The Assistance Publique – Hôpitaux de Paris is collaborating with Actelion Pharmaceutics on a study testing the efficacy, safety and tolerability of bosentan in patients with scleroderma renal crisis on renal function (ClinicalTrials.gov Identifier: NCT01241383).
Systemic sclerosis (SSc) is an auto-immune disorder that causes excessive collagen deposition, autoimmunity and vascular hyper-reactivity and obliterative microvascular phenomena that involves multiple organs. A condition that usually occurs in 5% of SSs patients is Scleroderma Renal Crisis (SRC). The routine use of angiotensin-converting enzyme inhibitors (ACEI) has been reported to dramatically improve outcomes, with a drop of the 12-month mortality from 76% to less than 15% in the United-States.
However, SRC remains a complicated manifestation of SSc with functional outcomes and survival numbers remaining poor. Bosentan is a dual endothelin receptor antagonist approved for the treatment of primary pulmonary arterial hypertension and for the prevention of ischemic digital ulcers.
In this non-randomized, single-group assignment efficacy study the primary endpoint is to explore the efficacy of bosentan (Tracleer) in patients with scleroderma renal crisis on renal function at 6 and 12 months. The secondary endpoints are to evaluate the safety and tolerability of bosentan in patients with scleroderma renal crisis over 6 months of treatment and 1 year overall survival.
Patients must be aged over 18 years, from both genders, able to provide informed consent; meeting the ACR /or LEROY et MEDSGER criteria for systemic sclerosis and fulfill criteria for renal systemic sclerosis. Patients cannot take part if they have taken bosentan within one month of inclusion for pulmonary arterial hypertension or digital ulcer prevention, or other treatments involving selective or nonselective antagonist endothelia receptors. In addition, those with a history of left ventricle systolic dysfunction, systolic blood pressure < 85mm Hg, progressive cancer or considered cured for less than 5 years cannot participate in the trial. Patients with a known hypersensitivity to bosentan or any of the excipients, with HIV, HCV, HBV infection or Liver disease Child-Pugh B and C cannot take part. Patients who are pregnant or breast-feeding are also excluded.
The study is recruiting participants until September 2015 at the Pôle de médecine interne Centre de référence Maladies rares Groupe at Hôpital Cochinin in Paris, France. The study is expected to be complete in March 2016. At the moment there are no results available.
More information about the study and how to enroll can be found at: https://clinicaltrials.gov/ct2/show/results/NCT01241383?term=scleroderma&rank=5