2024-510715-30-00招募中2 期
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
试验速览
- 阶段
- 2 期
- 状态
- 招募中
- 入组人数
- 50
- 试验地点
- 10
- 主要终点
- Primary endpoint: to assess the number and percentage of patients who experience a 50% relative reduction in VAF after completing six cycles of Azacitidine treatment.
研究概览
简要总结
This trial's primary goal is to evaluate the effectiveness of Azacitidine therapy in reducing the UBA1 Variant Allele Frequency (VAF) in patients.
入排标准
- 年龄范围
- 18 years 至 65+ years（65+ Years, 18-64 Years）
- 接受健康志愿者
- 否
入选标准
- •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.
- •It is mandatory that male participants, who engage in sex with pre-menopausal (i.e. fertile) women, are willing to use barrier contraceptives (i.e condoms), during treatment with Azacitidine, and until three months after the last treatment. Additionally, it is highly recommended that their fertile partner also uses highly effective contraceptives during and for three months after the participants last treatment. 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 during treatment with Azacitidine, and until three months after the last treatment.
排除标准
- •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 maximum 8 weeks prior to initiating Azacitidine treatment.
- •Patients who are diagnosed with severe comorbidities including decompensated cirrhosis, end-stage kidney disease requiring dialysis, Severe cardiac disease (LVEF <30%), Severe pulmonary disease (FEV1 < 50% of predicted or DLCO < 40%), or a severe immunodeficiency including a diagnosis of HIV. If any of these conditions are suspected but undiagnosed, relevant tests will be ordered prior to screening based on clinical suspicion. However, diagnostic tests for all potential severe comorbidities will not be conducted systematically for every candidate; clinical suspicion will guide the decision to investigate further.
- •Patients with Eastern Cooperative Oncology Group (ECOG) Performance Status > 2
- •Patients who have a condition where Azacitidine is contraindicated, including hypersensitivity to Azacitidine, advanced malignant hepatic tumors, pregnancy, and breastfeeding individuals.
结局指标
主要结局
Primary endpoint: to assess the number and percentage of patients who experience a 50% relative reduction in VAF after completing six cycles of Azacitidine treatment.
Primary endpoint: to assess the number and percentage of patients who experience a 50% relative reduction in VAF after completing six cycles of Azacitidine treatment.
次要结局
未报告次要终点
研究者
Jakob Werner Hansen
Scientific
Rigshospitalet
研究点 (10)
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