Study of Ambrisentan With Antifibrotic Agent Combination Therapy in Diffuse Systemic Sclerosis
Status:
Completed
Trial end date:
2016-07-01
Target enrollment:
Participant gender:
Summary
Systemic sclerosis is a chronic autoimmune connective tissue disorder with no universally
accepted disease modifying regimen. Recruiting patients for systemic sclerosis treatment
studies is difficult due to the limited availability of such patients and furthermore the use
of a placebo arm is often deemed unethical due to the poor survival of diffuse systemic
sclerosis patients.
Long-term controlled trials examining functional outcomes and survival from novel therapeutic
agents for systemic sclerosis are often difficult to undertake because of costs, rarity of
the disease and ethical issues with the use of a true placebo. Open label single center
studies while inferior to multicenter placebo controlled studies, have helped establish the
benefits of certain pharmaceutical agents in systemic sclerosis, and while not universally
accepted as disease modifying agents, have been used with some success to treat systemic
sclerosis.
The hypothesis on which we are basing this study is that an endothelin receptor antagonist
and disease modifying agent with antifibrotic properties will have additive influence on
fibrosis, inhibit cellular and humoral hyperactivity and interfere with smooth muscle
proliferation in the vessel wall. The combination of these two agents will also be the first
regimen to address the heterogeneity of scleroderma manifestations including ILD, pulmonary
arterial hypertension and skin manifestations