Imatinib Mesylate (Gleevec) in the Treatment of Systemic Sclerosis
Status:
Completed
Trial end date:
2011-12-01
Target enrollment:
Participant gender:
Summary
The purpose of this study is to assess the safety and tolerability of imatinib mesylate
(Gleevec) in patients with systemic sclerosis (scleroderma). Gleevec is a medication already
FDA approved for the treatment of chronic myelogenous leukemia (CML), gastrointestinal
stromal tumors (GIST), dermatofibrosarcoma protuberans tumors, Philadelphia
chromosome-positive acute lymphoblastic leukemia, hypereosinophilic syndrome, and aggressive
systemic mastocytosis. In-vitro studies have suggested that imatinib may inhibit collagen
production by scleroderma fibroblasts, and in mouse models of fibrosis imatinib has been
shown to decrease skin thickness.
This is a Phase IIa, single center, prospective open label clinical trial of Gleevec in
patients with systemic sclerosis. All patients will be treated with active drug for 12
months. The primary objective of this study will be to determine the safety and tolerability
of Gleevec in patients with systemic sclerosis, but important secondary outcomes of relevance
will be improvement in disease status as defined by skin scores and indices of pulmonary
function.
Patients who complete the initial phase (described above) of the study will be eligible to
participate in an extension phase. The purpose of the extension phase of the study is to give
patients who participated in the phase IIa clinical trial of Gleevec at the Hospital for
Special Surgery the opportunity to continue Gleevec treatment if both the treating physicians
and the patient are in agreement that Gleevec had acceptable safety and tolerability, as well
as possible efficacy during the initial year of therapy.