Ataxia Clinical Trial
Official title:
A Phase I Clinical Trial of Rituximab for Pediatric Opsoclonus-Myoclonus Syndrome
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.
Status | Completed |
Enrollment | 25 |
Est. completion date | December 2007 |
Est. primary completion date | December 2007 |
Accepts healthy volunteers | No |
Gender | Both |
Age group | 6 Months to 19 Years |
Eligibility |
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 |
Allocation: Non-Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment
Country | Name | City | State |
---|---|---|---|
United States | National Pediatric Myoclonus Center, Department of Neurology, SIU School of Medicine, 751 N Rutledge St | Springfield | Illinois |
Lead Sponsor | Collaborator |
---|---|
National Pediatric Myoclonus Center | Genentech, Inc. |
United States,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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. | |||
Secondary | 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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