Effectiveness of Rituximab in Pediatric OMS Patients.

• Status: Completed
• Trial end date: 2007-12-01
• Target enrollment: 0
• Participant gender: All

Summary

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.

• Phase: Phase 1/Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: National Pediatric Myoclonus Center; National Pediatric Neuroinflammation Organization, Inc.
• Collaborator: Genentech, Inc.
• Treatments: Rituximab

Criteria

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