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Clinical Trials (PDQ®)

Combination Chemotherapy, Autologous Stem Cell Transplant, and/or Radiation Therapy in Treating Young Patients With Extraocular Retinoblastoma

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

Basic Trial Information

PhaseTypeStatusAgeSponsorProtocol IDs
Phase IIITreatmentActive10 and underNCI, OtherARET0321
NCI-2009-00421, COG-ARET0321, CDR0000573987, U10CA098543, NCT00554788

Trial Description

Summary

This phase III trial is studying the side effects and how well giving combination chemotherapy together with autologous stem cell transplant and/or radiation therapy works in treating young patients with extraocular retinoblastoma. Giving chemotherapy before an autologous 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/or bone marrow and stored. More chemotherapy 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. Radiation therapy uses high energy x-rays to kill tumor cells. Giving radiation therapy after combination chemotherapy and/or autologous stem cell transplant may kill any remaining tumor cells.

Further Study Information

OBJECTIVES:

I. To estimate the proportion of 3 groups of patients with extraocular retinoblastoma (stage 2 and 3: regional extra-ocular disease;, stage 4a: disseminated metastatic disease not involving the central nervous system [CNS]; or stage 4b: patients with CNS disease) who achieve long-term event-free survival after treatment with aggressive multimodality therapy compared to historical controls.

II. To estimate the response rate to the induction phase of the regimen. III. To evaluate the toxicities associated with this regimen.

OUTLINE:

INDUCTION CHEMOTHERAPY: Patients receive vincristine intravenously (IV) on days 0, 7, and 14, cisplatin IV over 6 hours on day 0, cyclophosphamide IV over 1 hour and etoposide IV over 1 hour on days 1 and 2, and filgrastim (G-CSF) subcutaneously (SC) beginning on day 3 and continuing until blood counts recover.

Treatment repeats every 21 days for up to 4 courses in the absence of disease progression or unacceptable toxicity.

After completion of induction chemotherapy, patients with stage 2 or 3 disease who have at least a partial response proceed to radiotherapy. Patients with stage 4a or 4b disease who have at least a partial response proceed to high-dose consolidation chemotherapy and autologous stem cell infusion.

STEM CELL HARVESTING (stage 4a or 4b disease only): Peripheral blood stem cells (preferred) or bone marrow cells are collected after at least 1 course of induction chemotherapy.

HIGH-DOSE CONSOLIDATION CHEMOTHERAPY (stage 4a or 4b disease only): Patients receive carboplatin IV over 4 hours on days -8 to -6 and thiotepa IV over 3 hours and etoposide IV over 3 hours on days -5 to -3.

AUTOLOGOUS STEM CELL INFUSION (stage 4a or 4b disease only): Patients undergo autologous stem cell infusion on day 0. Patients then receive filgrastim subcutaneously (SC) beginning on day 1 and continuing until blood counts recover.

RADIOTHERAPY: Patients with stage 2 or 3 disease (orbital and/or regional involvement) undergo radiotherapy to sites that were initially involved beginning within 42 days after the start of course 4 of induction chemotherapy. Patients with stage 4a or 4b disease undergo radiotherapy to sites initially involved based on response beginning approximately 42 days after autologous stem cell infusion. Patients with stage 4a disease who achieve a complete response to induction chemotherapy or with less than 5 mm of residual tumor at the time of planned irradiation, or patients with stage 4b disease who achieve a complete response to induction chemotherapy do not undergo radiotherapy.

After completion of study therapy, patients are followed every 3 months for 1 year and then annually thereafter for 9 years.

Eligibility Criteria

Inclusion Criteria:

  • Patients must have histologic or cytologic verification of extra-ocular retinoblastoma; extra-ocular disease includes orbital disease, optic nerve involvement at the surgical margin, regional nodal disease, and/or overt distant metastatic disease (at sites such as bone, bone marrow, liver and/or the central nervous system); patients with trilateral retinoblastoma will also be included in this protocol
  • Patients with a CNS lesion consistent with trilateral or stage 4b disease may be enrolled without tissue confirmation if (1) unequivocal leptomeningeal disease is present on brain or spine magnetic resonance imaging (MRI) scan and/or (2) the primary tumor is at least 2 cm in diameter, predominantly solid, and demonstrates enhancement on the post-gadolinium images; however, even in such cases surgery should be given serious consideration
  • Patients must have a performance status corresponding to Eastern Cooperative Oncology Group (ECOG) scores of 0, 1, or 2; use Karnofsky for patients > 16 years of age and Lansky for patients =< 16 years of age
  • No prior chemotherapy or radiotherapy for the extra-ocular retinoblastoma may have been administered prior to entering this study; prior treatment (chemotherapy and/or radiation therapy) for intra-ocular retinoblastoma is permissible
  • Peripheral absolute neutrophil count (ANC) >= 750/uL
  • If the ANC and/or platelet count are not adequate, but due to bone marrow metastatic disease, these criteria will be waived
  • Platelet count >= 75,000/uL (transfusion independent)
  • If the ANC and/or platelet count are not adequate, but due to bone marrow metastatic disease, these criteria will be waived
  • Creatinine clearance OR radioisotope glomerular filtration rate >= 70 mL/min/1.73 m^2 or a serum creatinine based on age/gender as follows:
  • 0.4 mg/dL (1 month to < 6 months of age)
  • 0.5 mg/dL (6 months to < 1 year of age)
  • 0.6 mg/dL (1 years to < 2 years of age)
  • 0.8 mg/dL (2 years to < 6 years of age)
  • 1.0 mg/dL (6 years to < 10 years of age)
  • 1.2 mg/dL (10 years to < 13 years of age)
  • 1.5 mg/dL (male) or 1.4 mg/dL (female) (13 years to < 16 years of age)
  • 1.7 mg/dL (male) or 1.4 mg/dL (female) (>= 16 years of age)
  • Total bilirubin =< 1.5 times upper limit of normal (ULN)
  • Serum glutamic oxaloacetic transaminase (SGOT) (aspartate aminotransferase [AST]) or serum glutamate pyruvate transaminase (SGPT) (alanine aminotransferase [ALT]) < 2.5 x upper limit of normal (ULN)
  • 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) for human studies must be met

Trial Contact Information

Trial Lead Organizations/Sponsors

Children's Oncology Group

National Cancer Institute

Ira Dunkel, MDPrincipal Investigator

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
 UAB Comprehensive Cancer Center
 Alyssa T Reddy Ph: 205-934-0309
Arizona
  Phoenix
 Phoenix Children's Hospital
 Jessica Boklan Ph: 602-546-0920
Arkansas
  Little Rock
 Arkansas Children's Hospital at the University of Arkansas for Medical Sciences
 David L Becton Ph: 501-364-7373
California
  Downey
 Southern California Permanente Medical Group
 Robert M Cooper Ph: 626-564-3455
  Los Angeles
 Children's Hospital Los Angeles
 Leo Mascarenhas Ph: 323-361-4110
  Palo Alto
 Lucile Packard Children's Hospital at Stanford University Medical Center
 Neyssa M Marina Ph: 650-498-7061
  Email: clinicaltrials@med.stanford.edu
Colorado
  Aurora
 Children's Hospital Colorado Center for Cancer and Blood Disorders
 Brian S Greffe Ph: 720-777-6672
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
  Jacksonville
 Nemours Children's Clinic
 Eric S Sandler Ph: 904-697-3529
  Miami
 University of Miami Sylvester Comprehensive Cancer Center - Miami
 Julio C Barredo Ph: 866-574-5124
  Email: Sylvester@emergingmed.com
  Orlando
 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
Illinois
  Chicago
 Ann and Robert H. Lurie Children's Hospital of Chicago
 Joanna L Weinstein Ph: 773-880-4562
 University of Illinois Cancer Center
 Mary L Schmidt Ph: 312-355-3046
Indiana
  Indianapolis
 Riley's Children Cancer Center at Riley Hospital for Children
 Robert J Fallon Ph: 317-274-2552
Iowa
  Iowa City
 Holden Comprehensive Cancer Center at University of Iowa
 Ayman A El-Sheikh Ph: 800-237-1225
Kentucky
  Lexington
 University of Kentucky Chandler Medical Center
 Lars M Wagner Ph: 859-257-3379
Maryland
  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
 Massachusetts General Hospital
 Howard J Weinstein Ph: 877-726-5130
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
Nevada
  Las Vegas
 Cancer Institute of Nevada at Summerlin Hospital Medical Center
 Jonathan Bernstein Ph: 702-384-0013
 CCOP - Nevada Cancer Research Foundation
 Jonathan Bernstein Ph: 702-384-0013
 Children's Specialty Center of Nevada
 Jonathan Bernstein Ph: 702-384-0013
New Jersey
  Morristown
 Carol G. Simon Cancer Center at Morristown Memorial Hospital
 Steven L Halpern Ph: 973-971-5900
New York
  New York
 Memorial Sloan-Kettering Cancer Center
 Ira J Dunkel Ph: 212-639-7202
  Valhalla
 New York Medical College
 Mehmet F Ozkaynak Ph: 914-594-3794
North Carolina
  Durham
 Duke Cancer Institute
 Susan G Kreissman Ph: 888-275-3853
Ohio
  Akron
 Akron Children's Hospital
 Steven J Kuerbitz Ph: 330-543-3193
  Cincinnati
 Cincinnati Children's Hospital Medical Center
 John P Perentesis Ph: 513-636-2799
  Dayton
 Dayton Children's - Dayton
 Emmett H Broxson Ph: 800-228-4055
Oklahoma
  Oklahoma City
 Oklahoma University Cancer Institute
 Rene Y McNall-Knapp Ph: 405-271-4272
  Email: julie-traylor@ouhsc.edu
Pennsylvania
  Hershey
 Penn State Children's Hospital
 Lisa M McGregor Ph: 717-531-6012
  Philadelphia
 Children's Hospital of Philadelphia
 Ann-Marie Leahey Ph: 215-590-2810
 Children's Oncology Group
 Ira J Dunkel Ph: 212-639-2153
  Email: dunkeli@mskcc.org
  Pittsburgh
 Children's Hospital of Pittsburgh of UPMC
 Jean M Tersak Ph: 412-692-5573
Tennessee
  Memphis
 St. Jude Children's Research Hospital
 Wayne L Furman Ph: 901-595-4644
Texas
  Dallas
 Medical City Dallas Hospital
 Carl Lenarsky Ph: 972-566-5588
 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
  Houston
 Dan L. Duncan Cancer Center at Baylor College of Medicine
 Murali M Chintagumpala Ph: 713-798-1354
  Email: burton@bcm.edu
  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: 210-450-3800
  Email: CTO@uthscsa.edu
Virginia
  Richmond
 Virginia Commonwealth University Massey Cancer Center
 Asadullah Khan Ph: 804-628-1939
Wisconsin
  Milwaukee
 Midwest Children's Cancer Center at Children's Hospital of Wisconsin
 Michael E Kelly Ph: 414-805-4380
Australia
New South Wales
  Westmead
 Children's Hospital at Westmead
 Geoffrey B McCowage Ph: 61-2-9845 1400
Western Australia
  Perth
 Princess Margaret Hospital for Children
 Catherine H Cole Ph: (08) 9340 8330
  Email: admin@childcancerresearch.com.au
Canada
Nova Scotia
  Halifax
 IWK Health Centre
 Conrad V Fernandez Ph: 902-470-8394
Quebec
  Montreal
 Hopital Sainte Justine
 Yvan Samson Ph: 514-345-4931

Link to the current ClinicalTrials.gov record.
NLM Identifer NCT00554788
ClinicalTrials.gov processed this data on September 16, 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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