This is a research study of an investigational drug called ambrisentan (Letairis) in the treatment and prevention of digital ulcers in patients with systemic sclerosis.
Ambrisentan is an endothelin blocker approved for the treatment of pulmonary arterial hypertension. Patients with systemic sclerosis can have damage to their blood vessels, resulting in increased levels of endothelin in their bloodstream that then causes decreased blood flow to the digits. This can result in very painful digital ulcers. We hope to learn whether blocking the action of endothelin with ambrisentan will be helpful in the treatment and prevention of digital ulcers in patients with systemic sclerosis. This therapy is not approved for the treatment of systemic sclerosis, but this study will help us learn whether ambrisentan is safe and effective in the treatment and prevention of digital ulcers in patients with systemic sclerosis.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
20
Stanford University School of Medicine
Stanford, California, United States
New Digital Ulcers (DU) 4 Weeks Prior to Week 24
The number of new digital ulcers (DU) that have developed in the preceding 4 weeks assessed at week 24 from baseline.
Time frame: 4 weeks prior to week 24
New DU 4 Weeks Prior to Week 12
The number of new DU that have developed in the preceding 4 weeks assessed at week 12 from baseline.
Time frame: 4 weeks prior to week 12
Subjects Experiencing Complete (Total Reepithelialization) Healing of All Baseline DU at Week 12.
Time frame: 12 weeks
Subjects Experiencing Complete Healing of > 50% of the Number of Baseline DU at Week 12.
Time frame: 12 weeks
Change in the Scleroderma Health Assessment Questionnaire (SHAQ) at Week 24 Compared With Baseline.
Range is 0-3 with 0 meaning the least amount of disability and 3 the greatest of disability.
Time frame: 24 weeks
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