Phase II clinical trial to test the efficacy of a novel oral treatment (BI 685509) in adults with progressive systemic sclerosis

The Yale Scleroderma Program is recruiting patients for an industry-sponsored Phase II clinical trial to test the efficacy of a novel oral treatment (BI 685509) in adults with progressive systemic sclerosis compared with placebo on a background of local standard of care therapy in the treatment of adult patients with progressive systemic sclerosis and vasculopathy. BI 685509 is intended to target the underlying fibrosis and vasculopathy that cause the manifestations of progressive systemic sclerosis. Trial results will inform further development of BI 685509 and future trial designs.

Eligibility criteria:

• dcSSc ≤7y OR lcSSc w/ Scl-70+ antibody ≤2y (from first non-Raynaud SSc manifestation)
• Active disease, defined as having at least one of the following:
• New SSc onset ≤ 2 years
• New skin involvement or worsening of two new body areas ≤ 6 m
• New involvement or worsening of chest or abdomen skin in previous 6 m
• Worsening of skin thickening (≥2 mRSS points) within previous 6 m
• ≥1 tendon friction rub
• mRSS ≥ 12 for dcSSc, mRSS ≥ 4 for lcSSc
• FVC ≥50% predicted
• CRP ≥ 6mg/L OR ESR ≥ 28mm/h OR KL-6 ≥ 1000 U/mL

*If none of criteria above are met, patient can be entered if modified Disease Activity Index is ≥ 2.5

• Evidence of significant vasculopathy, defined as:
• Active or documented history of digital ulcer(s)
• Previous treatment of Raynaud phenomenon with prostacyclin analogues or ≥ 1 other medications: nitrates, NO donors in any form, including topical; phosphodiesterase 5 (PDE5) inhibitors (e.g., sildenafil, tadalafil, vardenafil); nonspecific PDE5 inhibitors (theophylline, dipyridamole)

*If none of criteria above are met, patient can be entered with diagnosis of interstitial lung disease If prescribed, patients must be on stable NSAIDs, ACE inhibitors, calcium channel blockers, endothelin-receptor antagonists or glucocorticoid treatment for ≥2 weeks [prednisone (or equivalent drug) dose may not exceed 10mg/day]. Patients must be on stable mycophenolate mofetil/sodium, methotrexate, azathioprine for ≥ 4 weeks.