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Cyclophosphamide and Prednisone With or Without Immunoglobulin in Treating Abnormal Muscle Movement in Children With Neuroblastoma
This study is currently recruiting participants.
Verified by National Cancer Institute (NCI), November 2009
First Received: April 9, 2002   Last Updated: November 6, 2009   History of Changes
Sponsor: Children's Oncology Group
Collaborator: National Cancer Institute (NCI)
Information provided by: National Cancer Institute (NCI)
ClinicalTrials.gov Identifier: NCT00033293
  Purpose

RATIONALE: Drugs used in chemotherapy, work in different ways to stop tumor cells from dividing so they stop growing or die. Steroid therapy decreases inflammation. Combining chemotherapy and steroid therapy with immunoglobulin may be effective in treating abnormal muscle movement associated with neuroblastoma.

PURPOSE: This randomized phase II trial is studying cyclophosphamide, prednisone, and immunoglobulin to see how well they work compared to cyclophsophamide and prednisone alone in treating patients with abnormal trunk muscle movements associated with neuroblastoma.


Condition Intervention Phase
Neuroblastoma
Biological: therapeutic immune globulin
Procedure: observation
Phase III

Study Type: Interventional
Study Design: Treatment, Randomized, Active Control
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

Resource links provided by NLM:


Further study details as provided by National Cancer Institute (NCI):

Primary Outcome Measures:
  • Efficacy as measured by assessment of response of opsoclonus, gait, neural, stance, arm and hand function at baseline, 2 months, 6 months, and 1 year [ Designated as safety issue: No ]
  • Response as measured by assessment of opsoclonus, gait, neural, stance, arm and hand function at baseline, 2 months, 6 months, and 1 year [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Motor coordination as assessed by neurological examination and Vineland Adaptive Behavior Scale at baseline, 1 and 5 years [ Designated as safety issue: No ]
  • Functional outcome as assessed by age-appropriate neuropsychological testing at baseline, 1 and 5 years [ Designated as safety issue: No ]
  • 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 [ Designated as safety issue: No ]
  • Long-term prognosis for neurologic recovery by neurological examination at baseline, 1 and 5 years [ Designated as safety issue: No ]
  • Tumor outcome in terms of event-free survival and overall survival at 1, 5, and 10 years [ Designated as safety issue: No ]

Estimated Enrollment: 52
Study Start Date: March 2004
Estimated Primary Completion Date: January 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Arm I (immune globulin therapy): Experimental
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.
Biological: therapeutic immune globulin
Given IV
Arm II (immune globulin therapy): No Intervention
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.
Procedure: observation
Patients do not receive immune globulin.

Detailed Description:

OBJECTIVES:

  • 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.
  • Determine whether immunosuppressive therapy with cyclophosphamide and prednisone is an effective backbone therapy for OMA upon which to build additional treatment for these patients.
  • Determine whether these regimens improve OMA syndrome in these patients.
  • Determine whether these regimens improve motor coordination in these patients.
  • Determine whether these regimens improve functional outcome in these patients.
  • Compare the event-free and overall survival of patients treated with these regimens.

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 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.

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

  Eligibility

Ages Eligible for Study:   up to 8 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

DISEASE CHARACTERISTICS:

  • Newly diagnosed neuroblastoma with tumor-associated opsoclonus-myoclonus-ataxia syndrome

    • 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

PATIENT CHARACTERISTICS:

Age:

  • 8 and under

Performance status:

  • Not specified

Life expectancy:

  • Not specified

Hematopoietic:

  • Not specified

Hepatic:

  • Not specified

Renal:

  • Not specified

PRIOR CONCURRENT THERAPY:

Biologic therapy:

  • Not specified

Chemotherapy:

  • No prior chemotherapy
  • Concurrent chemotherapy allowed

Endocrine therapy:

  • No prior prednisone or corticotropin

Radiotherapy:

  • Not specified

Surgery:

  • Concurrent surgery allowed
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00033293

  Show 78 Study Locations
Sponsors and Collaborators
Children's Oncology Group
Investigators
Study Chair: Pedro A. de Alarcon, MD Saint Jude Midwest Affiliate
  More Information

Additional Information:
No publications provided

Responsible Party: Children's Oncology Group - Group Chair Office ( Gregory H. Reaman )
Study ID Numbers: CDR0000069271, COG-ANBL00P3
Study First Received: April 9, 2002
Last Updated: November 6, 2009
ClinicalTrials.gov Identifier: NCT00033293     History of Changes
Health Authority: Unspecified

Keywords provided by National Cancer Institute (NCI):
localized resectable neuroblastoma
regional neuroblastoma
disseminated neuroblastoma
stage 4S neuroblastoma
localized unresectable neuroblastoma

Additional relevant MeSH terms:
Anti-Inflammatory Agents
Prednisone
Neuroectodermal Tumors, Primitive
Immunologic Factors
Antineoplastic Agents
Opsoclonus-Myoclonus Syndrome
Physiological Effects of Drugs
Neoplasms, Nerve Tissue
Hormones, Hormone Substitutes, and Hormone Antagonists
Neurodegenerative Diseases
Hormones
Neuroblastoma
Ocular Motility Disorders
Neoplasms by Site
Therapeutic Uses
Neoplasms, Germ Cell and Embryonal
Rho(D) Immune Globulin
Nervous System Neoplasms
Immunoglobulins
Myoclonus
Neoplasms by Histologic Type
Antineoplastic Agents, Hormonal
Eye Diseases
Nervous System Diseases
Central Nervous System Diseases
Glucocorticoids
Dyskinesias
Pharmacologic Actions
Neuroectodermal Tumors
Neoplasms

ClinicalTrials.gov processed this record on November 09, 2009