Phase 3
N=53
Cyclophosphamide and Prednisone With or Without Immunoglobulin in Treating Abnormal Muscle Movement in Children With Neuroblastoma
Localized Resectable Neuroblastoma · Localized Unresectable Neuroblastoma · Regional Neuroblastoma · Stage 4 Neuroblastoma · Stage 4S Neuroblastoma
Bottom Line
View on ClinicalTrials.gov: NCT00033293 ↗Enrolled (actual)
53
Serious AEs
3.8%
Results posted
Oct 2016
Primary outcome: Primary: Number of Responders — 21; 11 participants — p=0.0044
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Clinical Observation (Other); Cyclophosphamide (Drug); Laboratory Biomarker Analysis (Other); Magnetic Resonance Imaging (Procedure); Prednisone (Drug); Therapeutic Immune Globulin (Biological)
- Age
- Pediatric
- Sex
- All
- Sponsor
- Children's Oncology Group
- Primary completion
- Dec 2013
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Responders |
21; 11 | 0.0044 sig |
| SECONDARY Motor Coordination as Assessed by Neurological Examination and Vineland Adaptive Behavior Scale (VABS) |
84.53; 144.73 | 0.0919 |
| SECONDARY Functional Outcome as Assessed by Age-appropriate Neuropsychological Testing |
117.5; 100.75 | 0.2364 |
| SECONDARY Biology of Neuroblastoma Associated Opsoclonus-myoclonus-ataxia (OMA) Syndrome Specifically by MRI Findings, Anti-neuronal Antibodies, Cerebrospinal Fluid (CSF) Findings and Tumor Biology |
— | — |
| SECONDARY Long-term Prognosis for Neurologic Recovery by Neurological Examination |
— | — |
| SECONDARY Tumor Outcome in Terms of Event-free Survival (EFS) Rate Defined as a Relapse or Progression of Neuroblastoma, a Second Malignancy, or Death |
92.3; 96.0 | — |
| SECONDARY Tumor Outcome in Terms of Overall Survival (OS) Rate |
100; 96.0 | — |
Summary
This randomized phase III 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. 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.
Eligibility Criteria
Inclusion Criteria
- 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
- 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)
- No prior IV gamma globulin therapy
- No prior chemotherapy
- Concurrent chemotherapy allowed
- No prior prednisone or corticotropin
- Patients who have received ≤ 14 days of steroids are eligible
- Concurrent surgery allowed
Data sourced from ClinicalTrials.gov (NCT00033293). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.