Contact, 780-407-8798, pedsoncologyresearch@ahs.ca
Sarah J. McKillop
- Recruiting
IWK Health Centre
Halifax, Nova Scotia, B3K 6R8
Site Public Contact, 902-470-8520, Research@iwk.nshealth.ca
Chelsea Ash
- Recruiting
McMaster Children's Hospital at Hamilton Health Sciences
Hamilton, Ontario, L8N 3Z5
Site Public Contact, 905-521-2100
Uma H. Athale
- Recruiting
Children's Hospital
London, Ontario, N6A 5W9
Site Public Contact, 519-685-8306
Shayna M. Zelcer
- Recruiting
The Montreal Children's Hospital of the MUHC
Montreal, Quebec, H3H 1P3
Site Public Contact, 514-412-4445, info@thechildren.com
Stephanie Mourad
- Recruiting
Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, H3T 1C5
Site Public Contact, 514-345-4931, yvan.samson@umontreal.ca
Monia Marzouki
- Recruiting
Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1
Site Public Contact, 613-737-7600
Donna L. Johnston
- Recruiting
CHU de Quebec-Centre Hospitalier de l'Universite Laval (CHUL)
Québec, G1V 4G2
Site Public Contact, 418-525-4444, rechclinique@crchudequebec.ulaval.ca
Bruno Michon
- Recruiting
Jim Pattison Children's Hospital
Saskatoon, Saskatchewan, S7N 0W8
Site Public Contact, 306-655-3544, Jessica.Marien@saskhealthauthority.ca
Kathleen Felton
- Recruiting
Centre Hospitalier Universitaire de Sherbrooke-Fleurimont
Sherbrooke, Quebec, J1H 5N4
Site Public Contact, 819-820-6480, crcinformation.chus@ssss.gouv.qc.ca
Stephanie Vairy
- Recruiting
Janeway Child Health Centre
St. John's, Newfoundland and Labrador, A1B 3V6
Site Public Contact, 709-777-8727, beverlyj.mitchell@easternhealth.ca
Lisa A. Goodyear
- Recruiting
Hospital for Sick Children
Toronto, Ontario, M5G 1X8
Site Public Contact, 416-813-7654, ask.CRS@sickkids.ca
Daniel A. Morgenstern
- Recruiting
British Columbia Children's Hospital
Vancouver, British Columbia, V6H 3V4
Site Public Contact, 604-875-2345 ext. 6477
Rebecca J. Deyell
- Recruiting
CancerCare Manitoba
Winnipeg, Manitoba, R3E 0V9
Site Public Contact, 866-561-1026, ctu_web@cancercare.mb.ca
Stephanie M. Villeneuve
Australia5 sites, 5 recruiting
- Recruiting
Royal Children's Hospital
Parkville, Victoria, 3052
Site Public Contact, 61 3 9345 5656, Jordan.Hansford@rch.org.au
Martin A. Campbell
- Recruiting
Perth Children's Hospital
Perth, Western Australia, 6009
Site Public Contact, helpdesk@childrensoncologygroup.org
Michelle Ng
- Recruiting
Sydney Children's Hospital
Randwick, New South Wales, 2031
Site Public Contact, (02) 9382-1721
Draga Barbaric
- Recruiting
Queensland Children's Hospital
South Brisbane, Queensland, 4101
Site Public Contact, 61 7 3068 1111
Steven A. Foresto
- Recruiting
The Children's Hospital at Westmead
Westmead, New South Wales, 2145
Site Public Contact, 61-2-9845 1400
Bhavna Padhye
New Zealand1 site, 1 recruiting
- Recruiting
Starship Children's Hospital
Grafton, Auckland, 1145
Site Public Contact, 0800 728 436
Andrew C. Wood
Puerto Rico1 site, 1 recruiting
- Recruiting
University Pediatric Hospital
San Juan, 00926
Site Public Contact, 787-474-0333
Maria E. Echevarria
About this study
Official title: A Phase 3 Study of Dinutuximab Added to Intensive Multimodal Therapy for Children With Newly Diagnosed High-Risk Neuroblastoma
This phase III trial tests how well the addition of dinutuximab to Induction chemotherapy along with standard of care surgical resection of the primary tumor, radiation, stem cell transplantation, and immunotherapy works for treating children with newly diagnosed high-risk neuroblastoma. Dinutuximab is a monoclonal antibody that binds to a molecule called GD2, which is found on the surface of neuroblastoma cells, but is not present on many healthy or normal cells in the body. When dinutuximab binds to the neuroblastoma cells, it helps signal the immune system to kill the tumor cells. This helps the cells of the immune system kill the cancer cells, this is a type of immunotherapy. When chemotherapy and immunotherapy are given together, during the same treatment cycle, it is called chemoimmunotherapy. This clinical trial randomly assigns patients to receive either standard chemotherapy and surgery or chemoimmunotherapy (chemotherapy plus dinutuximab) and surgery during Induction therapy. Chemotherapy drugs administered during Induction include, cyclophosphamide, topotecan, cisplatin, etoposide, vincristine, and doxorubicin. These drugs work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing or by stopping them from spreading. Upon completion of 5 cycles of Induction therapy, a disease evaluation is completed to determine how well the treatment worked. If the tumor responds to therapy, patients receive a tandem transplantation with stem cell rescue. If the tumor has little improvement or worsens, patients receive chemoimmunotherapy on Extended Induction. During Extended Induction, dinutuximab is given with irinotecan, temozolomide. Patients with a good response to therapy move on to Consolidation therapy, when very high doses of chemotherapy are given at two separate points to kill any remaining cancer cells. Following, transplant, radiation therapy is given to the site where the cancer originated (primary site) and to any other areas that are still active at the end of Induction. The final stage of therapy is Post-Consolidation. During Post-Consolidation, dinutuximab is given with isotretinoin, with the goal of maintaining the response achieved with the previous therapy. Adding dinutuximab to Induction chemotherapy along with standard of care surgical resection of the primary tumor, radiation, stem cell transplantation, and immunotherapy may be better at treating children with newly diagnosed high-risk neuroblastoma.
Read the detailed description
PRIMARY OBJECTIVE:
- To determine if the event-free survival (EFS) of patients with newly diagnosed high-risk neuroblastoma assigned to early chemoimmunotherapy during Induction differs from that of patients who are not assigned to treatment that includes early chemoimmunotherapy.
SECONDARY OBJECTIVES:
- To determine if early chemoimmunotherapy during Induction therapy improves end of Induction (EOI) response rates and overall survival (OS) for patients with newly diagnosed high-risk neuroblastoma.
II. To determine response rates, EFS, and OS following an Extended Induction regimen with chemoimmunotherapy in patients with progressive disease or a poor response to Induction therapy.
III. To compare the toxicities experienced by patients treated with chemoimmunotherapy during Induction versus those experienced by patients treated with standard Induction and to describe toxicities experienced during Extended Induction.
IV. To determine GD2 expression on tumor tissue and tumor cells in bone marrow and assess for associations with response and outcome.
EXPLORATORY OBJECTIVES:
- To describe the association between tumor and host factors and outcomes in patients receiving protocol therapy.
II. To evaluate circulating biomarkers and markers of minimal residual disease at baseline and during therapy, and assess for associations with response and outcome.
III. To compare patterns of failure between patients treated with and without dinutuximab during induction.
IV. To determine the effect of telomere maintenance mechanisms on end of Induction response rates, EFS, and OS.
- To explore the impact of high-risk neuroblastoma (HRNBL) and its therapy, including the addition of dinutuximab to Induction chemotherapy, on functional and quality of life outcomes in patients with HRNBL, as measured by caregiver (parent/legal guardian) and patient questionnaires.
VI. To describe the adequacy of diagnostic biopsy specimens, including those obtained by percutaneous core needle biopsy.
VII. To explore the associations between family-reported adverse social determinants of health and both clinical outcomes and biology.
VIII. To develop and validate deep learning predictors of Induction response based on diagnostic MIBG scans. (Imaging Objective) IX. To compare institutional versus central determination of overall response, individual response components (primary tumor, soft tissue and bone metastatic disease, and bone marrow metastatic disease), and poor end of induction response (PEIR) and good end of induction response (GEIR) determination. (Imaging Objective) X. To describe late toxicities (including impaired organ function, neuropsychiatric toxicity, and incidence of secondary malignancy) in patients treated with dinutuximab during Induction or Extended Induction to late toxicities in patients who have not received dinutuximab during these phases of therapy.
XI. To evaluate whether reduced dose radiotherapy to the primary site clinical target volume (CTV) in patients with complete response of the primary site at EOI results in comparable local control relative to historical cohorts.
XII. To compare post-transplant complications between treatment arms, and assess for associations with outcome.
XIII. To assess for associations between EOI response (including good end of Induction response [GEIR] and poor end of Induction response [PEIR]) and individual response components (primary tumor, soft tissue and bone metastatic disease, and bone marrow metastatic disease) with outcome (EFS and OS).
XIV. To describe and compare the changes in image-defined risk factors (IDRFs) between patients treated with and without dinutuximab during Induction and associate with surgical outcomes and local failure rates following primary tumor resection.
XV. To bank serial samples of blood, bone marrow, and tumor tissue for future research.
OUTLINE: Patients receive Induction cycle 1 and are then randomized to 1 of 2 treatment arms.
INDUCTION CYCLE 1: Patients receive cyclophosphamide intravenously (IV) over 30 minutes and topotecan IV over 30 minutes on days 1-5 in the absence of unacceptable toxicity.
ARM A:
INDUCTION CYCLES 2-5: Patients receive cyclophosphamide IV over 30 minutes and topotecan IV over 30 minutes on days 1-5 of cycle 2 in the absence of unacceptable toxicity. Patients then undergo stem cell harvest via apheresis. Patients then receive cisplatin IV over 4 hours and etoposide IV over 2 hours on days 1-3 of cycles 3 and 5, and vincristine IV on day 1, doxorubicin IV over 15 minutes, and cyclophosphamide IV over 1 hour on days 1-2 of cycle 4 in the absence of unacceptable toxicity. Patients undergo primary tumor resection after Induction cycle 4 or 5. Following Induction cycle 5, patients undergo testing to determine response to Induction therapy. Patients with a good tumor response proceed to Consolidation, while patients with a poor tumor response proceed to Extended Induction.
EXTENDED INDUCTION: Patients with a poor tumor response or progression during Induction receive temozolomide orally (PO), via nasogastric tube (NG), or via gastric tube (G-tube) on days 1-5, irinotecan IV over 90 minutes on days 1-5, and dinutuximab IV over 10 hours. Treatment repeats every 21 days for up to a maximum of 6 cycles in the absence of disease progression or unacceptable toxicity. If at any time during Extended Induction testing shows a good tumor response, patients proceed to Consolidation. If after 6 cycles of Extended Induction or if at any time progression is noted, patients are removed from the study.
CONSOLIDATION: Patients undergo two autologous hematopoietic stem cell transplantations (HSCTs) during Consolidation. Patients receive thiotepa IV over 2 hours on days -7 to -5 and cyclophosphamide IV over 1 hour on days -5 to -2 during HSCT 1. Patients then receive stem cell infusion IV on day 0. Between 6 and 10 weeks after stem cell infusion, patients receive melphalan IV over 30 minutes on days -7 to -5, etoposide IV over 24 hours on days -7 to -4, and carboplatin over 24 hours on days -7 to -4 during HSCT 2. Patients receive stem cell infusion IV on day 0. Between day +42 and day +80 after HSCT 2. Patients receive radiation daily for 12 treatments in the absence of disease progression or unacceptable toxicity.
POST CONSOLIDATION: Patients receive dinutuximab IV over 10 hours on days 4-7 and isotretinoin PO twice daily (BID) on days 11-24 of cycles 1-5. Treatment repeats every 28 days for up to 5 cycles in the absence of disease progression or unacceptable toxicity. Patients then receive isotretinoin PO BID on days 15-28 for 1 additional cycle, cycle 6.
Patients undergo blood and urine sample collection, echocardiogram (ECHO) or multigated acquisition scan (MUGA), bone marrow aspiration and/or biopsy, computed tomography (CT) scan, magnetic resonance imaging (MRI), iodine-123 meta-iodobenzylguanidine (I-MIBG) scan and possible fluorodeoxyglucose position emission tomography (FDG-PET) scan throughout the study.
ARM B:
INDUCTION CYCLES 2-5: Patients receive cyclophosphamide IV over 30 minutes, topotecan IV over 30 minutes on days 1-5, and dinutuximab IV over 10 hours on days 2-5 of cycle 2 in the absence of unacceptable toxicity. Patients then undergo stem cell harvest via apheresis. Patients receive cisplatin IV over 4 hours and etoposide IV over 2 hours on days 1-3 and dinutuximab IV over 10 hours on days 2-5 of cycles 3 and 5, and vincristine IV on day 1, doxorubicin IV over 15 minutes, and cyclophosphamide IV over 1 hour on days 1-2, and dinutuximab IV over 10 hours on days 2-5 of cycle 4 in the absence of unacceptable toxicity. Patients undergo primary tumor resection after Induction cycle 4 or 5. Following Induction cycle 5, patients undergo testing to determine response to Induction therapy. Patients with a good tumor response proceed to Consolidation, while patients with a poor tumor response proceed to Extended Induction.
EXTENDED INDUCTION: Patients with a poor tumor response or progression during Induction receive temozolomide PO, via NG tube, or via G-tube on days 1-5, irinotecan IV over 90 minutes on days 1-5, and dinutuximab IV over 10 hours. Treatment repeats every 21 days for up to a maximum of 6 cycles in the absence of disease progression or unacceptable toxicity. If at any time during Extended Induction testing shows a good tumor response, patients proceed to Consolidation. If after 6 cycles of Extended Induction or if at any time progression is noted, patients are removed from the study.
CONSOLIDATION: Patients undergo two autologous HSCTs during Consolidation. Patients receive thiotepa IV over 2 hours on days -7 to -5 and cyclophosphamide IV over 1 hour on days -5 to -2 during HSCT 1. Patients then receive stem cell infusion IV on day 0. Between 6 and 10 weeks after stem cell infusion patients receive melphalan IV over 30 minutes on days -7 to -5, etoposide IV over 24 hours on days -7 to -4, and carboplatin over 24 hours on days -7 to -4 during HSCT 2. Patients receive stem cell infusion IV on day 0. Between day +42 and day +80 after HSCT 2, patients receive radiation daily for 12 treatments in the absence of disease progression or unacceptable toxicity.
POST CONSOLIDATION: Patients receive dinutuximab IV over 10 hours on days 4-7 and isotretinoin PO BID on days 11-24 of cycles 1-5. Treatment repeats every 28 days for up to 5 cycles in the absence of disease progression or unacceptable toxicity. Patients then receive isotretinoin PO BID on days 15-28 for 1 additional cycle, cycle 6.
Patients undergo blood and urine sample collection, ECHO or MUGA, bone marrow aspiration and/or biopsy, CT scan, MRI, I-MIBG scan and possible FDG-PET scan throughout the study.
After completion of study treatment, patients are followed up at 3, 6, 9,12, 15, 18, 24, 30, 36, 42, 48, 54, and 60 months and then periodically for up to 10 years from enrollment.
- Conditions
- Ganglioneuroblastoma, Nodular, Neuroblastoma
- Being tested
- Biospecimen Collection, Bone Marrow Aspiration, Bone Marrow Biopsy, Carboplatin, Cisplatin
- Medical subject headings
- Ganglioneuroblastoma, Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Biospecimen CollectionProcedure or surgery
Also called Biological Sample Collection, Biospecimen Collected, Sample Collection, Specimen Collection
Undergo blood and urine sample collection
Bone Marrow AspirationProcedure or surgery
Undergo bone marrow aspiration
Bone Marrow BiopsyProcedure or surgery
Also called Biopsy of Bone Marrow, Biopsy, Bone Marrow
Undergo bone marrow biopsy
CarboplatinDrug
Also called Blastocarb, Carboplat, Carboplatin Hexal, Carboplatino
Given IV
CisplatinDrug
Also called Abiplatin, Blastolem, Briplatin, CDDP
Given IV
Computed TomographyProcedure or surgery
Also called CAT, CAT Scan, Computed Axial Tomography, Computerized Axial Tomography
Undergo CT scan
CyclophosphamideDrug
Also called (-)-Cyclophosphamide, 2H-1,3,2-Oxazaphosphorine, 2-[bis(2-chloroethyl)amino]tetrahydro-, 2-oxide, monohydrate, Asta B 518, B 518
Given IV
DinutuximabBiological
Also called Ch 14.18UTC, Ch14.18, Dinutuximab Beta, MOAB Ch14.18
Given IV
DoxorubicinDrug
Also called Adriablastin, Hydroxydaunomycin, Hydroxyl Daunorubicin, Hydroxyldaunorubicin
Given IV
Echocardiography TestProcedure or surgery
Also called EC, Echocardiography
Undergo ECHO
EtoposideDrug
Also called Demethyl Epipodophyllotoxin Ethylidine Glucoside, EPEG, Lastet, Toposar
Given IV
FDG-Positron Emission Tomography and Computed Tomography ScanProcedure or surgery
Also called FDG PET/CT
Undergo FDG PET
Hematopoietic Cell TransplantationProcedure or surgery
Also called HCT, Hematopoietic Stem Cell Infusion, HEMATOPOIETIC STEM CELL TRANSPLANT, Hematopoietic Stem Cell Transplantation
Undergo stem cell infusion
IrinotecanDrug
Given IV
IsotretinoinDrug
Also called 13-cis retinoic acid, 13-cis-Retinoate, 13-cis-Retinoic Acid, 13-cis-Vitamin A Acid
Given PO
LeukapheresisProcedure or surgery
Also called Leukocyte Adsorptive Apheresis, Leukocytopheresis, Therapeutic Leukopheresis, White Blood Cell Reduction Apheresis
Undergo apheresis
Magnetic Resonance ImagingProcedure or surgery
Also called Magnetic Resonance, Magnetic Resonance Imaging (MRI), Magnetic resonance imaging (procedure), Magnetic Resonance Imaging Scan
Undergo MRI
MelphalanDrug
Also called Alanine Nitrogen Mustard, CB-3025, L-PAM, L-Phenylalanine Mustard
Given IV
Multigated Acquisition ScanProcedure or surgery
Also called Blood Pool Scan, Equilibrium Radionuclide Angiography, Gated Blood Pool Imaging, Gated Heart Pool Scan
Undergo MUGA
Radiation TherapyRadiation
Also called Cancer Radiotherapy, Energy Type, ENERGY_TYPE, Irradiate
Undergo radiation therapy
Radionuclide ImagingProcedure or surgery
Also called Gamma Scan, NM, Nuclear Medicine, nuclear medicine scan
Undergo I-MIBG scan
Survey AdministrationOther
Ancillary studies
TemozolomideDrug
Also called CCRG-81045, Gliotem, Imidazo[5,1-d]-1,2,3,5-tetrazine-8-carboxamide, 3, 4-dihydro-3-methyl-4-oxo-, M & B 39831
Given PO or via NG or G tube
ThiotepaDrug
Also called 1,1',1''-Phosphinothioylidynetrisaziridine, Girostan, N,N', N''-Triethylenethiophosphoramide, Oncotiotepa
Given IV
TopotecanDrug
Also called Hycamptamine, Topotecan Lactone
Given IV
Tumor ResectionProcedure or surgery
Undergo tumor resection surgery
VincristineDrug
Also called LCR, Leurocristine, VCR, Vincrystine
Given IV
| Study group | Role | What they receive |
|---|---|---|
| Arm A (SOC treatment) | Active comparator | See detailed description Receives: Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration; Procedure: Bone Marrow Biopsy; Drug: Carboplatin; Drug: Cisplatin; Procedure: Computed Tomography; Drug: Cyclophosphamide; Biological: Dinutuximab; Drug: Doxorubicin; Procedure: Echocardiography Test; Drug: Etoposide; Procedure: FDG-Positron Emission Tomography and Computed Tomography Scan; Procedure: Hematopoietic Cell Transplantation; Drug: Irinotecan; Drug: Isotretinoin; Procedure: Leukapheresis; Procedure: Magnetic Resonance Imaging; Drug: Melphalan; Procedure: Multigated Acquisition Scan; Radiation: Radiation Therapy; Procedure: Radionuclide Imaging; Other: Survey Administration; Drug: Temozolomide; Drug: Thiotepa; Drug: Topotecan; Procedure: Tumor Resection; Drug: Vincristine |
| Arm B (Dinutuximab in induction) | Experimental | See detailed description Receives: Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration; Procedure: Bone Marrow Biopsy; Drug: Carboplatin; Drug: Cisplatin; Procedure: Computed Tomography; Drug: Cyclophosphamide; Biological: Dinutuximab; Drug: Doxorubicin; Procedure: Echocardiography Test; Drug: Etoposide; Procedure: FDG-Positron Emission Tomography and Computed Tomography Scan; Procedure: Hematopoietic Cell Transplantation; Drug: Irinotecan; Drug: Isotretinoin; Procedure: Leukapheresis; Procedure: Magnetic Resonance Imaging; Drug: Melphalan; Procedure: Multigated Acquisition Scan; Radiation: Radiation Therapy; Procedure: Radionuclide Imaging; Other: Survey Administration; Drug: Temozolomide; Drug: Thiotepa; Drug: Topotecan; Procedure: Tumor Resection; Drug: Vincristine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Event free survival (EFS)
EFS time is calculated from time of randomization to Arms A or B to first episode of disease relapse or progression, second malignancy, or death, or until last contact if no event has occurred.
Measured: Up to 3 years
Secondary outcomes (4)
End of Induction (EOI) response rate
The response rate will be calculated among all evaluable patients at end of Induction. Responders are defined as patients who achieve a >= partial response per the revised 2017 International Neuroblastoma Response Criteria (INRC)
Measured: From randomization to end of extended Induction, up to 12 months
Overall survival (OS)
OS time is calculated from time of randomization to Arms A or B until death, or until last contact if patient is alive.
Measured: Up to 3 years
Incidence of adverse events
The proportion of patients with at least one grade 3 or higher non-hematologic toxicity or grade 4 or higher hematologic toxicity during protocol therapy, graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events version 5.0, will be reported.
Measured: Up to 2 years
GD2 expression
Dinutuximab binding to pre-therapy patient tumor samples will be measured and categorized as high or low.
Measured: Up to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Dec 2023
- Study start19 Apr 2024
- Primary completion31 Dec 2029 (estimated)
- Study completion31 Dec 2029 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Ganglioneuroblastoma, NodularStudies of the same condition, open and under way.
- More trials run by National Cancer Institute (NCI)The sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 478 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Cancer Institute (NCI)
- Sponsor type
- US National Institutes of Health
- Responsible party
- Sponsor
- Investigators
- Sara M Federico, Principal investigator, Children's Oncology Group
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06172296
- Sponsor's protocol ID
- NCI-2023-08530
- Other IDs
- NCI-2023-08530, ANBL2131, ANBL2131, U10CA180886
- Status last verified
- Apr 2026
- First submitted
- 13 Dec 2023
- Last updated
- 21 Oct 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: "NCI is committed to sharing data in accordance with NIH policy. For more details on how clinical trial data is shared, access the link to the NIH data sharing policy page."