NCT06297993招募中不适用
A Global Multi-Center Prospective Observational Cohort to Support Drug Development in Adult Patients With Primary Sclerosing Cholangitis (WIND-PSC)
适应症
试验速览
- 阶段
- 不适用
- 状态
- 招募中
- 入组人数
- 2,000
- 试验地点
- 25
- 主要终点
- Develop an appropriate real-world data (RWD) comparator cohort to support the design, execution, and serve as an external control for interventional clinical trials in PSC.
研究概览
简要总结
Develop an appropriate real-world data comparator cohort to support the design, execution, and serve as an external control for interventional clinical trials in PSC.
详细描述
Global, multi-center longitudinal observational cohort study. Collection of prospective clinical data to include liver-related clinical outcomes and safety events, hepatobiliary malignancies, relevant key biomarkers, imaging assessments, PSC-related clinical symptoms, patient-reported outcomes, and medication use in adult patients with PSC.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Other
入排标准
- 年龄范围
- 18 Years 至 75 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •Adult patients between 18 and 75 years of age (inclusive) who can comprehend instructions, follow the study procedures and are willing to sign an Informed Consent Form (ICF).
- •Confirmed clinical diagnosis of large duct PSC.
排除标准
- •Clinically significant acute or chronic liver disease of an etiology other than PSC (including but not limited to metabolic-dysfunction associated steatohepatitis (MASH), PBC, HCV, HBV, or alcoholic hepatitis, Wilson's disease, alpha-1 antitryp-sin deficiency, acute or chronic drug-induced liver injury)
- •Patients with PSC and elements of AIH overlap are allowed to enroll
- •Patients with metabolic dysfunction associated steatotic liver disease (MASLD) or benign steatosis are allowed to enroll
- •Small-Duct PSC.
- •Clinically diagnosed secondary or IgG4-related sclerosing cholangitis.
- •Clinically diagnosed infections (including acute cholangitis) and receiving treatment within the past 7 days; patients on chronic suppressive antibiotics for acute cholangitis will be allowed to enroll
- •Hospitalization in the past 7 days
- •UDCA dose >28 mg/kg
- •Evidence of current or historical decompensated cirrhosis based on the following clinical events:
- •Ascites > Grade 2 and requiring treatment
- •Esophageal or gastric variceal bleeding requiring hospitalization
- •Hepatic encephalopathy (as defined by a West Haven score ≥ 2)
- •Spontaneous bacterial peritonitis defined as ascites absolute neutrophil count >250/mm3 in the absence of an intra-abdominal source of infection
- •AKI-HRS according to AASLD Guidelines (Flamm 2021)
- •Portal hypertension based on a platelet count < 150 × 109/L and LSM > 15 kPa with clinical, laboratory, imaging and/or other relevant parameters
- •Prior liver transplantation
- •MELD 3.0 Score >
- •For subjects on anticoagulation medication, baseline INR determination for MELD score calculation should take this use into account.
- •History, evidence, or high suspicion of hepatobiliary malignancies or active colon cancer based on imaging, screening laboratory values, and/or clinical symptoms. Patients with a history of colon cancer who have undergone a colectomy and have no current evidence of colon cancer will be allowed to enroll in the study.
- •Participants with current clinical or laboratory evidence of any severe, progressive, or uncontrolled disease, related or unrelated to PSC and which, in the opinion of the investigator, has an expected survival of less than 52 weeks.
- •Participants who are impaired, incapacitated, or incapable of completing study-related assessments or giving informed consent.
- •Prisoners or participants who are involuntarily incarcerated.
- •Participants who are currently participating in an investigational PSC therapy clinical study or who have participated in such a study within the past 12 weeks
- •Absence of data in medical records to assess inclusion and exclusion criteria.
研究组 & 干预措施
Adult patients with a confirmed diagnosis of large duct PSC.
- Adult patients between 18 and 75 years of age (inclusive) who can comprehend instructions, follow the study procedures and are willing to sign an Informed Consent Form (ICF).
- Confirmed clinical diagnosis of large duct PSC
结局指标
主要结局
Develop an appropriate real-world data (RWD) comparator cohort to support the design, execution, and serve as an external control for interventional clinical trials in PSC.
时间窗: Quarterly for five years from enrollment
Collection of liver-related data including PSC symptoms, medical history, adverse events, and outcomes at enrollment and each quarterly and annual visit.
次要结局
- Evaluate direct participant experiences with standardized tools to determine changes over time, the association with clinical events, biomarkers, and disease progression(Quarterly for five years from enrollment)
- Develop a large clinical and biomarker data set to identify individual and/or composite surrogate end-points likely to predict clinical benefit for use in the design of interventional studies in PSC.(Quarterly for five years from enrollment)
研究者
研究点 (25)
Loading locations...
相似试验
进行中（未招募）
不适用
MiDura-Study (Neuro-Patch in Duraplasty)Tumor, BrainChiari MalformationVascular MalformationTethered CordDural FistulaCraniotomySpinal SurgeryDuraplastyHydrocephalusMeningiomaNCT04189172Aesculap AG328
招募中
不适用
Multi-center Collaborative to Enhance Quality and Outcomes in the Management of Cardiogenic ShockAcute Myocardial InfarctionCardiogenic ShockNCT05185492STAVROS G DRAKOS500
已完成
不适用
Pediatric Femur Fracture RegistryFemoral Shaft FractureNCT03211546AO Innovation Translation Center322
终止
不适用
An Observational Study of Patients Receiving T-DXd for Treatment of HER2+, and HER2-low Unresectable and/or Metastatic Breast CancerBreast CancerNCT05592483AstraZeneca78
已完成
不适用
A Study Evaluating the Associations Between Clinical Characteristics and Use of Oral Anticoagulants in Italian Elderly Participants With Non-Valvular Atrial FibrillationNon-Valvular Atrial Fibrillation (NVAF)NCT05232643Bristol-Myers Squibb636