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

Phase	Type	Status	Age	Sponsor	Protocol IDs
Phase III	Treatment	Active	8 and under	NCI	COG-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

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

Outcomes

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

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

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
	Long Beach
	Jonathan Jaques Children's Cancer Center at Miller Children's Hospital
		Jerry Finklestein	Ph:  562-492-1062
	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 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
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
	Saint 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
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
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
Maryland
	Baltimore
	Alvin and Lois Lapidus Cancer Institute at Sinai Hospital
		Joseph Wiley	Ph:  410-601-5864
Michigan
	Flint
	Hurley Medical Center
		Clinical Trials Office - Hurley Medical Center	Ph:  810-762-8057
	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
Mississippi
	Jackson
	University of Mississippi Cancer Clinic
		Gail Megason	Ph:  601-984-5220
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
	Newark
	Newark Beth Israel Medical Center
		Clinical Trials Office - Newark Beth Israel Medical Center	Ph:  973-926-3136
	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
South Carolina
	Greenville
	Greenville Hospital Cancer Center
		Clinical Trials Office - Greenville Hospital Cancer Center	Ph:  864-241-6251
Tennessee
	Knoxville
	East Tennessee Children's Hospital
		Ray Pais	Ph:  865-541-8266
Texas
	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
	Methodist Children's Hospital of South Texas
		Michael Grimley	Ph:  210-575-7268
	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
		Christopher Mpofu	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		2010-02-09
NCI Grant/Contract Number		CA13539

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