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A Phase III Randomized Trial of Intravenous Gammaglobulin Therapy for Patients With Neuroblastoma Associated Opsoclonus-Myoclonus-Ataxia Syndrome Treated With Chemotherapy and Prednisone

Phase 3
8 Years
Open (Enrolling)
Disseminated Neuroblastoma, Localized Resectable Neuroblastoma, Localized Unresectable Neuroblastoma, Regional Neuroblastoma, Stage 4S Neuroblastoma

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Trial Information

A Phase III Randomized Trial of Intravenous Gammaglobulin Therapy for Patients With Neuroblastoma Associated Opsoclonus-Myoclonus-Ataxia Syndrome Treated With Chemotherapy and Prednisone


I. 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.

II. Determine whether immunosuppressive therapy with cyclophosphamide and prednisone is an
effective backbone therapy for OMA upon which to build additional treatment for these


I. Determine whether these regimens improve OMA syndrome in these patients. II. Determine
whether these regimens improve motor coordination in these patients.

III. Determine these regimens improve functional outcome in these patients. IV. Investigate
the biology of neuroblastoma associated OMA, with specific regard to magnetic resonance
imaging (MRI) findings, anti-neuronal antibodies, cerebrospinal fluid (CSF) findings and
tumor biology.

VI. Define better the long-term prognosis for neurologic recovery in the child with
neuroblastoma associated with OMA syndrome. VII. Compare the event-free and overall survival
of patients treated with these regimens.


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
for up to 10 years.

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

Type of Study:


Study Design:

Allocation: Randomized, Endpoint Classification: Efficacy Study, Intervention Model: Parallel Assignment, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

OMA change from baseline as assessed by Vineland Adaptive Behavior Scale (VABS)

Outcome Time Frame:

Change from baseline to 2 months, 6 months, and 1 year

Safety Issue:


Principal Investigator

Pedro De Alarcon

Investigator Role:

Principal Investigator

Investigator Affiliation:

Children's Oncology Group


United States: Food and Drug Administration

Study ID:




Start Date:

March 2004

Completion Date:

Related Keywords:

  • Disseminated Neuroblastoma
  • Localized Resectable Neuroblastoma
  • Localized Unresectable Neuroblastoma
  • Regional Neuroblastoma
  • Stage 4S Neuroblastoma
  • Neuroblastoma
  • Opsoclonus-Myoclonus Syndrome



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