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