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Last Modified: 11/20/2009     First Published: 4/1/2002  
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Phase III Randomized Study of Cyclophosphamide and Prednisone With or Without Immune Globulin in Pediatric Patients With Neuroblastoma-Associated Opsoclonus-Myoclonus-Ataxia Syndrome

Alternate Title
Basic Trial Information
Objectives
Entry Criteria
Expected Enrollment
Outcomes
Outline
Trial Contact Information
Related Information
Registry Information

Alternate Title

Cyclophosphamide and Prednisone With or Without Immunoglobulin in Treating Abnormal Muscle Movement in Children With Neuroblastoma

Basic Trial Information

PhaseTypeStatusAgeSponsorProtocol IDs
Phase IIITreatmentActive8 and underNCICOG-ANBL00P3
ANBL00P3, NCT00033293

Objectives

  1. Determine whether cyclophosphamide and prednisone with or without immune globulin is a reasonable baseline standard therapy for pediatric patients with neuroblastoma-associated opsoclonus-myoclonus-ataxia (OMA) syndrome.
  2. Determine whether immunosuppressive therapy with cyclophosphamide and prednisone is an effective backbone therapy for OMA upon which to build additional treatment for these patients.
  3. Determine whether these regimens improve OMA syndrome in these patients.
  4. Determine whether these regimens improve motor coordination in these patients.
  5. Determine whether these regimens improve functional outcome in these patients.
  6. Compare the event-free and overall survival of patients treated with these regimens.

Entry Criteria

Disease Characteristics:

  • Newly diagnosed neuroblastoma (NBL) or ganglioneuroblastoma with tumor-associated opsoclonus-myoclonus-ataxia syndrome (OMA)
    • Patients with NBL diagnosed within 6 months of OMA diagnosis AND patients with OMA diagnosed within 6 months of NBL diagnosis are eligible
    • Must enroll on study within 4 weeks of diagnosis
    • Presence of opsoclonus, myoclonus, and/or ataxia associated with neuroblastoma considered eligible

  • Currently enrolled on COG neuroblastoma protocols: COG-ANBL00B1 or its successor

Prior/Concurrent Therapy:

Biologic therapy:

  • No prior IV gamma globulin therapy

Chemotherapy:

  • No prior chemotherapy
  • Concurrent chemotherapy allowed

Endocrine therapy:

  • No prior prednisone or corticotropin
    • Patients who have received ≤ 14 days of steroids are eligible

Radiotherapy:

  • Not specified

Surgery:

  • Concurrent surgery allowed

Patient Characteristics:

Age:

  • 8 and under at the time of diagnosis

Performance status:

  • Not specified

Life expectancy:

  • Not specified

Hematopoietic:

  • Not specified

Hepatic:

  • Not specified

Renal:

  • Creatinine clearance or radioisotope GFR ≥ 70 mL/min OR serum creatinine based on age/gender as follows:
    • ≤ 0.4 mg/dL (for patients 1 to 5 months of age)
    • ≤ 0.5 mg/dL (for patients 6 to 11 months of age)
    • ≤ 0.6 mg/dL (for patients 1 year of age)
    • ≤ 0.8 mg/dL (for patients 2 to 5 years of age)
    • ≤ 1.0 mg/dL (for patients 6 to 9 years of age)
    • ≤ 1.2 mg/dL (for patients 10 to 12 years of age)
    • ≤ 1.4 mg/dL (for female patients ≥ 13 years of age)
    • ≤ 1.5 mg/dL (for male patients 13 to 15 years of age)
    • ≤ 1.6 mg/dL (for male patients ≥ 16 years of age)

Expected Enrollment

52

A total of 18-52 patients (9-26 per treatment arm) will be accrued for this study within 2-5.8 years.

Outcomes

Primary Outcome(s)

Efficacy as measured by assessment of response of opsoclonus, gait, neural, stance, arm and hand function at baseline, 6 months, and 1 year
Response as measured by assessment of opsoclonus, gait, neural, stance, arm and hand function at baseline, 6 months, and 1 year

Secondary Outcome(s)

Motor coordination as assessed by neurological examination and Vineland Adaptive Behavior Scale at baseline, 1 and 5 years
Functional outcome as assessed by age-appropriate neuropsychological testing at baseline, 1 and 5 years
Biology of neuroblastoma associated opsoclonus-myoclonus-ataxia syndrome, specifically by MRI findings, anti-neuronal antibodies, SCF findings and tumor biology at baseline, 6 months, and 1 year
Long-term prognosis for neurologic recovery by neurological examination at baseline, 1 and 5 years
Tumor outcome in terms of event-free survival and overall survival at 1, 5, and 10 years

Outline

This is a randomized, multicenter study. Patients are stratified according to risk group per protocol COG-ANBL00B1 (low risk vs intermediate risk on COG-A3961 vs high risk on COG-A3973).

  • Chemotherapy: Patients with intermediate-risk or high-risk neuroblastoma receive chemotherapy (including cyclophosphamide) according to the standard of care for the stage of primary neuroblastoma, beginning on day 0.

    Patients with low-risk neuroblastoma (and not receiving other chemotherapy) receive cyclophosphamide IV over 1 hour on day 0. Treatment repeats every 4 weeks for 6 courses in the absence of disease progression or unacceptable toxicity.

    All patients receive oral prednisone twice daily for 3 months and then every other day for 7-15 months.

  • Immune globulin therapy: Patients are randomized to 1 of 2 treatment arms.
    • Arm I: Patients receive immune globulin IV on days -2 and -1, at weeks 4, 8, 12, 16, 20, and 24, and then at months 8, 10, and 12 after therapy. Treatment continues in the absence of disease progression or unacceptable toxicity. Patients with no response after 6 months go off treatment.

    • Arm II: Patients do not receive immune globulin. Patients with unresponsive opsoclonus-myoclonus-ataxia syndrome after 2 months or progression after 6 months may cross over to arm I.

Patients are followed during therapy every month for 6 months, at 1 year, and then annually thereafter.

Trial Contact Information

Trial Lead Organizations

Children's Oncology Group

Pedro de Alarcon, MD, Protocol chair
Ph: 309-655-4242

Trial Sites

U.S.A.
Arizona
  Tucson
 Arizona Cancer Center at University of Arizona Health Sciences Center
 Clinical Trials Office - Arizona Cancer Center at University of Arizona Health Sciences Center
Ph: 520-626-9008
Arkansas
  Little Rock
 Arkansas Cancer Research Center at University of Arkansas for Medical Sciences
 Clinical Trial Office - Arkansas Cancer Research Center at University of Arkansas for Medical Sciences
Ph: 501-686-8274
California
  Loma Linda
 Loma Linda University Cancer Institute at Loma Linda University Medical Center
 Clinical Trials Office - Loma Linda University Cancer Institute
Ph: 909-558-3375
  Los Angeles
 Samuel Oschin Comprehensive Cancer Institute at Cedars-Sinai Medical Center
 Carole Hurvitz
Ph: 310-423-4423
  Madera
 Children's Hospital Central California
 Vonda Crouse
Ph: 559-353-5480
  Sacramento
 Kaiser Permanente Medical Center - Oakland
 Vincent Kiley
Ph: 916-474-2326
  San Diego
 Rady Children's Hospital - San Diego
 Clinical Trials Office - Rady Children's Hospital - San Diego
Ph: 858-966-5934
  San Francisco
 UCSF Helen Diller Family Comprehensive Cancer Center
 Clinical Trials Office - UCSF Helen Diller Family Comprehensive Cancer Center
Ph: 877-827-3222
Colorado
  Aurora
 Children's Hospital Center for Cancer and Blood Disorders
 Kelly Maloney
Ph: 720-777-6673
  Denver
 Presbyterian - St. Luke's Medical Center
 Clinical Trials Office - Presbyterian - St. Luke's Medical Center
Ph: 303-839-6000
Delaware
  Wilmington
 Alfred I. duPont Hospital for Children
 Clinical Trials Office - Alfred I. duPont Hospital for Children
Ph: 302-651-5755
District of Columbia
  Washington
 Children's National Medical Center
 Clinical Trials Office - Children's National Medical Center
Ph: 202-884-2549
 Lombardi Comprehensive Cancer Center at Georgetown University Medical Center
 Clinical Trials Office - Lombardi Comprehensive Cancer Center
Ph: 202-444-0381
Florida
  Fort Myers
 Lee Cancer Care of Lee Memorial Health System
 Clinical Trials Office - Lee Cancer Care of Lee Memorial Health System
Ph: 877-680-0008
  Jacksonville
 Nemours Children's Clinic
 Eric Sandler
Ph: 904-697-3793
  Miami
 University of Miami Sylvester Comprehensive Cancer Center - Miami
 University of Miami Sylvester Comprehensive Cancer Center Clinical Trial Matching Service
Ph: 866-574-5124
 Email: Sylvester@emergingmed.com
  Orlando
 Nemours Children's Clinic - Orlando
 Paul Gordon
Ph: 407-650-7330
  Pensacola
 Sacred Heart Cancer Center at Sacred Heart Hospital
 Clinical Trials Office - Sacred Heart Cancer Center
Ph: 850-416-4611
  St. Petersburg
 All Children's Hospital
 Gregory Hale
Ph: 727-767-4176
  Tampa
 St. Joseph's Cancer Institute at St. Joseph's Hospital
 Clinical Trials Office - St. Joseph's Cancer Institute
Ph: 800-882-4123
  West Palm Beach
 Kaplan Cancer Center at St. Mary's Medical Center
 Narayana Gowda
Ph: 561-844-6363
Georgia
  Atlanta
 Winship Cancer Institute of Emory University
 Howard Katzenstein
Ph: 404-785-0853
Idaho
  Boise
 Mountain States Tumor Institute at St. Luke's Regional Medical Center
 Eugenia Chang
Ph: 208-381-2731
Illinois
  Chicago
 Children's Memorial Hospital - Chicago
 Stewart Goldman
Ph: 773-880-3270
 University of Chicago Cancer Research Center
 Clinical Trials Office - University of Chicago Cancer Research Center
Ph: 773-834-7424
  Peoria
 Saint Jude Midwest Affiliate
 Stephen Smith
Ph: 309-624-4945
Indiana
  Indianapolis
 Indiana University Melvin and Bren Simon Cancer Center
 Clinical Trials Office - Indiana University Cancer Center
Ph: 317-274-2552
Kentucky
  Lexington
 Lucille P. Markey Cancer Center at University of Kentucky
 Clinical Trials Office - Markey Cancer Center at University of Kentucky Chandler Medical Center
Ph: 859-257-3379
  Louisville
 Kosair Children's Hospital
 Clinical Trials Office - Kosair Children's Hospital
Ph: 502-629-5500
 Email: CancerResource@nortonhealthcare.org
Louisiana
  Alexandria
 Tulane Cancer Center Office of Clinical Research
 Clinical Trials Office - Tulane Cancer Center
Ph: 504-988-6121
Maryland
  Baltimore
 Alvin and Lois Lapidus Cancer Institute at Sinai Hospital
 Joseph Wiley
Ph: 410-601-5864
Massachusetts
  Boston
 Dana-Farber/Harvard Cancer Center at Dana Farber Cancer Institute
 Suzanne Shusterman
Ph: 617-632-4901
 Massachusetts General Hospital
 Clinical Trials Office - Massachusetts General Hospital
Ph: 877-726-5130
Michigan
  Detroit
 Barbara Ann Karmanos Cancer Institute
 Clinical Trials Office - Barbara Ann Karmanos Cancer Institute
Ph: 313-576-9363
  Flint
 Hurley Medical Center
 Clinical Trials Office - Hurley Medical Center
Ph: 810-762-8057
  Grand Rapids
 Butterworth Hospital at Spectrum Health
 David Dickens
Ph: 616-391-2086
  Grosse Pointe Woods
 Van Elslander Cancer Center at St. John Hospital and Medical Center
 Clincial Trials Office - Van Elslander Cancer Center at St. John Hospital and Medical Center
Ph: 313-343-3166
  Lansing
 Breslin Cancer Center at Ingham Regional Medical Center
 Clinical Trials Office - Breslin Cancer Center at Ingham Regional Medical Center
Ph: 517-334-2765
Mississippi
  Jackson
 University of Mississippi Cancer Clinic
 Gail Megason
Ph: 601-984-5220
Missouri
  Kansas City
 Children's Mercy Hospital
 Maxine Hetherington
Ph: 816-234-3265
Nevada
  Las Vegas
 CCOP - Nevada Cancer Research Foundation
 Jonathan Bernstein
Ph: 702-732-0971
 Jonathan Bernstein
Ph: 702-732-1493
New Jersey
  Hackensack
 Hackensack University Medical Center Cancer Center
 Clinical Trials Office - Hackensack University Medical Center Cancer Center
Ph: 201-996-2879
  Morristown
 Overlook Hospital
 Hazem Mahmoud
Ph: 973-971-6720
  Paterson
 St. Joseph's Hospital and Medical Center
 Mary Bonilla
Ph: 973-754-3349
New York
  Albany
 Albany Medical Center Hospital
 Vikramjit Kanwar
Ph: 518-262-5513x25265
  New York
 Herbert Irving Comprehensive Cancer Center at Columbia University Medical Center
 Clinical Trials Office - Herbert Irving Comprehensive Cancer Center at Columbia University Medical Center
Ph: 212-305-8615
  Syracuse
 SUNY Upstate Medical University Hospital
 Clinical Trials Office - SUNY Upstate Medical University Hospital
Ph: 315-464-5476
North Carolina
  Chapel Hill
 Lineberger Comprehensive Cancer Center at University of North Carolina - Chapel Hill
 Clinical Trials Office - Lineberger Comprehensive Cancer Center
Ph: 877-668-0683; 919-966-4432
  Charlotte
 Blumenthal Cancer Center at Carolinas Medical Center
 Clinical Trials Office - Blumenthal Cancer Center at Carolinas Medical Center
Ph: 704-355-2884
  Durham
 Duke Comprehensive Cancer Center
 Clinical Trials Office - Duke Comprehensive Cancer Center
Ph: 888-275-3853
  Winston-Salem
 Wake Forest University Comprehensive Cancer Center
 Clinical Trials Office - Wake Forest University Comprehensive Cancer Center
Ph: 336-713-6771
Ohio
  Akron
 Akron Children's Hospital
 Clinical Trials Office - Akron Children's Hospital
Ph: 330-543-3193
  Columbus
 Nationwide Children's Hospital
 Amanda Termuhlen
Ph: 614-722-3552
  Dayton
 Children's Medical Center - Dayton
 Emmett Broxson
Ph: 937-641-3111
Oklahoma
  Oklahoma City
 Oklahoma University Cancer Institute
 Rene McNall-Knapp
Ph: 405-271-5311
Oregon
  Portland
 Legacy Emanuel Hospital and Health Center and Children's Hospital
 Clinical Trials Office - Legacy Emanuel Hospital and Health Center and Children's Hospital
Ph: 503-413-8199
Pennsylvania
  Hershey
 Penn State Cancer Institute at Milton S. Hershey Medical Center
 Clinical Trials Office - Penn State Cancer Institute at Milton S. Hershey Medical Center
Ph: 717-531-3779
 Email: CTO@hmc.psu.edu
  Philadelphia
 Children's Hospital of Philadelphia
 Richard Aplenc
Ph: 267-426-7252
 St. Christopher's Hospital for Children
 Clinical Trials Office - St. Christopher's Hospital for Children
Ph: 215-427-8991
  Pittsburgh
 Children's Hospital of Pittsburgh
 Clinical Trials Office - Children's Hospital of Pittsburgh
Ph: 412-692-5573
South Carolina
  Greenville
 Greenville Hospital Cancer Center
 Clinical Trials Office - Greenville Hospital Cancer Center
Ph: 864-241-6251
South Dakota
  Sioux Falls
 Sanford Cancer Center at Sanford USD Medical Center
 Clinical Trials Office - Sanford Cancer Center
Ph: 605-328-1367
Tennessee
  Knoxville
 East Tennessee Children's Hospital
 Ray Pais
Ph: 865-541-8266
Texas
  Amarillo
 Texas Tech University Health Sciences Center School of Medicine - Amarillo
 Curtis Turner
Ph: 806-354-5434X264
  Corpus Christi
 Driscoll Children's Hospital
 Clinical Trials Office - Driscoll Children's Hospital
Ph: 361-694-5311
  Dallas
 Medical City Dallas Hospital
 Carl Lenarsky
Ph: 972-566-6647x4439
  Fort Worth
 Cook Children's Medical Center - Fort Worth
 Clinical Trials Office - Cook's Children's Medical Center
Ph: 682-885-2103
  San Antonio
 University of Texas Health Science Center at San Antonio
 Paul Thomas
Ph: 210-704-2187
Washington
  Seattle
 Children's Hospital and Regional Medical Center - Seattle
 Julie Park
Ph: 206-987-2106
  Spokane
 Providence Cancer Center at Sacred Heart Medical Center
 Judy Felgenhauer
Ph: 509-474-2777
West Virginia
  Charleston
 West Virginia University Health Sciences Center - Charleston
 Allen Chauvenet
Ph: 304-388-1552
Wisconsin
  Milwaukee
 Midwest Children's Cancer Center at Children's Hospital of Wisconsin
 Michael Kelly
Ph: 414-456-4170
Australia
New South Wales
  Westmead
 Westmead Institute for Cancer Research at Westmead Hospital
 Geoffrey McCowage
Ph: 61298452122
Western Australia
  Perth
 Princess Margaret Hospital for Children
 Catherine Cole
Ph: 011-6189340-8238
Canada
British Columbia
  Vancouver
 Children's & Women's Hospital of British Columbia
 Mason Bond
Ph: 604-875-2322
Nova Scotia
  Halifax
 IWK Health Centre
 Margaret Yhap
Ph: 902-470-8778
Quebec
  Montreal
 Hopital Sainte Justine
 Yvan Samson
Ph: 418-656-4141x47191
 Yvan Samson
Ph: 514-345-4969
Saskatchewan
  Saskatoon
 Saskatoon Cancer Centre at the University of Saskatchewan
 Kaiser Ali
Ph: 306-655-2744

Related Information

PDQ® clinical trial COG-A3961
PDQ® clinical trial COG-A3973
PDQ® clinical trial COG-ANBL00B1

Registry Information
Official Title A Phase III Randomized Trial of Intravenous Gammaglobulin Therapy for Patients with Neuroblastoma Associated Opsoclonus-Myoclonus-Ataxia Syndrome Treated with Chemotherapy and Prednisone
Trial Start Date 2004-03-15
Trial Completion Date 2010-01-01 (estimated)
Registered in ClinicalTrials.gov NCT00033293
Date Submitted to PDQ 2002-02-01
Information Last Verified 2009-11-20
NCI Grant/Contract Number CA13539

Note: The purpose of most clinical trials listed in this database is to test new cancer treatments, or new methods of diagnosing, screening, or preventing cancer. Because all potentially harmful side effects are not known before a trial is conducted, dose and schedule modifications may be required for participants if they develop side effects from the treatment or test. The therapy or test described in this clinical trial is intended for use by clinical oncologists in carefully structured settings, and may not prove to be more effective than standard treatment. A responsible investigator associated with this clinical trial should be consulted before using this protocol.

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