Clinical trial · Interventional
Cyclophosphamide and Prednisone With or Without Immunoglobulin in Treating Abnormal Muscle Movement in Children With Neuroblastoma
A Phase III Randomized Trial of Intravenous Gammaglobulin Therapy for Patients With Neuroblastoma Associated Opsoclonus-Myoclonus-Ataxia Syndrome Treated With Chemotherapy and Prednisone
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Summary
Brief summary (as posted)
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.
Conditions
Conditions (5)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Localized Resectable Neuroblastoma | Neuroblastoma | CURATED_BROADER | 0.78 |
| Localized Unresectable Neuroblastoma | Neuroblastoma | CURATED_BROADER | 0.78 |
| Regional Neuroblastoma | Regional Neuroblastoma | ONTOLOGY_EXACT | 0.98 |
| Stage 4 Neuroblastoma | — | UNRESOLVED | — |
| Stage 4S Neuroblastoma | — | UNRESOLVED | — |
Interventions
Interventions (6)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| Clinical Observation | Other | — | UNRESOLVED |
| Cyclophosphamide | Drug | Cyclophosphamide | ALIAS |
| Laboratory Biomarker Analysis | Other | — | UNRESOLVED |
| Magnetic Resonance Imaging | Procedure | — | UNRESOLVED |
| Prednisone | Drug | Prednisone | ALIAS |
| Therapeutic Immune Globulin | Biological | — | UNRESOLVED |
Design
Arms and outcomes
Arms (2)
- type
- EXPERIMENTAL
- label
- Arm I (chemotherapy, immunoglobulin therapy)
- description
- 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. 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.
- interventionNames
- Drug: Cyclophosphamide
- Other: Laboratory Biomarker Analysis
- Procedure: Magnetic Resonance Imaging
- Drug: Prednisone
- Biological: Therapeutic Immune Globulin
- type
- ACTIVE_COMPARATOR
- label
- Arm II (chemotherapy, observation)
- description
Eligibility
Eligibility (as posted)
- Sex
- All
- Maximum age
- 8 Years
Show eligibility criteria text
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
References
Publications (1)
- DERIVEDde Alarcon PA, Matthay KK, London WB, Naranjo A, Tenney SC, Panzer JA, Hogarty MD, Park JR, Maris JM, Cohn SL. Intravenous immunoglobulin with prednisone and risk-adapted chemotherapy for children with opsoclonus myoclonus ataxia syndrome associated with neuroblastoma (ANBL00P3): a randomised, open-label, phase 3 trial. Lancet Child Adolesc Health. 2018 Jan;2(1):25-34. doi: 10.1016/S2352-4642(17)30130-X. Epub 2017 Nov 3. PMID 29376112