Try our beta test site
IMPORTANT: Listing of a study on this site does not reflect endorsement by the National Institutes of Health. Talk with a trusted healthcare professional before volunteering for a study. Read more...
Trial record 1 of 1 for:    Dyssynergia Cerebellaris Myoclonica
Previous Study | Return to List | Next Study

Use of Rituximab in Opsoclonus-Myoclonus in Children With Neuroblastoma

The recruitment status of this study is unknown. The completion date has passed and the status has not been verified in more than two years.
Verified September 2006 by Tersak, Jean M., M.D..
Recruitment status was:  Recruiting
Genentech, Inc.
Information provided by:
Tersak, Jean M., M.D. Identifier:
First received: September 12, 2005
Last updated: September 19, 2006
Last verified: September 2006
The purpose of this study is to evaluate the feasibility of giving four weekly doses of Rituximab (anti-CD20 antibody) in the treatment of children with refractory neuroblastoma associated opsoclonus-myoclonus. Patients must have continued symptoms of opsoclonus, myoclonus and or ataxia despite surgical resection and a minimum of one month of steroid therapy. Evaluations include clinical symptoms of opsoclonus-myoclonus and ataxia as well as detailed evaluation of learning and development.

Condition Intervention Phase
Drug: anti-CD20 (Rituximab)
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Use of Rituximab in Opsoclonus-Myoclonus in Children With Neuroblastoma

Resource links provided by NLM:

Further study details as provided by Tersak, Jean M., M.D.:

Primary Outcome Measures:
  • Feasibility and toxicity
  • Response

Secondary Outcome Measures:
  • Pharmacokinetic data and HACA development in the pediatric population

Estimated Enrollment: 10
Study Start Date: July 2005
Estimated Study Completion Date: July 2008
Detailed Description:

Opsoclonus-myoclonus ataxia syndrome (OMS) is a rare immune mediated paraneoplastic syndrome that occurs in approximately 2 to 3% of children with neuroblastoma. Children with neuroblastoma associated opsoclonus-myoclonus tend to have a favorable prognosis from the standpoint of the cure of their cancer. Unfortunately,approximately two-thirds of this subgroup of patients are left with long term sequellae of the syndrome, including residual symptoms of opsoclonus, myoclonus, ataxia, learning difficulties and disturbance of sleep and mood.

Multiple lines of evidence indicate an immune mechanism to this rare disorder. This includes occurence of OMS in the post-infectious state, aggressive lymphocytic infiltration of the tumor in children with OMS, and documented responses to therapries that act through suppression of the immune system.

The current study utilizes four weekly doses of anti-CD 20 antibody (rituximab) to treat children with refractory OMS. Refractory disease is defined as continued symptoms of OMS despite surgical resection of the tumor and a minimum of one month of steroid therapy.

All patients have baseline OMS evaluation and detailed neurocognitive testing with all studies being repeated at the completion of the four weekly infusions. OMS testing is repeated at Month 3. OMS testing and detailed neurocognitive testing is conducted at 6 months intervals until 2 years from the initial infusion.

The goal of the study is to utilize this novel therapy to improve long term neurologic and neurodevelopmental outcome in children with refratory neuroblastoma associated opsoclonus-myoclonus.


Ages Eligible for Study:   2 Months to 18 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

Pathologic confirmation of diagnosis of neuroblastoma Surgical resection of primary tumor Symptoms of OMS despite a minimum of one month of steroid therapy Must meet all laboratory criteria to demonstrate adequate organ function -

Exclusion Criteria:

Patients currently receiving systemic chemotherapy for treatment of neuroblastoma Patients with documented active infection Patients who are HIV, Hep B or Hep C positive Organ toxicity from any prior therapy or surgical intervention must be resolved prior to study entry

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its identifier: NCT00202930

Contact: Jean M. Tersak, MD 412-692-5055

United States, Pennsylvania
Children's Hospital of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15213
Contact: Jean M Tersak, MD    412-692-5055   
Contact: Nina F Schor, MD, PhD    412-692-5528   
Principal Investigator: Jean M Tersak, MD         
Sponsors and Collaborators
Tersak, Jean M., M.D.
Genentech, Inc.
Principal Investigator: Jean M Tersak, M.D. Children's Hospital of Pittsburgh Department of Hematology Oncology and BMT
  More Information Identifier: NCT00202930     History of Changes
Other Study ID Numbers: IRB# 0405652
Study First Received: September 12, 2005
Last Updated: September 19, 2006

Keywords provided by Tersak, Jean M., M.D.:

Additional relevant MeSH terms:
Opsoclonus-Myoclonus Syndrome
Ocular Motility Disorders
Neuroectodermal Tumors, Primitive, Peripheral
Neuroectodermal Tumors, Primitive
Neoplasms, Neuroepithelial
Neuroectodermal Tumors
Neoplasms, Germ Cell and Embryonal
Neoplasms by Histologic Type
Neoplasms, Glandular and Epithelial
Neoplasms, Nerve Tissue
Paraneoplastic Syndromes, Nervous System
Nervous System Neoplasms
Neoplasms by Site
Paraneoplastic Syndromes
Central Nervous System Diseases
Nervous System Diseases
Cranial Nerve Diseases
Neurodegenerative Diseases
Neurologic Manifestations
Eye Diseases
Signs and Symptoms
Antineoplastic Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents processed this record on April 26, 2017