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Sponsors and Collaborators: |
National Pediatric Myoclonus Center Genentech |
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Information provided by: | National Pediatric Myoclonus Center |
ClinicalTrials.gov Identifier: | NCT00244361 |
The purpose of this study is to reduce the symptoms of OMS by testing rituximab (Rituxan®), to remove B lymphocytes that make antibodies and trigger brain inflammation. Evidence suggests that autoimmune brain inflammation causes the symptoms of OMS. This study of blood and spinal fluid intends to find out what effect rituximab has on OMS and on the spinal fluid B-cells.
Rituximab targets and destroys B-cells, which make antibodies that can attack the brain and cause may OMS. It is infused through a vein over a period of several hours. Rituximab has been used widely and studied extensively since its approval in 1997 by the U.S. Food and Drug Administration (FDA) for non-Hodgkin’s B-cell Lymphoma (NHL). Today, more than 300,000 patients have received rituximab, and it is part of more than 200 completed, ongoing, or planned clinical trials. Rituximab is not FDA-approved for OMS.
Condition | Intervention | Phase |
---|---|---|
Opsoclonus-Myoclonus Syndrome Opsoclonus Myoclonus Ataxia |
Drug: rituximab |
Phase I Phase II |
Study Type: | Interventional |
Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Safety/Efficacy Study |
Official Title: | A Phase I Clinical Trial of Rituximab for Pediatric Opsoclonus-Myoclonus Syndrome |
Estimated Enrollment: | 25 |
Study Start Date: | June 2005 |
Estimated Study Completion Date: | December 2007 |
Opsoclonus-myoclonus syndrome (OMS) is a rare but pervasive, paraneoplastic neurological disorder, purported to be autoantibody-mediated. We demonstrated expansion of B-cells in cerebrospinal fluid (CSF) despite tumor resection, chemotherapy, or conventional immunotherapy. Whether B-cells can be purged from the CSF compartment with benefit to the patient is unknown. Targeting of CSF B lymphocytes represents a novel and valuable paradigm shift in the therapy of centrally-mediated paraneoplastic disorders. The objective of this preliminary study is to determine if rituximab, a monoclonal antibody against CD20+ B-cells, reduces or eliminates CSF B-cells in OMS and whether the reduction results in clinical improvement. B lymphocyte subsets and relevant T-cell subsets will be immunophenotyped in the CSF and peripheral blood of children with OMS by four-color dual-laser flow cytometry. Sixteen children with an increased percentage of CSF B-cells will be treated with rituximab 375 mg/m2 IV once weekly for four consecutive weeks and CSF testing will be repeated at six months with more frequent clinical evaluations and blood testing out to 12 months. Clinical outcome will be rated blindly from videotapes by an experienced observer using a validated 12-item motor evaluation scale and quantifiable parameters of sleep, behavior and motor function. Immunological outcome variables will include percentages of B-cell subsets and quantitative immunoglobulins. Post-treatment results will be compared to pre-treatment values statistically. If rituximab proves to be an efficacious and safe method of treating CSF B-cell expansion and the neurological syndrome, this study will lead to a phase II trial with the eventual aim of gaining FDA approval of rituximab for this indication.
Ages Eligible for Study: | 6 Months to 19 Years |
Genders Eligible for Study: | Both |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
United States, Illinois | |
National Pediatric Myoclonus Center, Department of Neurology, SIU School of Medicine, 751 N Rutledge St | |
Springfield, Illinois, United States, 62794 |
Principal Investigator: | Michael R Pranzatelli, M.D. | National Pediatric Myoclonus Center |
Study ID Numbers: | IND #11,771, SCRIHS (04-112) |
Study First Received: | October 24, 2005 |
Last Updated: | April 19, 2007 |
ClinicalTrials.gov Identifier: | NCT00244361 |
Health Authority: | United States: Food and Drug Administration |
opsoclonus myoclonus |
Myoclonus Motor neuro-ophthalmic disorders Rituximab Opsoclonus-Myoclonus Syndrome Eye Diseases Central Nervous System Diseases Neurodegenerative Diseases Dyskinesias Ocular motility disorders |
Dancing eyes-dancing feet syndrome Ocular Motility Disorders Signs and Symptoms Paraneoplastic Syndromes Ataxia Neurologic Manifestations Paraneoplastic Syndromes, Nervous System Nervous System Neoplasms |
Disease Immunologic Factors Antineoplastic Agents Nervous System Diseases Physiological Effects of Drugs Pharmacologic Actions Neoplasms |
Pathologic Processes Neoplasms by Site Syndrome Therapeutic Uses Cranial Nerve Diseases Antirheumatic Agents |