Rios Rodriguez Valeria, Sánchez-Riera Lidia, Haibel Hildrun, Höppner Caroline, Torgutalp Murat, Proft Fabian, Rademacher Judith, Binder Elke, Diehl Annette, Vranic Ivana, Zhao Yuxi, Mundayat Rajiv, Yndestad Arne, Poddubnyy Denis
Department of Gastroenterology, Infectious Diseases and Rheumatology (including Nutrition Medicine), Charité-Universitätsmedizin Berlin, Hindenburgdamm 30, Berlin 12203, Germany.
Pfizer SLU, Madrid, Spain.
Ther Adv Musculoskelet Dis. 2025 Mar 12;17:1759720X251324429. doi: 10.1177/1759720X251324429. eCollection 2025.
Early treatment initiation is one of the strongest predictors of good treatment response in axial spondyloarthritis (axSpA). Recently, the Assessment in SpondyloArthritis International Society (ASAS) defined early axSpA as a diagnosis of axSpA with a duration of axial symptoms equal to or less than 2 years. Tofacitinib is a Janus kinase (JAK) inhibitor for the treatment of ankylosing spondylitis.
Compare the efficacy and safety of tofacitinib versus placebo (both on non-steroidal anti-inflammatory drug (NSAID) background) in patients with active early axSpA and inadequate response to at least one NSAID.
This is a phase IV, randomized, double-blind, placebo-controlled, multicenter clinical trial.
The study will recruit 104 patients aged ⩾18 and ⩽45 years with active early axSpA (chronic back pain ⩽2 years), inadequate response to at least one NSAID, and objective signs of active inflammation (on magnetic resonance imaging (MRI) of sacroiliac joints (SIJs) or elevated C-reactive protein). Patients will be randomized 1:1 to receive tofacitinib 5 mg twice daily or placebo, with background naproxen 500 mg twice daily for 16 weeks. Patients not meeting early treatment response criteria at week 4 will receive open-label tofacitinib until week 16. Primary and key secondary endpoints at week 16 will be the proportion of patients achieving disease remission (Axial Spondyloarthritis Disease Activity Score <1.3) and change from baseline in MRI SIJ Spondyloarthritis Research Consortium of Canada osteitis score, respectively. Safety will be monitored up to 4 weeks after the last study drug dose.
The study will be performed according to the ethical principles of the Declaration of Helsinki and will be approved by independent ethics committees of each center.
This is one of the first randomized clinical trials designed to evaluate the efficacy and safety of a JAK inhibitor in the recently ASAS-defined "early" axSpA population. ClinicalTrials.gov: NCT06112665; CTIS: 2023-505050-18-00.
早期开始治疗是轴向性脊柱关节炎（axSpA）治疗反应良好的最强预测因素之一。最近，国际脊柱关节炎评估协会（ASAS）将早期axSpA定义为轴向症状持续时间等于或小于2年的axSpA诊断。托法替布是一种用于治疗强直性脊柱炎的 Janus激酶（JAK）抑制剂。
比较托法替布与安慰剂（均在非甾体抗炎药（NSAID）背景下）在活动性早期axSpA且对至少一种NSAID反应不足的患者中的疗效和安全性。
这是一项IV期、随机、双盲、安慰剂对照、多中心临床试验。
该研究将招募104名年龄在18至45岁之间、患有活动性早期axSpA（慢性背痛≤2年）、对至少一种NSAID反应不足且有活动性炎症客观体征（骶髂关节（SIJ）磁共振成像（MRI）或C反应蛋白升高）的患者。患者将按1:1随机分组，接受每日两次5mg托法替布或安慰剂治疗，同时每日两次服用500mg萘普生作为背景治疗，持续16周。在第4周未达到早期治疗反应标准的患者将接受开放标签的托法替布治疗直至第16周。第16周的主要和关键次要终点分别是达到疾病缓解（轴向性脊柱关节炎疾病活动评分<1.3）的患者比例和加拿大脊柱关节炎研究联盟MRI SIJ骨炎评分相对于基线的变化。在最后一剂研究药物给药后长达4周内监测安全性。
该研究将按照《赫尔辛基宣言》的伦理原则进行，并将获得各中心独立伦理委员会的批准。
这是首批旨在评估JAK抑制剂在最近ASAS定义的“早期”axSpA人群中的疗效和安全性的随机临床试验之一。ClinicalTrials.gov：NCT06112665；CTIS：2023 - 505050 - 18 - 00。
