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Busulfan, Melphalan, and Stem Cell Transplant After Chemotherapy in Treating Patients With Newly Diagnosed High-Risk Neuroblastoma

Basic Trial Information
Trial Description
     Summary
     Further Trial Information
     Eligibility Criteria
Trial Contact Information

Basic Trial Information

PhaseTypeStatusAgeSponsorProtocol IDs
No phase specifiedBiomarker/Laboratory analysis, TreatmentActive30 and underNCI, OtherANBL12P1
COG-ANBL12P1, NCI-2012-02211, U10CA098543, NCT01798004

Trial Description

Summary

This pilot clinical trial studies busulfan, melphalan, and stem cell transplant after chemotherapy in treating patients with newly diagnosed high-risk neuroblastoma. Giving chemotherapy to the entire body before a stem cell transplant stops the growth of tumor cells by stopping them from dividing or killing them. After treatment, stem cells are collected from the patient's blood and stored. More chemotherapy or radiation therapy is given to prepare the bone marrow for the stem cell transplant. The stem cells are then returned to the patient to replace the blood-forming cells that were destroyed by the chemotherapy.

Further Study Information

This groupwide pilot study examines the toxicity profile of the Busulfan-Melphalan (BuMel) myeloablative preparative regimen in children and young adults with newly diagnosed high-risk neuroblastoma. The primary objective of the proposed study will be to examine the toxicity profile of this regimen in the context of COG therapy, with specific focus on the incidence and severity of pulmonary and hepatic toxicity. The Induction regimen will be 5 cycles of Induction. Consolidation therapy will consist of 4 doses of busulfan IV given once daily followed by a single dose of melphalan with a rest day prior to and following the melphalan dose. After recovery from Consolidation radiation therapy, patients will be encouraged to participate in clinical trials of ch14.18 immunotherapy (ie, ANBL0032 or other).

Additional examinations will include pharmacokinetic measurements of busulfan and melphalan that will be collected and correlated with toxicity and survival. We will examine the ability to perform Curie scoring in real time, within 21 days of scan acquisition. This will be the first prospective use of Curie scoring in a cooperative group setting. This study will examine our ability to perform ALK gene testing prospectively, within 4 to 6 weeks of sample acquisition, by a centralized lab. Aberrations of the ALK gene in neuroblastoma tumors have been reported by multiple investigators, with potential therapeutic implications. Potential targeted inhibitors of ALK aberrations are now available, and may impact future clinical trial designs. In addition, molecular profiling of MYCN non-amplified tumors with a 14-gene signature panel will be performed. This study will test our ability to obtain tumor samples prospectively and identify molecular profiles within 6-8 weeks of sample acquisition which may also impact future clinical trial design.

Eligibility Criteria

Inclusion Criteria:

  • Patients must have a diagnosis of neuroblastoma (International Classification of Diseases for Oncology [ICD-O] morphology 9500/3) or ganglioneuroblastoma (nodular or intermixed) verified by histology or demonstration of clumps of tumor cells in bone marrow with elevated urinary catecholamine metabolites; patients with the following disease stages at diagnosis are eligible, if they meet the other specified criteria
  • Patients with newly diagnosed neuroblastoma with International Neuroblastoma Staging System (INSS) stage 4 are eligible with the following:
  • MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features
  • Age > 18 months (> 547 days) regardless of biologic features
  • Age 12-18 months (365-547 days) with any of the following 3 unfavorable biologic features (MYCN amplification, unfavorable pathology and/or DNA index = 1) or any biologic feature that is indeterminate/unsatisfactory/unknown
  • Patients with newly diagnosed neuroblastoma with INSS stage 3 are eligible with the following:
  • MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features
  • Age > 18 months (> 547 days) with unfavorable pathology, regardless of MYCN status
  • Patients with newly diagnosed neuroblastoma with INSS stage 2A/2B with MYCN amplification (> 4-fold increase in MYCN signals as compared to reference signals), regardless of age or additional biologic features
  • Patients with newly diagnosed neuroblastoma with INSS Stage 4S with MYCN amplification (> 4-fold increase in MYCN expression signals as compared to reference signals), regardless of additional biologic features
  • Patients >= 365 days initially diagnosed with neuroblastoma INSS stage 1, 2, 4S who progressed to a stage 4 without interval chemotherapy; these patients must have been enrolled on ANBL00B1; study enrollment on ANBL12P1 must occur within 4 weeks of progression to stage 4 for INSS stage 1, 2, 4S
  • Patients must not have had prior systemic therapy except for localized emergency radiation to sites of life-threatening or function-threatening disease and/or no more than 1 cycle of chemotherapy per a low or intermediate risk neuroblastoma regimen (as per P9641, A3961, ANBL0531, or similar) prior to determination of MYCN amplification status and histology
  • Creatinine clearance or radioisotope glomerular filtration rate (GFR) >= 70 mL/min/1.73 m^2 or a serum creatinine based on age/gender as follows:
  • Age 1 month to < 6 months: 0.4 mg/dL
  • Age 6 months to < 1 year: 0.5 mg/dL
  • Age 1 to < 2 years: 0.6 mg/dL
  • Age 2 to < 6 years: 0.8 mg/dL
  • Age 6 to < 10 years: 1 mg/dL
  • Age 10 to < 13 years: 1.2 mg/dL
  • Age 13 to < 16 years: 1.5 mg/dL (males), 1.4 mg/dL (females)
  • Age >= 16 years: 1.7 mg/dL (males), 1.4 mg/dL (females)
  • Total bilirubin =< 1.5 x upper limit of normal (ULN) for age, and
  • Serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST]) or serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) < 10 x ULN for age
  • Shortening fraction of >= 27% by echocardiogram, or ejection fraction of >= 50% by radionuclide evaluation
  • No known contraindication to peripheral blood stem cell (PBSC) collection; examples of contraindications might be a weight or size less than that determined to be feasible at the collecting institution, or a physical condition that would limit the ability of the child to undergo apheresis catheter placement (if necessary) and/or the apheresis procedure.

Exclusion Criteria:

  • Patients that are 12-18 months of age with INSS stage 4 and all 3 favorable biologic features (ie, nonamplified MYCN, favorable pathology, and DNA index > 1) are not eligible
  • Female patients who are pregnant are ineligible due to risks of fetal and teratogenic adverse events
  • Lactating females are not eligible unless they have agreed not to breastfeed their infants
  • Female patients of childbearing potential are not eligible unless a negative pregnancy test result has been obtained
  • Sexually active patients of reproductive potential are not eligible unless they have agreed to use an effective contraceptive method for the duration of their study participation.

Trial Contact Information

Trial Lead Organizations/Sponsors

Children's Oncology Group

National Cancer Institute

Meaghan P. GrangerStudy Chair

Meera Raman, MS, PMP, CCRPPh: (626) 241-1532
  Email: mraman@childrensoncologygroup.org

Trial Sites

U.S.A.
Alabama
  Birmingham
 Children's Hospital of Alabama at University of Alabama at Birmingham
 Alyssa T Reddy Ph: 205-934-0309
California
  Duarte
 City of Hope Comprehensive Cancer Center
 Anna B Pawlowska Ph: 800-826-4673
  Email: becomingapatient@coh.org
  Loma Linda
 Loma Linda University Cancer Institute at Loma Linda University Medical Center
 Antranik A Bedros Ph: 909-558-3375
  Los Angeles
 Children's Hospital Los Angeles
 Leo Mascarenhas Ph: 323-361-4110
  Oakland
 Children's Hospital and Research Center Oakland
 Carla B Golden Ph: 510-450-7600
 Kaiser Permanente-Oakland
 Steven K Bergstrom Ph: 626-564-3455
  Orange
 Children's Hospital of Orange County
 Violet Shen Ph: 714-997-3000
  Palo Alto
 Lucile Packard Children's Hospital at Stanford University Medical Center
 Neyssa M Marina Ph: 650-498-7061
  Email: clinicaltrials@med.stanford.edu
  Sacramento
 University of California Davis Cancer Center
 Jay Michael S Balagtas Ph: 916-734-3089
  San Diego
 Rady Children's Hospital - San Diego
 William D Roberts Ph: 858-966-5934
  San Francisco
 UCSF Helen Diller Family Comprehensive Cancer Center
 Steven G DuBois Ph: 877-827-3222
Connecticut
  Hartford
 Connecticut Children's Medical Center
 Michael S Isakoff Ph: 860-545-9981
Delaware
  Wilmington
 Alfred I. duPont Hospital for Children
 Christopher N Frantz Ph: 302-651-5755
District of Columbia
  Washington
 Children's National Medical Center
 Jeffrey S Dome Ph: 202-884-2549
Florida
  Fort Myers
 Children's Hospital of Southwest Florida
 Emad K Salman Ph: 239-343-5333
  Hollywood
 Joe DiMaggio Children's Hospital
 Iftikhar Hanif Ph: 954-265-2234
  Jacksonville
 Nemours Children's Clinic
 Scott M Bradfield Ph: 904-697-3529
  Orlando
 Florida Hospital Cancer Institute at Florida Hospital Orlando
 Fouad M Hajjar Ph: 407-303-5623
 Nemours Children's Hospital
 Ramamoorthy Nagasubramanian Ph: 407-650-7150
  Pensacola
 Nemours Children's Clinic - Pensacola
 Jeffrey H Schwartz Ph: 904-697-3529
  Saint Petersburg
 All Children's Hospital
 Gregory A Hale Ph: 727-767-2423
  Email: HamblinF@allkids.org
  Tampa
 St. Joseph's Children's Hospital of Tampa
 Hardeo K Panchoosingh Ph: 800-882-4123
Georgia
  Atlanta
 AFLAC Cancer Center and Blood Disorders Service of Children's Healthcare of Atlanta - Egleston Campus
 Glen Lew Ph: 404-785-1112
  Savannah
 Curtis and Elizabeth Anderson Cancer Institute at Memorial Health University Medical Center
 J. M Johnston Ph: 912-350-8568
Illinois
  Chicago
 Ann and Robert H. Lurie Children's Hospital of Chicago
 Yasmin C Gosiengfiao Ph: 773-880-4562
 University of Chicago Cancer Research Center
 Susan L Cohn Ph: 773-834-7424
 University of Illinois Cancer Center
 Mary L Schmidt Ph: 312-355-3046
  Maywood
 Cardinal Bernardin Cancer Center at Loyola University Medical Center
 Ricarchito B Manera Ph: 708-226-4357
  Peoria
 Saint Jude Midwest Affiliate
 Pedro A De Alarcon Ph: 309-655-3258
  Springfield
 Simmons Cooper Cancer Institute
 Gregory P Brandt Ph: 217-545-7929
Indiana
  Indianapolis
 Riley's Children Cancer Center at Riley Hospital for Children
 Robert J Fallon Ph: 317-274-2552
 St. Vincent Indianapolis Hospital
 Bassem I Razzouk Ph: 317-338-2194
Iowa
  Des Moines
 Blank Children's Hospital
 Wendy L Woods-Swafford Ph: 515-241-6729
Kentucky
  Lexington
 University of Kentucky Chandler Medical Center
 Martha F Greenwood Ph: 859-257-3379
  Louisville
 Kosair Children's Hospital
 Alexandra C Cheerva Ph: 866-530-5516
Louisiana
  New Orleans
 Children's Hospital of New Orleans
 Lolie C Yu Ph: 504-894-5377
Maine
  Scarborough
 Maine Children's Cancer Program at Barbara Bush Children's Hospital
 Aaron R Weiss Ph: 732-235-8675
Maryland
  Baltimore
 Alvin and Lois Lapidus Cancer Institute at Sinai Hospital
 Joseph M Wiley Ph: 410-601-6120
  Email: pridgely@lifebridgehealth.org
 Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
 Allen R. Chen Ph: 410-955-8804
  Email: jhcccro@jhmi.edu
  Bethesda
 National Naval Medical Center
 Anne B Warwick Ph: 301-319-2100
Massachusetts
  Boston
 Dana-Farber/Harvard Cancer Center at Dana-Farber Cancer Institute
 Carlos Rodriguez-Galindo Ph: 866-790-4500
 Floating Hospital for Children at Tufts - New England Medical Center
 Michael J Kelly Ph: 617-636-5000
  Email: ContactUsCancerCenter@TuftsMedicalCenter.org
 Massachusetts General Hospital
 Howard J Weinstein Ph: 877-726-5130
Michigan
  Ann Arbor
 C.S. Mott Children's Hospital at University of Michigan Medical Center
 Rajen Mody Ph: 800-865-1125
  Detroit
 Wayne State University
 Roland L Chu Ph: 313-576-9363
Minnesota
  Minneapolis
 Children's Hospitals and Clinics of Minnesota - Minneapolis
 Bruce C Bostrom Ph: 612-813-5193
 Masonic Cancer Center at University of Minnesota
 Emily G Greengard Ph: 612-624-2620
  Rochester
 Mayo Clinic Cancer Center
 Carola A. S. Arndt Ph: 507-538-7623
Mississippi
  Jackson
 University of Mississippi Cancer Clinic
 Gail C Megason Ph: 601-815-6700
Missouri
  Kansas City
 Children's Mercy Hospital
 Maxine L Hetherington Ph: 816-234-3265
  Saint Louis
 David C. Pratt Cancer Center at St. John's Mercy
 Bethany G. Sleckman Ph: 913-948-5588
 Siteman Cancer Center at Barnes-Jewish Hospital - Saint Louis
 Shalini Shenoy Ph: 800-600-3606
  Email: info@siteman.wustl.edu
Nebraska
  Omaha
 Children's Hospital
 Minnie Abromowitch Ph: 402-955-3949
 UNMC Eppley Cancer Center at the University of Nebraska Medical Center
 Minnie Abromowitch Ph: 402-955-3949
New Hampshire
  Lebanon
 Norris Cotton Cancer Center at Dartmouth-Hitchcock Medical Center
 Sara Chaffee Ph: 800-639-6918
  Email: cancer.research.nurse@dartmouth.edu
New Jersey
  Hackensack
 Hackensack University Medical Center Cancer Center
 Burton E Appel Ph: 201-996-2879
  Morristown
 Carol G. Simon Cancer Center at Morristown Memorial Hospital
 Steven L Halpern Ph: 973-971-5900
  New Brunswick
 Cancer Institute of New Jersey at UMDNJ - Robert Wood Johnson Medical School
 Richard A Drachtman Ph: 732-235-8675
  Newark
 Newark Beth Israel Medical Center
 Peri Kamalakar Ph: 973-926-7230
  Paterson
 St. Joseph's Hospital and Medical Center
 Mary A Bonilla Ph: 973-754-2909
New Mexico
  Albuquerque
 University of New Mexico Cancer Center
 Koh B Boayue Ph: 505-272-6972
New York
  Bronx
 Montefiore Medical Center
 Rosanna J Ricafort Ph: 718-904-2730
  Email: aecc@aecom.yu.edu
  Mineola
 Winthrop University Hospital
 Mark E Weinblatt Ph: 866-946-8476
  New Hyde Park
 Schneider Children's Hospital
 Jonathan D Fish Ph: 718-470-3470
  New York
 Herbert Irving Comprehensive Cancer Center at Columbia University Medical Center
 Alice Lee Ph: 212-305-8615
  Rochester
 James P. Wilmot Cancer Center at University of Rochester Medical Center
 Jeffrey R Andolina Ph: 585-275-5830
  Syracuse
 SUNY Upstate Medical University Hospital
 Karol H Kerr Ph: 315-464-5476
North Carolina
  Asheville
 Mission Hospitals - Memorial Campus
 Krystal S Bottom Ph: 828-213-4150
  Chapel Hill
 Lineberger Comprehensive Cancer Center at University of North Carolina - Chapel Hill
 Stuart H Gold Ph: 877-668-0683
  Email: cancerclinicaltrials@med.unc.edu
  Durham
 Duke Cancer Institute
 Susan G Kreissman Ph: 888-275-3853
Ohio
  Cincinnati
 Cincinnati Children's Hospital Medical Center
 John P Perentesis Ph: 513-636-2799
  Cleveland
 Cleveland Clinic Taussig Cancer Center
 Margaret C Thompson Ph: 866-223-8100
 Seidman Cancer Center at University Hospitals/Case Medical Center
 Yousif (Joe) H Matloub Ph: 216-844-5437
  Columbus
 Nationwide Children's Hospital
 Mark A Ranalli Ph: 614-722-2708
  Dayton
 Dayton Children's - Dayton
 Emmett H Broxson Ph: 800-228-4055
  Toledo
 Mercy Children's Hospital
 Rama Jasty Ph: 419-251-8210
 Toledo Hospital
 Jamie L Dargart Ph: 419-824-1842
Oklahoma
  Oklahoma City
 Oklahoma University Cancer Institute
 Rene Y McNall-Knapp Ph: 405-271-4272
  Email: julie-traylor@ouhsc.edu
Pennsylvania
  Philadelphia
 Children's Hospital of Philadelphia
 Rochelle Bagatell Ph: 215-590-2810
  Pittsburgh
 Children's Hospital of Pittsburgh of UPMC
 Jean M Tersak Ph: 412-692-5573
South Carolina
  Charleston
 Hollings Cancer Center at Medical University of South Carolina
 Jacqueline M Kraveka Ph: 843-792-9321
  Columbia
 Palmetto Health South Carolina Cancer Center
 Ronnie W. Neuberg Ph: 803-434-3680
  Greenville
 BI-LO Charities Children's Cancer Center
 Nichole L Bryant Ph: 864-241-6251
Tennessee
  Chattanooga
 T.C. Thompson Children's Hospital
 Donald H. Chamberlain Ph: 423-698-2050
  Knoxville
 East Tennessee Children's Hospital
 Ray C Pais Ph: 865-541-8266
  Nashville
 Vanderbilt-Ingram Cancer Center
 Haydar A Frangoul Ph: 800-811-8480
Texas
  Dallas
 Simmons Comprehensive Cancer Center at University of Texas Southwestern Medical Center - Dallas
 Naomi J Winick Ph: 214-648-7097
  Fort Worth
 Cook Children's Medical Center - Fort Worth
 Mary Meaghan P Granger Ph: 682-885-2103
  San Antonio
 Methodist Children's Hospital of South Texas
 Jaime Estrada Ph: 210-575-7000
 University of Texas Health Science Center at San Antonio
 Anne-Marie R Langevin Ph: 210-567-0653
  Email: che@uthscsa.edu
Virginia
  Richmond
 Virginia Commonwealth University Massey Cancer Center
 Christina M Wiedl Ph: 804-628-1939
Washington
  Seattle
 Children's Hospital and Regional Medical Center - Seattle
 Douglas S Hawkins Ph: 866-987-2000
  Spokane
 Providence Cancer Center at Sacred Heart Medical Center
 Judy L Felgenhauer Ph: 800-228-6618
  Email: HopeBeginsHere@providence.org
Wisconsin
  Madison
 University of Wisconsin Paul P. Carbone Comprehensive Cancer Center
 Kenneth B DeSantes Ph: 608-262-5223
  Marshfield
 Marshfield Clinic - Marshfield Center
 Michael J McManus Ph: 715-389-4457
Australia
Western Australia
  Perth
 Princess Margaret Hospital for Children
 Catherine H Cole Ph: (08) 9340 8330
  Email: admin@childcancerresearch.com.au
Canada
British Columbia
  Vancouver
 Children's and Women's Hospital of British Columbia
 Caron Strahlendorf Ph: 604-875-2345ext6477
Manitoba
  Winnipeg
 CancerCare Manitoba
 Rochelle A Yanofsky Ph: 866-561-1026
  Email: CIO_Web@cancercare.mb.ca
Nova Scotia
  Halifax
 IWK Health Centre
 Margaret C Yhap Ph: 902-470-8394
Ontario
  Toronto
 Hospital for Sick Children
 Meredith S Irwin Ph: 416-813-7654ext2027
  Email: jason.mcguire@sickkids.ca
Quebec
  Montreal
 Hopital Sainte Justine
 Yvan Samson Ph: 514-345-4931
 Montreal Children's Hospital at McGill University Health Center
 Sharon B Abish Ph: 514-412-4445
  Email: info@thechildren.com
  Ste-Foy
 Centre de Recherche du Centre Hospitalier de l'Universite Laval
 Bruno Michon Ph: 418-525-4444
New Zealand
Auckland
  Grafton
 Starship Children's Health
 Lochie R Teague Ph:  0800 728 436

Link to the current ClinicalTrials.gov record.
NLM Identifer NCT01798004
ClinicalTrials.gov processed this data on February 05, 2014

Note: Information about this trial is from the ClinicalTrials.gov database. The versions designated for health professionals and patients contain the same text. Minor changes may be made to the ClinicalTrials.gov record to standardize the names of study sponsors, sites, and contacts. Cancer.gov only lists sites that are recruiting patients for active trials, whereas ClinicalTrials.gov lists all sites for all trials. Questions and comments regarding the presented information should be directed to ClinicalTrials.gov.

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