Effectiveness of Rituximab in Pediatric OMS Patients.
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ClinicalTrials.gov Identifier: NCT00244361 |
Recruitment Status
:
Completed
First Posted
: October 26, 2005
Last Update Posted
: May 10, 2011
|
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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 or disease | Intervention/treatment | Phase |
---|---|---|
Opsoclonus-myoclonus Syndrome Opsoclonus Myoclonus Ataxia | Drug: rituximab | Phase 1 Phase 2 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 25 participants |
Allocation: | Non-Randomized |
Intervention Model: | Single Group Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official Title: | A Phase I Clinical Trial of Rituximab for Pediatric Opsoclonus-Myoclonus Syndrome |
Study Start Date : | June 2005 |
Actual Primary Completion Date : | December 2007 |
Actual Study Completion Date : | December 2007 |

- Determine the effectiveness & selectivity of rituximab at depleting CSF B-cells in OMS with intrathecal B-cell expansion. This requires CSF & blood lympocyte immunophenotyping prior to the first infusion & intervals for 1 year after the final infusion.
- Evaluate the clinical efficacy & safety of rituximab by clinical assessments, scoring of videotapes for neurological severity, and various blood tests prior to the first infusion and then at one, three, six, and twelve months post the final infusion.

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Ages Eligible for Study: | 6 Months to 19 Years (Child, Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- written consent from parents
- have symptomatic OMS
- have CSF B-cell expansion (>1% B-cells)
- adequate renal function as indicated by normal BUN [10-25 mg/dL] and creatinine [0.4-1.2 mg/dL]
- adequate liver function, as indicated by up to 2x normal AST [0-35 U/L] and ALT [0-35 U/L].
- men and women of reproductive potential must agree to use an acceptable method of birth control during treatment and for twelve months after completion of treatment
Exclusion Criteria:
- treatment with any investigational agent within 4 weeks of screening or 5 half-lives of the investigational drug (which ever is longer)
- receipt of a live vaccine within 4 weeks prior to enrollment
- previous treatment with Rituximab
- prior antibody therapy (does not include IVIg) within past 6 months
- history of severe allergic or anaphylactic reactions to humanized or murine monoclonal antibodies
- history of HIV (patients considered high risk will be screened)
- history of hepatitis B and/or hepatitis C (patients considered high risk will be screened)
- history of recurrent significant infection or history of recurrent bacterial infections
- known active bacterial, viral fungal mycobacterial, or other infection (including tuberculosis or atypical mycobacterial disease, but excluding fungal infections of nail beds) or any major episode of infection requiring hospitalization or treatment with i.v. antibiotics within 4 weeks of screening or oral antibiotics within 2 weeks prior to screening
- pregnancy (a negative serum pregnancy test should be performed for all women of childbearing potential within 7 days of treatment)
- significant cardiac (symptomatic arrhythmias or symptomatic structural heart disease) or pulmonary disease (including obstructive pulmonary disease)
- concomitant chemotherapy
- hemoglobin: >13.5 gm/dL or <10.0 gm/dL
- platelets: <100,000/mm or >500,000/mm K/cumm

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00244361
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 Neuroinflammation Organization, Inc. |
Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
Responsible Party: | Michael R Pranzatelli, MD, National Pediatric Myoclonus Center/ SIU School of Medicine |
ClinicalTrials.gov Identifier: | NCT00244361 History of Changes |
Other Study ID Numbers: |
IND #11,771 SCRIHS (04-112) |
First Posted: | October 26, 2005 Key Record Dates |
Last Update Posted: | May 10, 2011 |
Last Verified: | May 2011 |
Keywords provided by National Pediatric Neuroinflammation Organization, Inc.:
opsoclonus myoclonus |
Additional relevant MeSH terms:
Myoclonus Opsoclonus-Myoclonus Syndrome Syndrome Ocular Motility Disorders Disease Pathologic Processes Dyskinesias Neurologic Manifestations Nervous System Diseases Signs and Symptoms Central Nervous System Diseases Cranial Nerve Diseases |
Eye Diseases Paraneoplastic Syndromes, Nervous System Nervous System Neoplasms Neoplasms by Site Neoplasms Paraneoplastic Syndromes Neurodegenerative Diseases Rituximab Antineoplastic Agents Immunologic Factors Physiological Effects of Drugs Antirheumatic Agents |