Request for Single Patient IND for Compassionate/Emergency Use of Tocilizumab

Emergency IND for Provision of Tocilizumab

Application for Compassionate Use of Tocilizumab in a Boy with Severe Polyarticular Juvenile Idiopathic Arthritis (JIA) Unresponsive to All Licensed Medications.

This trial is for a single subject with juvenile idiopathic arthritis unresponsive to all licensed treatments for JIA, and some licensed only for rheumatoid arthritis. He is currently wheelchair bound and completely dependent for activities of daily living and self-care. He is in constant pain, has abundant warmth and swelling of nearly all joints including TMJs, neck, shoulders, elbows, wrists, MCP and finger joints (even some DIP joints), hips, knees, ankles, subtalar and intertarsal joints, MTP and IP joints of the toes.

Drug at a dose of 8 mg/kg would be administered IV every 2 weeks, with close safety and monitoring studies.

Treatment would be monitored every 2 weeks. Efficacy will be measured using the ACR Pedi 30, 50 and 90 definitions as previously published, based on: 1) physician global assessment of disease activity; 2) parent/patient assessment of overall well-being; 3) functional ability; 4) number of joints with active arthritis; 5) number of joints with limited range of motion; and 6) erythrocyte sedimentation rate.

Inclusion Criteria:

• Subject X, polyarticular JIA, not responding to any other drug

Exclusion Criteria:

• Not identified subject