VEX-AZA: Treating VEXAS Syndrome with Azacitidine. A Single-Arm, Multicenter Phase II Study on the Efficacy and Safety of Azacitidine in Patients with VEXAS Syndrome

Rigshospitalet0 个研究点目标入组 16 人开始时间: 2024年2月13日最近更新: 2024年7月22日

适应症

试验速览

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•Participants must possess one of the currently described UBA1 mutations with a VAF > 10%. The currently described UBA1 mutations are: p.Met41Leu (c.121A.C), p.Met41Val (c.121A.G), p.Met41Thr (c.122T.C), p.Ser56Phe (c.167C>T), p.(splice)(c.118-1G>C) or intronic deletions like (c.118-9_118-2del) affecting the splice site, Participants must meet one of the following conditions: (1) Present with any degree of cytopenia, or (2) Exhibit inflammatory symptoms of VEXAS with/without cytopenia., Participants must be at least 18 years old and possess the cognitive capacity to provide informed consent., Fertile female participants must exhibit a negative result on a pregnancy test before inclusion, demonstrate a commitment to monthly pregnancy testing and employment of safe contraceptive measures throughout the duration of the clinical protocol.

•Patients having received Prior therapy with hypomethylating agents (i.e., Azacitidine, Decitabine)., Patients having received or is planned to receive alloHSCT., Patients with active cancer requiring treatment are excluded from participation in this study. Active cancer is defined as a diagnosis of cancer for which the patient is currently receiving treatment, such as chemotherapy, radiation therapy, immunotherapy, targeted therapy, or any other curative or disease-controlling intervention., Concomitant treatment with Disease modifying antirheumatic drugs (DMARDs), Chemotherapy/Cytostatic agents, Immunosuppressives, Radiotherapy any novel biological response modifying agents, is not allowed in the trials regardless of the underlying condition for which they are used. These treatments must be discontinued at least 14 days prior to inclusion., Failure to obtain bone marrow examination for at least a month prior to initiating Azacitidine treatment., Patients with these conditions might have a higher risk of adverse events or complications during the study, and their presence could confound the interpretation of treatment effects. Excluding them aims to ensure the well-being of participants and the internal validity of the study., Patients who have a condition where Azacitidine is contraindicated, including hypersensitivity to Azacitidine, advanced malignant hepatic tumors, pregnancy, and breastfeeding individuals.

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