Safety and Efficacy Study of Bosentan in Progressive Pulmonary Sarcoidosis (BOPSAC)
Progressive pulmonary sarcoidosis occurs in up to twenty percent of patients who require persistent treatment, but available treatment options have shown considerable long-term toxicity and uncertain or unproven efficacy. In these patients, pulmonary fibrosis and pulmonary hypertension are common complications which have major prognostic impact. Endothelin-1 (ET-1) has been demonstrated to play a key role in pulmonary fibrosis and pulmonary hypertension, and a potential role in pulmonary sarcoidosis. ET-1 is a potent vasoconstrictor and can promote fibrosis, cell proliferation, and remodeling, and is pro-inflammatory. Preliminary data have shown the therapeutic potential of the endothelin receptor antagonist (ERA) bosentan in sarcoidosis associated pulmonary hypertension.
In this light, the therapeutic potential of bosentan as an add-on treatment in progressive pulmonary sarcoidosis needs to be evaluated.
|Study Design:||Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
|Official Title:||A Prospective Randomized, Double Blind, Placebo-controlled, Safety and Efficacy Study of Bosentan as add-on Therapy in Progressive Pulmonary Sarcoidosis|
- Treatment efficacy is assessed by a composite clinical score, including six parameters: Pulmonary function test (FVC and DLCO), Blood gas analysis (AaDO2), HRCT (Oberstein score), 6 minute walk test (6-MWD), Dyspnoea (ATS dyspnea scale) [ Time Frame: 6 months ] [ Designated as safety issue: No ]
- Assess safety and tolerability of bosentan in progressive pulmonary sarcoidosis [ Time Frame: 6 months ] [ Designated as safety issue: Yes ]
- To evaluate the efficacy of bosentan treatment in the subgroups of patents with and without sarcoidosis-associated pulmonary hypertension. [ Time Frame: 6 months ] [ Designated as safety issue: No ]
|Study Start Date:||April 2009|
|Study Completion Date:||March 2010|
|Primary Completion Date:||March 2010 (Final data collection date for primary outcome measure)|
Placebo Comparator: Placebo
identical preparation as the study drug, but without the active substance, administered b.i.d.
62.5 mg/125 mg bosentan b.i.d.
62.5 mg tablets b.i.d. administered orally for 4 weeks followed by the maintenance dose of 125 mg b.i.d. (62.5 mg b.i.d. if body weight < 40 kg/90 lb)
Please refer to this study by its ClinicalTrials.gov identifier: NCT00926627
|Vienna, Austria, 1180|
|General Hospital Vienna|
|Vienna, Austria, 1090|
|Study Director:||Daniel Doberer, MD, MSc||Medical University of Vienna|