Nerandomilast for Systemic Sclerosis

(VERANDA™-SSc Trial)

This trial tests a new medicine, nerandomilast, to determine its effectiveness for systemic sclerosis, a condition that hardens and tightens the skin and can affect internal organs. Researchers aim to discover if nerandomilast can improve symptoms and slow disease progression. Participants are randomly assigned to take either nerandomilast or a placebo (a pill with no active medicine) twice daily. The trial seeks individuals who have had systemic sclerosis for less than seven years from their first symptom (excluding Raynaud's phenomenon) and are experiencing active disease. Participants will regularly visit the study site for check-ups and tests to monitor their health and any changes in their condition. As a Phase 3 trial, this study represents the final step before FDA approval, offering participants a chance to contribute to a potentially groundbreaking treatment.

You may not need to stop your current medications if they are stable and permitted by the trial. However, all treatments must remain stable before and during the screening period.

In previous studies, nerandomilast has proven to be safe. Research indicates that its side effects were similar to those of a placebo, a harmless pill used for comparison. Importantly, people taking nerandomilast did not experience an increase in serious issues like lung infections or pneumonia. This suggests that most patients can tolerate the treatment well. Additionally, studies involving individuals with other lung conditions found that nerandomilast is generally safe, with few needing to stop treatment due to side effects. While more information is always useful, these findings suggest that nerandomilast could be a safe option for people with systemic sclerosis.¹²³⁴⁵

Nerandomilast is unique because it offers a new approach to treating systemic sclerosis by targeting specific inflammatory pathways that current treatments might not address as directly. Unlike the standard options, which often include immunosuppressants and vasodilators, Nerandomilast is designed to modulate the immune response more precisely. Researchers are excited about this treatment because it could lead to improved outcomes with potentially fewer side effects, offering hope for a condition that has been challenging to manage effectively.

Research has shown that nerandomilast, which participants in this trial may receive, may help with conditions like systemic sclerosis by slowing the decline in lung function. In previous studies, nerandomilast significantly reduced lung problems in patients with conditions similar to systemic sclerosis. It blocks certain enzymes, potentially helping to control fibrosis—a major issue in systemic sclerosis. While nerandomilast has shown promise, not all patient-reported outcomes improved. Overall, early evidence suggests that nerandomilast could help treat symptoms of systemic sclerosis.¹²⁴⁶⁷