Cyclophosphamide and Prednisone With or Without Immunoglobulin in Treating Abnormal Muscle Movement in Children With Neuroblastoma
Recruitment status was Recruiting
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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 cyclophosphamide and prednisone alone in treating patients with abnormal trunk muscle movements associated with neuroblastoma.
| Condition | Intervention | Phase |
|---|---|---|
|
Neuroblastoma |
Biological: therapeutic immune globulin Other: clinical observation |
Phase 3 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Primary Purpose: Treatment |
| 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 |
- Efficacy as measured by assessment of response of opsoclonus, gait, neural, stance, arm and hand function at baseline, 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, 6 months, and 1 year [ Designated as safety issue: No ]
- 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 |
|---|---|
|
Experimental: Arm I (immune globulin therapy)
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
|
|
No Intervention: Arm II (immune globulin therapy)
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.
|
Other: clinical 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 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| Ages Eligible for Study: | up to 8 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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
Contacts and Locations
Show 91 Study Locations| Study Chair: | Pedro A. de Alarcon, MD | Saint Jude Midwest Affiliate |
More Information
Additional Information:
No publications provided
| Responsible Party: | Gregory H. Reaman, Children's Oncology Group - Group Chair Office |
| ClinicalTrials.gov Identifier: | NCT00033293 History of Changes |
| Other Study ID Numbers: | CDR0000069271, COG-ANBL00P3 |
| Study First Received: | April 9, 2002 |
| Last Updated: | April 5, 2011 |
| 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:
|
Neuroblastoma Opsoclonus-Myoclonus Syndrome Neuroectodermal Tumors, Primitive, Peripheral Neuroectodermal Tumors, Primitive Neoplasms, Neuroepithelial Neuroectodermal Tumors Neoplasms, Germ Cell and Embryonal Neoplasms by Histologic Type Neoplasms Neoplasms, Glandular and Epithelial Neoplasms, Nerve Tissue Paraneoplastic Syndromes, Nervous System Nervous System Neoplasms Neoplasms by Site Paraneoplastic Syndromes |
Ocular Motility Disorders Central Nervous System Diseases Nervous System Diseases Cranial Nerve Diseases Neurodegenerative Diseases Myoclonus Dyskinesias Neurologic Manifestations Eye Diseases Antibodies Immunoglobulins Immunoglobulins, Intravenous Rho(D) Immune Globulin Prednisone Immunologic Factors |
ClinicalTrials.gov processed this record on May 19, 2013