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

Basic Trial Information

Trial Description

Summary

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 cyclophosphamide and prednisone alone in treating patients with abnormal trunk muscle movements associated with neuroblastoma.

Further Study Information

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 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.

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 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

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)

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

Trial Contact Information

Trial Lead Organizations/Sponsors

Children's Oncology Group

Pedro A. de Alarcon

Study Chair

Link to the current ClinicalTrials.gov record.
NLM Identifer NCT00033293
Information obtained from ClinicalTrials.gov on April 04, 2013

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