Details

Autor(en) / Beteiligte

• Morita, Akimichi
• Choon, Siew Eng
• Bachelez, Hervé
• Anadkat, Milan J.
• Marrakchi, Slaheddine
• Zheng, Min
• Tsai, Tsen-Fang
• Turki, Hamida
• Hua, Harry
• Rajeswari, Sushmita
• Thoma, Christian
• Burden, A. David

Titel

Design of Effisayil™ 2: A Randomized, Double-Blind, Placebo-Controlled Study of Spesolimab in Preventing Flares in Patients with Generalized Pustular Psoriasis

Ist Teil von

• Dermatology and therapy, 2023-01, Vol.13 (1), p.347-359

Ort / Verlag

Cheshire: Springer Healthcare

Erscheinungsjahr

2023

Link zum Volltext

Quelle

Nexis

Beschreibungen/Notizen

• Introduction Generalized pustular psoriasis (GPP) is a rare autoinflammatory skin disease characterized by flares of widespread erythema with sterile pustules, and can be relapsing with recurrent flares, or persistent with intermittent flares. Spesolimab, a humanized anti-interleukin-36 (IL-36) receptor monoclonal antibody, targets the key IL-36 pathogenetic pathway in GPP. A previous study showed that spesolimab treatment led to rapid pustular and skin clearance in patients with GPP flares, which was sustained for up to 12 weeks. This study investigates the long-term effects of spesolimab on GPP flares, for which no specific treatments are currently available. The Effisayil™ 2 study will assess whether maintenance treatment with subcutaneous spesolimab prevents the occurrence of GPP flares and determine the optimal dosing regimen to achieve this aim. Methods Patients will have a documented history of GPP with a Generalized Pustular Psoriasis Physician Global Assessment (GPPGA) score of 0 or 1 (clear or almost clear) at screening and randomization. Patients will be randomized 1:1:1:1 to three groups receiving a 600-mg subcutaneous loading dose of spesolimab followed by a 300-mg maintenance dose administered every 4 or 12 weeks, or a 300-mg loading dose followed by a 150-mg maintenance dose administered every 12 weeks, and one group receiving placebo, for 48 weeks. The primary endpoint is time to first GPP flare. If a patient experiences a GPP flare during the randomized maintenance treatment period, an open-label intravenous dose of 900-mg spesolimab will be administered, with an option for a second intravenous dose after 1 week. Conclusions Effisayil™ 2 is the first placebo-controlled study in patients with GPP to investigate whether maintenance treatment with spesolimab can prevent flares and provide sustained disease control. This study will provide valuable insights on the long-term management of patients with this potentially life-threatening skin disease. Trial Registration Number NCT04399837. Plain Language Summary The aim of the Effisayil™ 2 study is to see whether long-term treatment with the antibody spesolimab helps prevent skin flares in people with generalized pustular psoriasis (GPP). Patients can take part in the Effisayil™ 2 study if they have well-controlled GPP before they begin treatment in the study; that is, they will have skin that is clear or almost clear. Patients will be randomly divided into four groups, with similar numbers of patients in each group. In three of the four groups, patients will be given different doses of spesolimab for 48 weeks. In the fourth group, patients will be given a placebo for 48 weeks. The main goal of the study is to see how long it takes patients to have a GPP flare, while they are being given spesolimab or placebo. If any patient has a GPP flare during the study, they can be treated with another dose of spesolimab (and possibly a second dose 1 week later if needed), to help control the GPP flare. In this way, the Effisayil™ 2 study will help doctors and patients to learn how to manage GPP over time, so that GPP flares can be avoided.