DESCRIPTION (provided by applicant): The goal of the proposed efforts is to prepare a group of international myosotis investigators to successfully complete a double-blind, placebo-controlled trial of the safety and efficacy of intravenous infliximab and intravenous gamma globulin in refractory dermatomyositis (DM) patients receiving corticosteroids and methotrexate. This will be accomplished in part by sponsoring the completion of ongoing multi-center studies in adult and juvenile DM, which are developing and validating disease activity and damage assessment tools for use in clinical trials. They will conduct surveys, meetings and studies to accomplish the following goals: 1: To use the core set of outcome measures recently identified by an international group of specialists to develop a myosotis disease activity index, a preliminary definition of improvement and a damage index for adult and juvenile myosotis; 2: To discuss and resolve issues related to the general conduct of clinical trials in myosotis, which will include defining: the extent of improvement to be expected and the amount of damage acceptable over the course of a trial; specific inclusion and exclusion criteria; appropriate background therapy; trial duration; trial dropout criteria; safety assessment; and appropriate post-trial evaluations; and 3: To finalize details for a clinical trial to assess the safety and efficacy of intravenous infliximab and intravenous gamma globulin in refractory adult and pediatric DM patients receiving corticosteroids and methotrexate. This will include: selection of participating centers and personnel; finalization of all trial design issues; and the preparation of an investigator brochure. This study will develop the validated outcome measures needed and set the stage to allow for the successful completion of large multicenter trials in the Idiopathic Inflammatory myopathies.
|Effective start/end date||9/30/01 → 9/29/03|
- National Institutes of Health: $107,250.00