中国临床试验注册中心 - 世界卫生组织国际临床试验注册平台一级注册机构

注册号： Registration number：	ChiCTR2600120336
最近更新日期： Date of Last Refreshed on：	2026-03-12 14:31:03
注册时间： Date of Registration：	2026-03-12 00:00:00
注册号状态：	预注册
Registration Status：	Prospective registration
注册题目：	伏欣奇拜单抗治疗CAPS、FMF、Schnitzler综合征及AOSD的多中心、开放标签、单臂、干预性篮式研究
Public title：	A multicenter, prospective, basket interventional study of vixencizumab in the treatment of CAPS, FMF, Schnitzler syndrome and AOSD
注册题目简写：
English Acronym：
研究课题的正式科学名称：	伏欣奇拜单抗治疗CAPS、FMF、Schnitzler综合征及AOSD的多中心、开放标签、单臂、干预性篮式研究
Scientific title：	A multicenter, prospective, basket interventional study of vixencizumab in the treatment of CAPS, FMF, Schnitzler syndrome and AOSD
研究课题代号(代码)： Study subject ID：
在二级注册机构或其它机构的注册号： The registration number of the Partner Registry or other register：

申请注册联系人：	沈敏	研究负责人：	沈敏
Applicant：	Min Shen	Study leader：	Min Shen
申请注册联系人电话： Applicant telephone：	+86 10 69155780	研究负责人电话： Study leader's telephone：	+86 10 6915 9956
申请注册联系人传真： Applicant Fax：		研究负责人传真： Study leader's fax：
申请注册联系人电子邮件： Applicant E-mail：	shenmpumch@163.com	研究负责人电子邮件： Study leader's E-mail：	shenmpumch@163.com
申请单位网址(自愿提供)： Applicant website(voluntary supply)：		研究负责人网址(自愿提供)： Study leader's website(voluntary supply)：
申请注册联系人通讯地址：	北京市东城区帅府园1号	研究负责人通讯地址：	北京市东城区帅府园1号
Applicant address：	Shuaifuyuan 1, Dongcheng District, Beijing	Study leader's address：	No.1 Shuaifuyuan, Wangfujing, Dongcheng District, Beijing
申请注册联系人邮政编码： Applicant postcode：		研究负责人邮政编码： Study leader's postcode：
申请人所在单位：	北京协和医院
Applicant's institution：	Peking Union Medical College Hospital
研究负责人所在单位：	中国医学科学院北京协和医院
Affiliation of the Leader：	Peking Union Medical College Hospital

是否获伦理委员会批准：	是
Approved by ethic committee：	Yes
伦理委员会批件文号： Approved No. of ethic committee：	I-26PJ0340	伦理委员会批件附件： Approved file of Ethical Committee：	查看附件View
批准本研究的伦理委员会名称：	中国医学科学院北京协和医院伦理审查委员会
Name of the ethic committee：	Ethics Review Committee of Peking Union Medical College Hospital, Chinese Academy of Medical Sciences
伦理委员会批准日期： Date of approved by ethic committee：	2026-02-03 00:00:00
伦理委员会联系人：	李佳月
Contact Name of the ethic committee：	Jiayue Li
伦理委员会联系地址：	北京市东城区帅府园1号
Contact Address of the ethic committee：	No.1 Shuaifuyuan, Wangfujing, Dongcheng District, Beijing
伦理委员会联系人电话： Contact phone of the ethic committee：	+86 10 69156874	伦理委员会联系人邮箱： Contact email of the ethic committee：	dott1994@163.com

研究实施负责（组长）单位：

中国医学科学院北京协和医院

Primary sponsor：

Peking Union Medical College Hospital, Chinese Academy of Medical Sciences

研究实施负责（组长）单位地址：

北京市东城区帅府园1号

Primary sponsor's address：

No.1 Shuaifuyuan, Wangfujing, Dongcheng District, Beijing

试验主办单位(项目批准或申办者)：

Secondary sponsor：

国家：	中国	省(直辖市)：	北京市	市(区县)：
Country：	China	Province：	Beijing	City：
单位(医院)：	中国医学科学院北京协和医院	具体地址：	北京市东城区帅府园1号
Institution hospital：	Peking Union Medical College Hospital, Chinese Academy of Medical Sciences	Address：	No.1 Shuaifuyuan, Wangfujing, Dongcheng District, Beijing

经费或物资来源：

长春金赛药业有限责任公司

Source(s) of funding：

Changchun GeneScience Pharmaceutical Co., Ltd

研究疾病：

成人起病的斯蒂尔病，家族性地中海热，冷炎素相关周期性综合征，Schnitzler综合征

Target disease：

AOSD, FMF, CAPS, and Schnitzler syndrome

研究疾病代码：

Target disease code：

研究类型：

干预性研究

Study type：

Interventional study

研究所处阶段：

其它

Study phase：

N/A

研究设计：

单臂

Study design：

Single arm

研究目的：

该研究旨在评估伏欣奇拜单抗在IL-1β驱动的罕见自身免疫和炎症性疾病（AOSD、FMF、CAPS、Schnitzler综合征）中的短期和长期疗效、安全性及生物标志物变化，以支持其临床应用和适应症拓展。

Objectives of Study：

To evaluate the short-term and long-term effect and safety of Firsekibart in IL-1beta driven autoimmune and autoinflammatory disease (AOSD, FMF, CAPS, and Schnitzler syndrome), to measure the changes of the biomarkers, which is supposed to support the application of the medication in clinical practice and broaden the indications of this agent.

药物成份或治疗方案详述：

Description for medicine or protocol of treatment in detail：

纳入标准：

Inclusion criteria

1. Written informed consent form signed by the subject must have been obtained.
2. Willing and committed to return to the study site to complete all study visits and all study-related procedures.
3. Male and female subjects aged >= 18 years and <= 75 years.
4. Divided into four cohorts (the investigator may add or remove disease types based on real-time study results): (1): AOSD: Subjects meeting the classification criteria for AOSD (Yamaguchi et al., J. Rheumatology, 1992), with a Physician Global Assessment (PGA, 0–4 scale) >= 2, considered to be in active disease. (2): CAPS: Subjects meeting the diagnostic criteria for CAPS, with NLRP3 mutation and requiring treatment, and a Physician Global Assessment (PGA, 0–4 scale) >= 2, considered to be in active disease. (3): Schnitzler syndrome: Subjects meeting the diagnostic criteria for SchS, with a Physician Global Assessment (PGA, 0–4 scale) >= 2, considered to be in active disease. (4): FMF: Subjects meeting the Tel Hashomer diagnostic criteria (1997), with a prior attack frequency of at least 1 per month. For patients receiving biologic therapy, this criterion applies to the last 12 months before initiation of any biologic therapy, and a Physician Global Assessment (PGA, 0–4 scale) >= 2, considered to be in active disease. And meeting one of the following:   Active disease despite colchicine treatment (at least 1.5 mg to 3.0 mg/day). Patients currently on colchicine will remain on a stable dose during the study.   Colchicine intolerance (at least 1.5 mg to 3.0 mg/day).
5. .If receiving NSAID therapy: only one NSAID at a stable dose for >= 1 week before enrollment (dose <= maximum recommended daily dose) is allowed (excluding temporary antipyretic use with <= 1 dose per day and <= 2 cumulative days within 2 weeks before dosing), and the dose will not be increased during the study.
6. .If receiving glucocorticoid therapy: stable dose <= 10 mg/day (prednisolone or equivalent) for at least 2 weeks before enrollment, and the dose will not be increased during the study.
7. If receiving disease-modifying antirheumatic drug (DMARD) therapy: stable dose for at least 3 months before enrollment, and the dose will not be increased during the study.
8. Women of childbearing potential must use an effective contraceptive method and have a negative pregnancy test before study initiation.

排除标准：

1.在给药前4周关节腔内、关节周围、肌内或静脉糖皮质激素给药或使用麻醉性镇痛药，但研究期间内允许的镇痛药（可待因和曲马多）除外；
2..既往使用过以下任何一种药物治疗： - 入组前1周内使用过阿那白滞素； -入组前 2 周内使用过托法替布、巴瑞替尼、乌帕替尼、氘可来昔替尼、阿布昔替尼、芦可替尼； - 入组前3周内使用过托珠单抗、氨苯砜和霉酚酸酯； - 入组前4周内使用过利纳西普、依那西普、沙利度胺、环孢素、生长激素； - 入组前8周内使用过阿达木单抗或静脉注射免疫球蛋白(ivIg); - 入组前 10 周内使用过戈利木单抗； - 入组前12周内使用过英夫利西单抗、6-氟尿嘧啶、硫唑嘌呤、环磷酰胺或苯丁酸氮芥； - 入组前12周内使用过来氟米特，除外已停用4周且已接受过加速消除治疗者(如口服消胆胺或活性碳)，需要提供相应的病史记录； - 入组前26周内使用过利妥昔单抗； - 入组前4周内或5个药物半衰期内(以较长时间者为准)参与过任何其他临床试验，或应用其它的生物制剂者；
3.免疫功能低下的患者，乙肝抗原（HBsAg）、丙型肝炎抗体和/或人类免疫缺陷病毒（HIV）抗体阳性；
4.反复发作或4周内活动性感染（细菌、真菌或病毒），存在反复侵袭性真菌感染病史者；入组前 7 天内存在需要采用全身用抗病原微生物药物（包括抗细菌、抗病毒、抗真菌等药物）控制的感染；
5.筛选期结核T-Spot试验阳性。但如果筛查前无潜伏性或活动性结核病史，无提示活动性结核病的体征或症状，近期未与活动性结核病患者密切接触，方可纳入研究；
6.肝功能异常 (胆红素升高TBL >1.5x ULN；丙氨酸转氨酶（ALT）或天冬氨酸转氨酶 (AST)≥3倍正常值；
7.血清肌酐浓度>1.5mg/dL，eGFR ≤29 mL/min/1.73 m2；
8.血常规异常（血红蛋白[Hb]≤100g/L，中性粒细胞≤2.5x109/L，血小板≤100x109/L）；
9.被诊断患有其他恶性肿瘤的患者（经治愈的宫颈癌和原位皮肤癌，或其他已手术治愈且至少 5 年未复发的肿瘤除外）；
10.任何已接受器官移植的患者，或即将接受器官移植的患者；
11.存在任何其他严重的基础疾病包括但不限于心血管或肺部或其它系统严重病史，经研究者判断可能会影响受试者参加研究、治疗及随访，影响受试者依从性或可能导致发生与研究药物相关并发症的受试者；
12.筛选前 6 个月内存在心包炎，心肌炎，细菌性心脏瓣膜或心内膜炎等病史；
13.患有间质性肺病、肺纤维化、肺泡蛋白沉积症或肺动脉高压等病史者；筛选前 6 个月内存在需要胃肠外使用糖皮质激素治疗的哮喘；筛选前 6 个月内存在需要药物干预的特应性皮炎；
14.患有如多发性硬化或其他脱髓鞘疾病，或 Felty's 综合征者。患有严重代谢性疾病、血液系统疾病或反复慢性感染等疾病史，经研究者判断不适合接受免疫抑制治疗者；
15.研究者评估不能进行皮下注射者，例如正在接受抗凝血药治疗、血小板减少、已知患有出血性疾病或存在特发性血小板减少性紫癜；
16.筛选时存在相关的症状、体征，经研究者评估怀疑存在 MAS 者；
17.严重的神经疾病或精神疾病；
18.在研究开始前30天内参与任何其他临床研究；
19.在基线前3个月内接种活疫苗；
20.过去12个月内酗酒或滥用药物≥400毫升（mL）；
21.对任何研究药物或类似化学类别的药物或任何辅料过敏；
22.怀孕或母乳喂养；
23.研究者认为不宜参加本试验的其它情况；

Exclusion criteria：

1.Administration of intra-articular, peri-articular, intramuscular, or intravenous glucocorticoids, or use of narcotic analgesics within 4 weeks prior to dosing, except for analgesics permitted during the study (codeine and tramadol). 2.Prior treatment with any of the following agents: Anakinra within 1 week before enrollment; Tofacitinib, baricitinib, upadacitinib, deucravacitinib, abrocitinib, ruxolitinib within 2 weeks before enrollment; Tocilizumab, dapsone, mycophenolate mofetil within 3 weeks before enrollment; Rilonacept, etanercept, thalidomide, cyclosporine, growth hormone within 4 weeks before enrollment; Adalimumab or intravenous immunoglobulin (ivIg) within 8 weeks before enrollment; Golimumab within 10 weeks before enrollment; Infliximab, 6‑mercaptopurine, azathioprine, cyclophosphamide, or chlorambucil within 12 weeks before enrollment; Leflunomide within 12 weeks before enrollment, except for subjects who discontinued leflunomide for 4 weeks and underwent accelerated elimination (e.g., oral cholestyramine or activated charcoal), with appropriate medical records provided; Rituximab within 26 weeks before enrollment; Participation in any other clinical trial, or use of other biological agents, within 4 weeks or 5 elimination half-lives before enrollment (whichever is longer). 3.Immunocompromised patients; positive results for hepatitis B surface antigen (HBsAg), hepatitis C antibody, and/or human immunodeficiency virus (HIV) antibody. 4.Recurrent or active infection (bacterial, fungal, or viral) within 4 weeks; history of recurrent invasive fungal infection; 5.Positive T-Spot assay for tuberculosis during screening.Subjects may be included only if there is no history of latent or active tuberculosis before screening, no signs or symptoms suggestive of active tuberculosis, and no recent close contact with patients with active tuberculosis. 6.Abnormal liver function: total bilirubin (TBL) > 1.5 × upper limit of normal (ULN); alanine aminotransferase (ALT) or aspartate aminotransferase (AST) >= 3 × ULN. 7.Serum creatinine concentration > 1.5 mg/dL; eGFR <= 29 mL/min/1.73 m². 8.Abnormal complete blood count: hemoglobin (Hb) <= 100 g/L, neutrophils <= 2.5 × 10⁹/L, platelets <= 100 × 10⁹/L. 9.Patients diagnosed with other malignant tumors (except cured cervical cancer, in situ skin cancer, or other surgically cured tumors with no recurrence for at least 5 years). 10.Any patient who has undergone organ transplantation or is scheduled to undergo organ transplantation. 11.Presence of any other severe underlying disease including but not limited to severe cardiovascular, pulmonary, or other systemic disease, which in the investigator’s judgment may interfere with the subject’s ability to participate in the study, undergo treatment and follow-up, affect subject compliance, or potentially lead to complications related to the study drug. 12.History of pericarditis, myocarditis, bacterial heart valve disease, or endocarditis within 6 months prior to screening. 13.History of interstitial lung disease, pulmonary fibrosis, pulmonary alveolar proteinosis, or pulmonary hypertension;asthma requiring parenteral glucocorticoid therapy within 6 months prior to screening;atopic dermatitis requiring pharmacologic intervention within 6 months prior to screening. 14.History of multiple sclerosis or other demyelinating diseases, or Felty’s syndrome;history of severe metabolic disease, hematological disease, recurrent chronic infection, etc., who in the investigator’s judgment are not suitable for immunosuppressive therapy. 15.Subjects who, in the investigator’s assessment, cannot undergo subcutaneous injection, e.g., those receiving anticoagulants, thrombocytopenia, known bleeding disorders, or idiopathic thrombocytopenic purpura. 16.Subjects with relevant signs or symptoms at screening suspected of having MAS by the investigator. 17.Severe neurological or psychiatric disorders. 18.Participation in any other clinical study within 30 days prior to study initiation. 19.Administration of live vaccine within 3 months prior to baseline. 20.History of alcoholism or drug abuse >= 400 mL within the past 12 months. 21.Hypersensitivity to any study drug, drugs of a similar chemical class, or any excipients. 22.Pregnancy or breastfeeding. 23.Any other conditions deemed inappropriate by the investigator for participation in this trial.

研究实施时间：

Study execute time：

从 From 2026-01-28 00:00:00至 To 2027-01-28 00:00:00

征募观察对象时间：

Recruiting time：

从 From 2026-03-20 00:00:00 至 To 2027-01-28 00:00:00

干预措施：

Interventions：

组别：	Cryopyrin蛋白相关周期性综合征（CAPS）	样本量：	15
Group：	Cryopyrin-Associated Periodic Syndrome (CAPS)	Sample size：	15
干预措施：	伏欣奇拜单抗	干预措施代码：
Intervention：	Firsekibart	Intervention code：

组别：	成人斯蒂尔病（AOSD）	样本量：	35
Group：	Adult-onset Still's disease (AOSD)	Sample size：	35
干预措施：	伏欣奇拜单抗	干预措施代码：
Intervention：	Firsekibart	Intervention code：

组别：	族性地中海热（FMF）	样本量：	25
Group：	Familial Mediterranean Fever (FMF)	Sample size：	25
干预措施：	伏欣奇拜单抗	干预措施代码：
Intervention：	Firsekibart	Intervention code：

组别：	Schnitzler综合征	样本量：	15
Group：	Schnitzler Syndrome	Sample size：	15
干预措施：	伏欣奇拜单抗	干预措施代码：
Intervention：	Firsekibart	Intervention code：

研究实施地点：

Countries of recruitment and research settings：

国家：	中国	省(直辖市)：	北京市	市(区县)：
Country：	China	Province：	Beijing	City：
单位(医院)：	中国医学科学院北京协和医院	单位级别：	三级甲等
Institution hospital：	Peking Union Medical College Hospital	Level of the institution：	Tertiary A

国家：	中国	省(直辖市)：	广东省	市(区县)：
Country：	China	Province：	Guangdong	City：
单位(医院)：	香港大学深圳医院	单位级别：	三级甲等
Institution hospital：	The University of Hong Kong-Shenzhen Hospital	Level of the institution：	Tertiary A

国家：	中国	省(直辖市)：	重庆市	市(区县)：
Country：	China	Province：	Chongqing	City：
单位(医院)：	重庆医科大学附属第二医院	单位级别：	三级甲等
Institution hospital：	The Second Affiliated Hospital of Chongqing Medical University	Level of the institution：	Tertiary A

国家：	中国	省(直辖市)：	河南省	市(区县)：
Country：	China	Province：	Henan	City：
单位(医院)：	河南科技大学第一附属医院	单位级别：	三级甲等
Institution hospital：	The First Affiliated Hospital of Henan University of Science and Technology	Level of the institution：	Tertiary A

测量指标：

Outcomes：

指标中文名：	2周缓解率	指标类型：	主要指标
Outcome：	remission rate by week 2	Type：	Primary indicator
测量时间点：	首次用药后2周	测量方法：	客观及主观评分量表
Measure time point of outcome：	2 weeks after the first dose	Measure method：	objective and subjective scale

采集人体标本：

Collecting sample(s)
from participants：

标本中文名：	无	组织：
Sample Name：	NA	Tissue：
人体标本去向	其它	说明
Fate of sample：	0thers	Note：

征募研究对象情况：

Recruiting status：

尚未开始

Not yet recruiting

年龄范围：

Participant age：

最小 Min age	18	岁 years
最大 Max age	75	岁 years

性别：

男女均可

Gender：

Both

随机方法（请说明由何人用什么方法产生随机序列）：

无

Randomization Procedure (please state who generates the random number sequence and by what method)：

None

是否公开试验完成后的统计结果:

Calculated Results after the Study Completed public access:

不公开/Private

盲法：	无
Blinding：	None

是否共享原始数据： IPD sharing	否No
共享原始数据的方式（说明：请填入公开原始数据日期和方式，如采用网络平台，需填该网络平台名称和网址）：	无
The way of sharing IPD”(include metadata and protocol, If use web-based public database, please provide the url)：	None
数据采集和管理（说明：数据采集和管理由两部分组成，一为病例记录表(Case Record Form, CRF)，二为电子采集和管理系统(Electronic Data Capture, EDC)，如ResMan即为一种基于互联网的EDC：	eCRF and EDC
Data collection and Management (A standard data collection and management system include a CRF and an electronic data capture：	eCRF and EDC
数据与安全监察委员会： Data and Safety Monitoring Committee：	有/Yes
注册人： Name of Registration：	2026-03-12 14:30:55