GanglioneuroblastomaPhase 3NCT03126916
Testing the Addition of 131I-MIBG or Lorlatinib to Intensive Therapy in People With High-Risk Neuroblastoma (NBL)
A Phase 3 study testing Autologous Hematopoietic Stem Cell Transplantation, Biospecimen Collection and Bone Marrow Aspiration and Biopsy in people with Ganglioneuroblastoma and Ganglioneuroblastoma, Nodular, run by Children's Oncology Group at 164 sites in 3 countries.
Trial summary
This phase III trial studies iobenguane I-131 or lorlatinib and standard therapy in treating younger patients with newly-diagnosed high-risk neuroblastoma or ganglioneuroblastoma. Radioactive drugs, such as iobenguane I-131, may carry radiation directly to tumor cells and not harm normal cells. Read more about this study
- Who is it for?
- Ages 365 days to 30 years
- Conditions the study is for
- Ganglioneuroblastoma, Ganglioneuroblastoma, Nodular, Neuroblastoma
- What is being tested?
- Autologous Hematopoietic Stem Cell Transplantation, Biospecimen Collection and Bone Marrow Aspiration and Biopsy
- Type of treatment
- Pill or oral medicine, Injection or infusion, Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 750 participants
- Where
- 164 sitesin 3 countries
- Study dates
- 14 May 2018to 30 Sep 2030
- 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 365 days to 30 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Patients must be enrolled on ANBL00B1 (NCT00904241) or APEC14B1 (NCT02402244) prior to enrollment on ANBL1531 (NCT03126916)
- FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Patient must be >= 365 days and =< 30 years of age at diagnosis
- FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Patients must have a diagnosis of neuroblastoma or ganglioneuroblastoma (nodular) verified by tumor pathology analysis or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites; the following disease groups are eligible:
- Patients with International Neuroblastoma Risk Group (INRG) stage M disease are eligible if found to have either of the following features:
- MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals), regardless of additional biologic features; OR
- Age > 547 days regardless of biologic features
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- Patients with INRG stage MS disease with MYCN amplification
- Patients with INRG stage L2 disease with MYCN amplification
- Patients > 547 days of age initially diagnosed with INRG stage L1, L2 or MS disease who progressed to stage M without prior chemotherapy may enroll within 4 weeks of progression to stage M
- Patients >= 365 days of age initially diagnosed with MYCN amplified INRG stage L1 disease who progress to stage M without systemic therapy may enroll within 4 weeks of progression to stage M
- FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Patients initially recognized to have high-risk disease must have had no prior systemic therapy (other than topotecan/cyclophosphamide initiated on an emergent basis and within allowed timing); 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 will also be eligible; patients who receive localized emergency radiation to sites of life-threatening or function-threatening disease prior to or immediately after establishment of the definitive diagnosis will be eligible
- FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or a serum creatinine based on age/sex as follows:
- 1 to < 2 years: male = 0.6; female = 0.6
- 2 to < 6 years: male = 0.8; female = 0.8
- 6 to < 10 years: male = 1; female = 1
- 10 to < 13 years: male = 1.2; female = 1.2
- 13 to < 16 years: male = 1.5; female = 1.4
- >= 16 years: male = 1.7; female = 1.4
- FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Total bilirubin =< 1.5 x upper limit of normal (ULN) for age, and
- FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) < 10 x ULN; for the purposes of this study, ULN for SGPT (ALT) is 45
- FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Shortening fraction of >= 27% by echocardiogram, or ejection fraction of > 50% by echocardiogram or radionuclide angiogram
- FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: No known contraindication to peripheral blood stem cell (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
- PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): See ANBL2131 (NCT06172296) protocol for eligible high-risk neuroblastoma diagnoses
- PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): In addition, all patients transferring from ANBL2131 (NCT06172296) to ANBL1531 (NCT03126916) Arm E must have tumors with an ALK aberration
- PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): Given the lack of data with lorlatinib in infant populations, patients transferring from ANBL2131 (NCT06172296) to ANBL1531 (NCT03126916) must be > 1 year of age at time of transfer to ANBL1531 (NCT03126916). Patients < 1 year of age found to have a qualifying ALK alteration as part of ANBL2131 (NCT06172296) may continue to participate in ANBL2131 (NCT06172296)
- PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): Patients initially recognized to have high-risk disease must have received no more than one cycle of topotecan/cyclophosphamide either after enrollment to ANBL2131 (NCT06172296) or started emergently prior to enrollment to ANBL2131 (NCT06172296)
- PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): Patients may have received up to one cycle of intermediate risk chemotherapy prior to initial enrollment to ANBL2131 (NCT06172296)
- PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): Patients may have received localized emergency radiation to sites of life-threatening or function-threatening disease prior to or immediately after establishment of the definitive diagnosis
- PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): In order to facilitate patient transfer and ensure timely distribution of lorlatinib, there are no blood count requirements to meet at time of transfer from ANBL2131 (NCT06172296) to ANBL1531 ((NCT03126916) Arm E. Note the blood count criteria that must be met prior to start of Induction cycle 2 on Arm E. Lorlatinib therapy should start no sooner than day 1 of Induction cycle 2
- PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): No known irreversible grade 2 or greater atrioventricular (AV) block
- PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): Due the potential psychiatric risks from lorlatinib, patients should not have a personal history of a serious psychiatric disorder requiring pharmacologic intervention or severe enough to be considered life-threatening
- PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): No known contraindication to PBSC collection. Examples of contraindications might be a weight or size less than the collecting institution deems 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
- FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Patients with INRG stage L2 tumors without amplification of MYCN regardless of tumor histology (may meet criteria for high risk classification but are not eligible for this trial)
- FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Patients with bone marrow failure syndromes
- FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Patients for whom targeted radiopharmaceutical therapy would be contraindicated due to underlying medical disorders
- FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: 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 for female patients of childbearing potential
- FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Lactating females who plan to breastfeed their infants
- FOR PATIENTS ENROLLING TO ANBL1531 (NCT03126916) WITHOUT PRIOR ANBL2131 (NCT06172296) ENROLLMENT: Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation
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- PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): Patients who have previously received treatment with lorlatinib or other ALK inhibitor
- PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): Patients who have undergone treatment arm randomization callback or started induction cycle 2 on ANBL2131 (NCT06172296)
- PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): Patients who have an INRG Stage L2 tumor without amplification of MYCN regardless of tumor histology (may meet criteria for high risk classification but are not eligible for this trial)
- PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): Patients with bone marrow failure syndromes
- PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): 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 for female patients of childbearing potential
- PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): Lactating females who plan to breastfeed their infants
- PATIENTS WITH TUMORS HARBORING ALK ALTERATIONS TRANSFERRING TO ANBL1531 (NCT03126916) ARM E FROM ANBL2131 (NCT06172296) (EFFECTIVE WITH AMENDMENT 13C): Sexually active patients of reproductive potential who have not agreed to use an effective contraceptive method for the duration of their study participation
Trial locations and contacts
164 sites in 3 countries. Contact the study team or a site near you to ask about taking part.
164 sites in 3 countries
United States152 sites, 143 recruiting
- Recruiting
Albany Medical Center
Albany, New York, 12208
Site Public Contact, 518-262-5513
Lauren R. Weintraub
- Recruiting
University of New Mexico Cancer Center
Albuquerque, New Mexico, 87106
Site Public Contact, 505-925-0348, HSC-ClinicalTrialInfo@salud.unm.edu
Jessica M. Valdez
- 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
Margaret E. Macy
- 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
- Suspended
Walter Reed National Military Medical Center
Bethesda, Maryland, 20889-5600
- Recruiting
Children's Hospital of Alabama
Birmingham, Alabama, 35233
Site Public Contact, 205-638-9285, oncologyresearch@peds.uab.edu
Elizabeth D. Alva
- Recruiting
Saint Luke's Cancer Institute - Boise
Boise, Idaho, 83712
Site Public Contact, 208-381-2774, eslinget@slhs.org
Martha M. Pacheco
- Active, not recruiting
Tufts Children's Hospital
Boston, Massachusetts, 02111
- Recruiting
Massachusetts General Hospital Cancer Center
Boston, Massachusetts, 02114
Site Public Contact, 877-726-5130
Suzanne Shusterman
- Recruiting
Dana-Farber Cancer Institute
Boston, Massachusetts, 02215
Site Public Contact, 877-442-3324
Suzanne Shusterman
- Recruiting
Maimonides Medical Center
Brooklyn, New York, 11219
Site Public Contact, 718-765-2500
Mahmut Y. Celiker
- 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
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
Susan L. Cohn
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Site Public Contact, 513-636-2799, cancer@cchmc.org
Robin E. Norris
- 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
- Active, not recruiting
Medical City Dallas Hospital
Dallas, Texas, 75230
- 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
Rocky Mountain Hospital for Children-Presbyterian Saint Luke's Medical Center
Denver, Colorado, 80218
Site Public Contact, 303-832-2344, PSGResearchSharedMailbox@HCAHealthcare.com
Florence Choo
- Recruiting
Blank Children's Hospital
Des Moines, Iowa, 50309
Site Public Contact, 515-241-8912, samantha.mallory@unitypoint.org
Samantha L. Mallory
- Recruiting
Children's Hospital of Michigan
Detroit, Michigan, 48201
Site Public Contact, helpdesk@childrensoncologygroup.org
Roland L. Chu
- Active, not recruiting
Wayne State University/Karmanos Cancer Institute
Detroit, Michigan, 48201
- Active, not recruiting
Henry Ford Health Saint John Hospital
Detroit, Michigan, 48236
- Recruiting
Kaiser Permanente Downey Medical Center
Downey, California, 90242
Site Public Contact, 626-564-3455
Robert M. Cooper
- Recruiting
Duke University Medical Center
Durham, North Carolina, 27710
Site Public Contact, 888-275-3853
Jessica M. Sun
- Suspended
Michigan State University
East Lansing, Michigan, 48823
- 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
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
- Recruiting
Saint Vincent Hospital Cancer Center Green Bay
Green Bay, Wisconsin, 54301
Site Public Contact, 920-433-8889, WI_research_admin@hshs.org
Catherine A. Long
- 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
Penn State Children's Hospital
Hershey, Pennsylvania, 17033
Site Public Contact, 717-531-6012
Lisa M. McGregor
- Recruiting
Memorial Regional Hospital/Joe DiMaggio Children's Hospital
Hollywood, Florida, 33021
Site Public Contact, 954-265-1847, OHR@mhs.net
Iftikhar Hanif
- Suspended
Kapiolani Medical Center for Women and Children
Honolulu, Hawaii, 96826
- 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
Riley Hospital for Children
Indianapolis, Indiana, 46202
Site Public Contact, 800-248-1199
Sandeep Batra
- 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
Vibhuti Agarwal
- 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
University Medical Center of Southern Nevada
Las Vegas, Nevada, 89102
Site Public Contact, 702-384-0013, research@sncrf.org
Alan K. Ikeda
- Recruiting
Sunrise Hospital and Medical Center
Las Vegas, Nevada, 89109
Site Public Contact, 702-384-0013, research@sncrf.org
Alan K. Ikeda
- Recruiting
Alliance for Childhood Diseases/Cure 4 the Kids Foundation
Las Vegas, Nevada, 89135
Site Public Contact, 702-384-0013, research@sncrf.org
Alan K. Ikeda
- Recruiting
Summerlin Hospital Medical Center
Las Vegas, Nevada, 89144
Site Public Contact, 702-384-0013, research@sncrf.org
Alan K. Ikeda
- 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
Children's Hospital Los Angeles
Los Angeles, California, 90027
Site Public Contact, 323-361-4110
Araz Marachelian
- Recruiting
Mattel Children's Hospital UCLA
Los Angeles, California, 90095
Site Public Contact, 310-825-6708
Satiro N. De Oliveira
- 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
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
Maggie E. Fader
- 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
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 ext. 6163, kcovert@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
Ochsner Medical Center Jefferson
New Orleans, Louisiana, 70121
Site Public Contact, 504-842-8084, Elisemarie.curry@ochsner.org
Craig Lotterman
- Suspended
Laura and Isaac Perlmutter Cancer Center at NYU Langone
New York, New York, 10016
- 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
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
Nemours Children's Hospital
Orlando, Florida, 32827
Site Public Contact, 302-651-5572, Allison.bruce@nemours.org
Vibhuti Agarwal
- 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
Phoenix Childrens Hospital
Phoenix, Arizona, 85016
Site Public Contact, 602-546-0920
Francis K. Eshun
- 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
Rhode Island Hospital
Providence, Rhode Island, 02903
Site Public Contact, 401-444-1488
Bradley DeNardo
- Active, not recruiting
VCU Massey Comprehensive Cancer Center
Richmond, Virginia, 23298
- Recruiting
Mayo Clinic in Rochester
Rochester, Minnesota, 55905
Site Public Contact, 855-776-0015
Wendy Allen-Rhoades
- Recruiting
University of Rochester
Rochester, New York, 14642
Site Public Contact, 585-275-5830
Jeffrey R. Andolina
- Recruiting
Corewell Health Children's
Royal Oak, Michigan, 48073
Site Public Contact, 248-551-7695
Marie V. Nelson
- 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
Anne-Marie R. Langevin
- Recruiting
Rady Children's Hospital - San Diego
San Diego, California, 92123
Site Public Contact, 858-966-5934
William D. Roberts
- Recruiting
Naval Medical Center -San Diego
San Diego, California, 92134
Site Public Contact, 619-532-8712
Yoko T. Udaka
- Recruiting
UCSF Medical Center-Mission Bay
San Francisco, California, 94158
Site Public Contact, 877-827-3222, cancertrials@ucsf.edu
Kieuhoa T. Vo
- Recruiting
Memorial Health University Medical Center
Savannah, Georgia, 31404
Site Public Contact, 912-350-7887, Lorraine.OHara@hcahealthcare.com
Andrew L. Pendleton
- Recruiting
Maine Children's Cancer Program
Scarborough, Maine, 04074
Site Public Contact, 207-396-8670, clinicalresearch@mainehealth.org
Eric C. Larsen
- 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
Stony Brook University Medical Center
Stony Brook, New York, 11794
Site Public Contact, 800-862-2215
Laura E. Hogan
- 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
Madigan Army Medical Center
Tacoma, Washington, 98431
Site Public Contact, 253-968-6144, melissa.a.forouhar.mil@health.mil
Melissa A. Forouhar
- 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
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
Alfred I duPont Hospital for Children
Wilmington, Delaware, 19803
Site Public Contact, 302-651-5572, Allison.bruce@nemours.org
Vibhuti Agarwal
- Recruiting
Wake Forest University Health Sciences
Winston-Salem, North Carolina, 27157
Site Public Contact, 336-713-6771
Sarah Supples
Canada11 sites, 10 recruiting
- 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
Kingston Health Sciences Centre
Kingston, Ontario, K7L 2V7
Site Public Contact, 613-549-6666, cc-clinicaltrials@kgh.kari.net
Laura Wheaton
- Recruiting
Children's Hospital
London, Ontario, N6A 5W9
Site Public Contact, 519-685-8306
Shayna M. Zelcer
- 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
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
- Suspended
British Columbia Children's Hospital
Vancouver, British Columbia, V6H 3V4
- Recruiting
CancerCare Manitoba
Winnipeg, Manitoba, R3E 0V9
Site Public Contact, 866-561-1026, ctu_web@cancercare.mb.ca
Stephanie M. Villeneuve
Puerto Rico1 site
- Active, not recruiting
HIMA San Pablo Oncologic Hospital
Caguas, 00726
About this study
Official title: A Phase 3 Study of 131I-Metaiodobenzylguanidine (131I-MIBG) or ALK Inhibitor Therapy Added to Intensive Therapy for Children With Newly Diagnosed High-Risk Neuroblastoma (NBL)
This phase III trial studies iobenguane I-131 or lorlatinib and standard therapy in treating younger patients with newly-diagnosed high-risk neuroblastoma or ganglioneuroblastoma. Radioactive drugs, such as iobenguane I-131, may carry radiation directly to tumor cells and not harm normal cells. Lorlatinib may stop the growth of tumor cells by blocking some of the enzymes needed for cell growth. Giving iobenguane I-131 or lorlatinib and standard therapy may work better compared to lorlatinib and standard therapy alone in treating younger patients with neuroblastoma or ganglioneuroblastoma.
Read the detailed description
PRIMARY OBJECTIVES:
- To determine in the context of a randomized trial whether the event-free survival (EFS) of patients with newly diagnosed high-risk neuroblastoma (NBL) is improved with the addition of iobenguane I-131 (131I-MIBG) during induction, prior to tandem autologous stem cell transplantation (ASCT).
II. To determine whether the addition of lorlatinib to intensive multimodality therapy for patients with high-risk NBL whose tumors harbor activating point mutations in the ALK gene with a variant allele frequency (VAF) >= 5% results in superior EFS compared to a contemporaneously treated cohort of patients with tumors without documented ALK activating mutations.
SECONDARY OBJECTIVES:
- To describe the toxicities associated with treatment for high-risk NBL with and without the addition of 131I-MIBG or ALK inhibitor therapy.
II. To estimate EFS and describe toxicity in patients with newly diagnosed high-risk NBL randomized to treatment with an 131I-MIBG-containing induction prior to busulfan/melphalan (BuMel) ASCT.
III. To describe the overall survival (OS) and response rates (evaluated per International Neuroblastoma Response Criteria [INRC] criteria prior to ASCT and prior to post-consolidation therapy) for patients with high-risk neuroblastoma treated with or without 131I-MIBG or ALK inhibitor therapy.
IV. To prospectively evaluate the relationship of response rate per revised International Neuroblastoma Response Criteria (INRC) to EFS and OS in patients with high-risk NBL treated with and without the addition of 131I-MIBG or ALK inhibitor therapy.
EXPLORATORY OBJECTIVES:
- To evaluate whole body radiation dose, tumor factors, and host factors as potential predictors of efficacy and/or toxicity associated with 131I-MIBG therapy and transplant conditioning.
II. To describe end-Induction response, EFS, and OS according to specific ALK mutations, VAF, ALK amplification, the presence of additional genomic findings, or the ALK inhibitor administered.
III. To characterize changes in tumor markers (circulating tumor deoxyribonucleic acid [DNA], including ALK and other tumor specific genetic aberrations, and circulating GD2) over time in response to protocol therapy.
IV. To correlate results of tumor and host profiling with end-induction response and EFS.
- To prospectively evaluate EFS for patients with MIBG non-avid high-risk NBL compared to patients with MIBG-avid high-risk NBL who are randomized to treatment without 131I-MIBG.
VI. To correlate Curie scores calculated from 131I-MIBG post-treatment scans with end-induction response, EFS and OS.
VII. To describe changes in image defined risk factors (IDRFs) over the course of induction therapy, with correlation to surgical outcomes and local failure rates following primary tumor resection.
VIII. To define patterns of failure at time of first relapse or progression in patients with high-risk NBL.
IX. To determine the feasibility of prospectively monitoring adverse events using electronic health records.
- To compare local, central, and computer assisted Curie score assignment at baseline and during therapy in patients with MIBG-avid high-risk NBL.
XI. To compare late toxicities (including impaired organ function and secondary tumor occurrence) in patients treated with 131I-MIBG or ALK inhibitor therapy to late toxicities in patients who have not received these therapies.
XII. To determine the association between household material hardship (HMH) and clinical outcomes, including event free and overall survival, and 131I-MIBG receipt.
XIII. To compare the outcomes (EFS, OS, and toxicity) of patients treated with post-consolidation therapy that does not contain aldesleukin to historical outcome data for patients treated with similar induction and consolidation regimens followed by post-consolidation therapy that contained aldesleukin.
XIV. To characterize and describe longitudinal neuropsychological and behavioral effects of high-risk neuroblastoma therapy.
XV. To evaluate change in neurobehavioral outcomes over time in patients with neuroblastoma treated with high-risk neuroblastoma therapy plus lorlatinib compared to high-risk therapy alone using parent- or self-report measures of adaptive, executive, and psychosocial functioning.
XVI. To characterize the pharmacokinetics and pharmaceutical properties of lorlatinib in children with high-risk neuroblastoma.
XVII. To compare the EFS of patients enrolled on Arm E and treated with lorlatinib to the EFS of a historical control comprised of patients with ALK aberrant disease treated on ANBL0532 (NCT00567567).
XVIII. To describe the EFS of patients enrolled on Arm E according to lorlatinib exposure during post-consolidation.
OUTLINE: Patients are randomized or assigned to 1 of 5 arms.
All patients receive cyclophosphamide intravenously (IV) over 15-30 minutes and topotecan hydrochloride IV over 30 minutes on days 1-5 during cycle 1 of induction therapy in the absence of disease progression or unacceptable toxicity. Patients not assigned to an Arm by the end of cycle 1 may receive an addition cycle of cyclophosphamide and topotecan.
ARM A (CLOSED TO ACCRUAL AS OF SEPTEMBER 28, 2023):
INDUCTION THERAPY: Patients receive cyclophosphamide IV over 15-30 minutes and topotecan hydrochloride IV over 30 minutes on days 1-5 of cycle 2 and cisplatin IV over 4 hours and etoposide phosphate IV over 2 hours on days 1-3 of cycles 3 and 5. Patients also receive vincristine sulfate IV over 1 minute on day 1 and dexrazoxane hydrochloride IV over 5-15 minutes, doxorubicin hydrochloride IV over 1-15 minutes, and cyclophosphamide IV over 1-6 hours on days 1-2 of cycle 4 in the absence of disease progression or unacceptable toxicity.
CONSOLIDATION THERAPY:
HSCT#1: Patients receive thiotepa IV over 2 hours on days -7 to -5 and cyclophosphamide IV over 1 hour on days -5 to -2 in the absence of disease progression or unacceptable toxicity.
HSCT#2: Patients receive melphalan hydrochloride IV over 30 minutes on days -7 to -5, and etoposide phosphate IV over 24 hours and carboplatin IV over 24 hours on days -7 to -4 in the absence of disease progression or unacceptable toxicity.
POST-CONSOLIDATION THERAPY: Patients receive sargramostim subcutaneously (SC) on days 1-14, dinutuximab IV over 10 hours on days 4-7 of cycles 1-5, and isotretinoin orally (PO) twice daily (BID) on days 11-24 of cycles 1-5, and days 15-28 during cycle 6 in the absence of disease progression or unacceptable toxicity.
Patients undergo echocardiography or multigated acquisition (MUGA) scan, magnetic resonance imaging (MRI) or computed tomography (CT) scan, receive 123I-MIBG and undergo MIBG imaging, bone marrow aspiration and biopsy and blood sample collection throughout the study.
ARM B (CLOSED TO ACCRUAL AS OF SEPTEMBER 28, 2023):
INDUCTION THERAPY: Patients receive cyclophosphamide, topotecan hydrochloride, cisplatin, and etoposide phosphate as in Arm A, iobenguane I-131 IV over 1.5-2 hours on day 1 beginning 3 weeks after the start of cycle 3, and vincristine sulfate, dexrazoxane hydrochloride, doxorubicin hydrochloride, and cyclophosphamide as in Arm A beginning no sooner than 35 days after the infusion of iobenguane I-131.
CONSOLIDATION THERAPY:
HSCT#1: Patients receive thiotepa and cyclophosphamide as in Arm A.
HSCT#2: Patients receive melphalan, etoposide phosphate, and carboplatin as in Arm A.
POST-CONSOLIDATION THERAPY: Patients receive sargramostim, dinutuximab, and isotretinoin as in Arm A-D.
Patients undergo echocardiography or MUGA scan, MRI or CT scan, receive 123I-MIGB and undergo MIBG imaging, bone marrow aspiration and biopsy and blood sample collection throughout the study.
ARM C (CLOSED TO ACCRUAL AS OF DECEMBER 17, 2020):
INDUCTION THERAPY: Patients receive cyclophosphamide, topotecan hydrochloride, cisplatin, etoposide phosphate, iobenguane I-131, vincristine sulfate, dexrazoxane hydrochloride, doxorubicin hydrochloride, and cyclophosphamide as in Arm B.
CONSOLIDATION THERAPY: Patients receive busulfan IV over 3 hours on days -6 to -3 and melphalan hydrochloride IV over 30 minutes on day -1 in the absence of disease progression or unacceptable toxicity.
POST-CONSOLIDATION THERAPY: Patients receive sargramostim, dinutuximab, and isotretinoin as in Arm A.
Patients undergo echocardiography or MUGA scan, MRI or CT scan, receive 123I-MIGB and undergo MIBG imaging, bone marrow aspiration and biopsy and blood sample collection throughout the study.
ARM D (CLOSED TO ACCRUAL AS OF SEPTEMBER 28, 2023): Patients receive treatment identical to Arm A.
Patients undergo echocardiography or MUGA scan, MRI or CT scan, receive 123I-MIGB and undergo MIBG imaging, bone marrow aspiration and biopsy and blood sample collection throughout the study and may undergo fludeoxyglucose- positron emission tomography (PET) scan on study.
ARM E:
INDUCTION THERAPY: Patients receive cyclophosphamide, topotecan hydrochloride, cisplatin, etoposide phosphate, vincristine sulfate, dexrazoxane hydrochloride, doxorubicin hydrochloride, and cyclophosphamide as in Arm A. Patients also receive lorlatinib PO once daily (QD) starting with cycle 2 and continue until HSCT #1 in the absence of disease progression or unacceptable toxicity. For patients transferring from ANBL2131 (NCT06172296) to ANBL1531 (NCT03126916) Arm E, protocol therapy on ANBL1531 (NCT03126916) begins with induction cycle 2 on Arm E (topotecan, cyclophosphamide and lorlatinib).
CONSOLIDATION THERAPY:
HSCT#1: Patients receive thiotepa and cyclophosphamide as in Arm A. Patients also receive lorlatinib PO QD until day -8 of HSCT#2 in the absence of disease progression or unacceptable toxicity.
HSCT#2: Patients receive melphalan hydrochloride, etoposide phosphate, carboplatin as in Arm A. Lorlatinib is restarted when patient has reached at least day +14 post-HSCT#2 and is able to tolerate enteral medications, provided there is no evidence of disease progression or unacceptable toxicity.
RADIATION THERAPY: Patients receive lorlatinib PO QD concurrently with radiation therapy in the absence of disease progression or unacceptable toxicity.
POST-CONSOLIDATION THERAPY: Patients receive sargramostim and dinutuximab as in Arm A-D. Patients also receive isotretinoin PO BID on days 11-24 of cycles 1-5 and days 15-28 of cycle 6, and lorlatinib PO QD on days 15-28 of cycles 2-5 and days 1-28 of cycle 6 in the absence of disease progression or unacceptable toxicity.
CONTINUATION THERAPY: Patients receive lorlatinib PO QD on days 1-28. Cycles repeat every 28 days for 18 months in the absence of disease progression or unacceptable toxicity.
Patients undergo echocardiography or MUGA scan, MRI or CT scan, undergo MIBG imaging, bone marrow aspiration and biopsy and blood sample collection throughout the study and may undergo PET scan on study.
After completion of study therapy, patients in Arms A-D are followed up every 3 months for 18 months, and then every 6 months for 42 months; patients in Arm E are followed up every 3 months for 6 months, and then every 6 months for 42 months.
- Being tested
- Autologous Hematopoietic Stem Cell Transplantation, Biospecimen Collection, Bone Marrow Aspiration and Biopsy, Busulfan, Carboplatin
- Medical subject headings
- Ganglioneuroblastoma, Neuroblastoma
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Autologous Hematopoietic Stem Cell TransplantationProcedure or surgery
Also called AHSCT, Autologous, Autologous Hematopoietic Cell Transplantation, Autologous Stem Cell Transplant
Undergo autologous HSCT
Biospecimen CollectionProcedure or surgery
Also called Biological Sample Collection, Biospecimen Collected, Specimen Collection
Undergo blood sample collection
Bone Marrow Aspiration and BiopsyProcedure or surgery
Undergo bone marrow aspiration and biopsy
BusulfanDrug
Also called 1, 4-Bis[methanesulfonoxy]butane, BUS, Busilvex, Bussulfam
Given IV
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
Dexrazoxane HydrochlorideDrug
Also called Cardioxane, Totect, Zinecard
Given IV
DinutuximabBiological
Also called Ch 14.18UTC, Ch14.18, Dinutuximab Beta, MOAB Ch14.18
Given IV
Also called 5,12-Naphthacenedione, 10-[(3-amino-2,3,6-trideoxy-alpha-L-lyxo-hexopyranosyl)oxy]-7,8, 9,10-tetrahydro-6,8,11-trihydroxy-8-(hydroxyacetyl)-1-methoxy-, hydrochloride, (8S-cis)- (9CI), ADM, Adriacin, Adriamycin
Given IV
Echocardiography TestProcedure or surgery
Also called EC, Echocardiography
Undergo echocardiography
Etoposide PhosphateDrug
Also called Etopophos
Given IV
External Beam Radiation TherapyRadiation
Also called Definitive Radiation Therapy, EBRT, External Beam Radiation, External Beam Radiotherapy
Undergo EBRT
Iobenguane I-123Radiation
Also called 123I-Metaiodobenzylguanidine, 123I-MIBG, AdreView, I-123-MIBG
Given 123 I-MIBG
Iobenguane I-131Radiation
Also called (131)I-MIBG, 131I-MIBG, I 131 Meta-iodobenzylguanidine, I-131 Metaiodobenzylguanidine
Given IV
IsotretinoinDrug
Also called 13-cis retinoic acid, 13-cis-Retinoate, 13-cis-Retinoic Acid, 13-cis-Vitamin A Acid
Given PO
LorlatinibDrug
Also called 2H-4,8-Methenopyrazolo(4,3-H)(2,5,11)benzoxadiazacyclotetradecine-3-carbonitrile, 7-amino-12-fluoro-10,15,16,17-tetrahydro-2,10,16-trimethyl-15-oxo-, (10R)-, Lorbrena, Lorviqua, PF 06463922
Given PO
Magnetic Resonance ImagingProcedure or surgery
Also called Magnetic Resonance, Magnetic Resonance Imaging (MRI), Magnetic resonance imaging (procedure), Magnetic Resonance Imaging Scan
Undergo MRI
Melphalan HydrochlorideDrug
Also called Alkeran, Alkerana, Evomela, Hepzato
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 scan
Positron Emission TomographyProcedure or surgery
Also called Medical Imaging, Positron Emission Tomography, PET, PET Scan, Positron emission tomography (procedure)
Undergo PET scan
SargramostimBiological
Also called 23-L-Leucinecolony-Stimulating Factor 2, DRG-0012, Leukine, Prokine
Given SC
Therapeutic Conventional SurgeryProcedure or surgery
Undergo standard of care surgery
ThiotepaDrug
Also called 1,1',1''-Phosphinothioylidynetrisaziridine, Girostan, N,N', N''-Triethylenethiophosphoramide, Oncotiotepa
Given IV
Topotecan HydrochlorideDrug
Also called Evotopin, Hycamptamine, Hycamtin, Nogitecan Hydrochloride
Given IV
Also called Kyocristine, Leurocristine Sulfate, Leurocristine, sulfate, Oncovin
Given IV
| Study group | Role | What they receive |
|---|---|---|
| Arm A (chemotherapy, HSCT, EBRT) | Experimental | See Arm A in detailed description. Receives: Procedure: Autologous Hematopoietic Stem Cell Transplantation; Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration and Biopsy; Drug: Carboplatin; Drug: Cisplatin; Procedure: Computed Tomography; Drug: Cyclophosphamide; Drug: Dexrazoxane Hydrochloride; Biological: Dinutuximab; Drug: Doxorubicin Hydrochloride; Procedure: Echocardiography Test; Drug: Etoposide Phosphate; Radiation: External Beam Radiation Therapy; Radiation: Iobenguane I-123; Drug: Isotretinoin; Procedure: Multigated Acquisition Scan; Procedure: Positron Emission Tomography; Biological: Sargramostim; Procedure: Therapeutic Conventional Surgery; Drug: Thiotepa; Drug: Topotecan Hydrochloride; Drug: Vincristine Sulfate |
| Arm B (Iobenguane I-131, chemotherapy, HSCT, EBRT) | Experimental | See Arm B in detailed description. Receives: Procedure: Autologous Hematopoietic Stem Cell Transplantation; Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration and Biopsy; Drug: Carboplatin; Drug: Cisplatin; Procedure: Computed Tomography; Drug: Cyclophosphamide; Drug: Dexrazoxane Hydrochloride; Biological: Dinutuximab; Drug: Doxorubicin Hydrochloride; Procedure: Echocardiography Test; Drug: Etoposide Phosphate; Radiation: External Beam Radiation Therapy; Radiation: Iobenguane I-123; Radiation: Iobenguane I-131; Drug: Isotretinoin; Procedure: Magnetic Resonance Imaging; Procedure: Multigated Acquisition Scan; Procedure: Positron Emission Tomography; Biological: Sargramostim; Procedure: Therapeutic Conventional Surgery; Drug: Thiotepa; Drug: Topotecan Hydrochloride; Drug: Vincristine Sulfate |
| Arm C (Iobenguane I-131, chemotherapy, BuMel, HSCT, EBRT) | Experimental | See Arm C in detailed description. Closed to accrual as of 12/17/20. Receives: Procedure: Autologous Hematopoietic Stem Cell Transplantation; Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration and Biopsy; Drug: Busulfan; Drug: Cisplatin; Procedure: Computed Tomography; Drug: Cyclophosphamide; Drug: Dexrazoxane Hydrochloride; Biological: Dinutuximab; Drug: Doxorubicin Hydrochloride; Procedure: Echocardiography Test; Drug: Etoposide Phosphate; Radiation: External Beam Radiation Therapy; Radiation: Iobenguane I-131; Drug: Isotretinoin; Procedure: Magnetic Resonance Imaging; Drug: Melphalan Hydrochloride; Procedure: Multigated Acquisition Scan; Procedure: Positron Emission Tomography; Biological: Sargramostim; Procedure: Therapeutic Conventional Surgery; Drug: Thiotepa; Drug: Topotecan Hydrochloride; Drug: Vincristine Sulfate |
| Arm D (chemotherapy, HSCT, EBRT) | Experimental | See Arm D in detailed description. Receives: Procedure: Autologous Hematopoietic Stem Cell Transplantation; Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration and Biopsy; Drug: Carboplatin; Drug: Cisplatin; Procedure: Computed Tomography; Drug: Cyclophosphamide; Drug: Dexrazoxane Hydrochloride; Biological: Dinutuximab; Drug: Doxorubicin Hydrochloride; Drug: Etoposide Phosphate; Radiation: External Beam Radiation Therapy; Radiation: Iobenguane I-123; Drug: Isotretinoin; Procedure: Magnetic Resonance Imaging; Procedure: Multigated Acquisition Scan; Procedure: Positron Emission Tomography; Biological: Sargramostim; Procedure: Therapeutic Conventional Surgery; Drug: Thiotepa; Drug: Topotecan Hydrochloride; Drug: Vincristine Sulfate |
| Arm E (lorlatinib, chemotherapy, HSCT, EBRT) | Experimental | See Arm E in detailed description. Receives: Procedure: Autologous Hematopoietic Stem Cell Transplantation; Procedure: Biospecimen Collection; Procedure: Bone Marrow Aspiration and Biopsy; Drug: Carboplatin; Drug: Cisplatin; Procedure: Computed Tomography; Drug: Cyclophosphamide; Drug: Dexrazoxane Hydrochloride; Biological: Dinutuximab; Drug: Doxorubicin Hydrochloride; Procedure: Echocardiography Test; Drug: Etoposide Phosphate; Radiation: External Beam Radiation Therapy; Radiation: Iobenguane I-123; Drug: Isotretinoin; Drug: Lorlatinib; Procedure: Multigated Acquisition Scan; Procedure: Positron Emission Tomography; Biological: Sargramostim; Procedure: Therapeutic Conventional Surgery; Drug: Thiotepa; Drug: Topotecan Hydrochloride |
What is being measured
The primary outcome is the main question the study is designed to answer.
Event free survival (EFS) (Arm A, B, D, and E)
EFS time is calculated from date of randomization or assignment to first episode of disease relapse or progression, second malignancy, or death, or until last contact if no event has occurred.
Measured: 3 years
Secondary outcomes (4)
Incidence of adverse events
The proportion of patients with at least one Grade 3 or higher 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 18 months for Arms A-D and 28 months for Arm E
EFS (Arm C)
EFS time is calculated from date of randomization to first episode of disease relapse or progression, second malignancy, or death, or until last contact if no event has occurred.
Measured: 3 years
Overall survival (OS)
OS time is calculated from date of randomization or assignment until death, or until last contact if patient is alive.
Measured: 3 years
Response rate
The response rate will be calculated among all evaluable patients at end-Induction. Responders are defined as patients who achieve a >= partial response (PR) per the revised International Neuroblastoma Response Criteria (INRC).
Measured: Up to 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Apr 2017
- Study start14 May 2018
- Primary completion30 Sep 2030 (estimated)
- Study completion30 Sep 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for GanglioneuroblastomaStudies of the same condition, open and under way.
- More trials run by Children's Oncology GroupThe 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
- 750 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Children's Oncology Group
- Sponsor type
- Research network
- Collaborators
- National Cancer Institute (NCI)
- Responsible party
- Sponsor
- Investigators
- Steven G DuBois, Principal investigator, Children's Oncology Group
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03126916
- Sponsor's protocol ID
- ANBL1531
- Other IDs
- NCI-2016-01734, ANBL1531, ANBL1531, U10CA180886
- Status last verified
- Feb 2026
- First submitted
- 17 Apr 2017
- Last updated
- 25 Aug 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Publications
Weiss BD, Yanik G, Naranjo A, Zhang FF, Fitzgerald W, Shulkin BL, Parisi MT, Russell H, Grupp S, Pater L, Mattei P, Mosse Y, Lai HA, Jarzembowski JA, Shimada H, Villablanca JG, Giller R, Bagatell R, Park JR, Matthay KK. A safety and feasibility trial of 131 I-MIBG in newly diagnosed high-risk neuroblastoma: A Children's Oncology Group study. Pediatr Blood Cancer. 2021 Oct;68(10):e29117. doi: 10.1002/pbc.29117. Epub 2021 May 24.
PubMed 34028986