Proof of Concept Trial of Gleevec (Imatinib) in Active Diffuse Scleroderma
Status:
Terminated
Trial end date:
2008-12-01
Target enrollment:
Participant gender:
Summary
The purpose of this study is to investigate the effectiveness and safety of the drug Gleevec
(imatinib) as a new treatment for patients with active diffuse scleroderma. This drug has not
been used previously to treat scleroderma, but it has been found to advance the treatment and
life span of patients with a type of leukemia called chronic myeloid leukemia or CML. Gleevec
acts on chemical signals in the cells that may decrease fibrosis (the hardening of the skin
that occurs in scleroderma). It works by interfering in the process that activates many
molecules that cause fibrosis, including TGFbeta (which may be a key part of disease activity
in scleroderma).
This study proposes to treat patients that have significant diffuse scleroderma with Gleevec
for 6 months and investigate several measures of scleroderma disease activity before, during
and at the end of treatment (0, 3 months and 6 months). This is a randomized, double blind,
placebo-controlled trial: 20 patients will be divided into two groups in a 4:1 ratio, with 16
patients taking 400mg of Gleevec per day and 4 taking a placebo. The differences between the
groups that will be measured include safety, Modified Rodnan skin score (mRSS), Health
Assessment Questionnaire (HAQ), global assessments (100mm VAS) and changes in biomarkers in
blood and skin biopsies.