NCT03722576已完成2 期
Investigation of the Activity of Vidofludimus Calcium, a Novel, Orally Available, Small Molecule Inhibitor of Dihydroorotate Dehydrogenase, as a Treatment for Primary Sclerosing Cholangitis (PSC)
适应症
干预措施
相关药物
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 入组人数
- 18
- 试验地点
- 3
- 主要终点
- Subjects Who Experience a Positive Outcome as Measured by Combination of Serum Alkaline Phosphatase (ALP) and Aspartate Aminotransferase (AST) Levels.
研究概览
简要总结
To examine the safety, tolerability, and efficacy of daily dosing with vidofludimus calcium over a 6-month period.
详细描述
Investigators will assess the following:
- Changes on serum alkaline phosphatase levels at 3 & 6 months.
- Changes in other liver biochemistries at 3 & 6 months.
- Changes in IL-17 &IFNγ levels at 6 weeks and 6 months.
研究设计
- 研究类型
- Interventional
- 分配方式
- Na
- 干预模型
- Single Group
- 主要目的
- Treatment
- 盲法
- None
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
- •Male or female subject age 18-75 years
- •Diagnosis of PSC consistent with the guidelines published by the AASLD. All subjects must have an elevated serum ALP of at least 1.5 times upper limit of normal (ULN) at baseline plus cholangiographic evidence of PSC (MRI, endoscopic retrograde cholangiography, or direct cholangiography).
- •Indirect bilirubin <1.2 times the ULN
- •An ultrasound (or equivalent imaging modality) that excludes biliary obstruction and malignancy within 6 months of study enrollment
- •PSC with or without inflammatory bowel disease, such as ulcerative colitis or Crohn's disease
- •Must agree to comply with the study protocol and provide informed consent
排除标准
- •Pregnancy, attempting to become pregnant, or breastfeeding
- •Active hepatitis A or B infection
- •Active hepatitis C infection (antibody positive); patients with a history of hepatitis C infection will be eligible for this study if they have undetectable levels of HCV RNA
- •HIV/AIDS (per medical record or HIVAb/HIA antigen), tuberculosis, or positive interferon-gamma assay (IGRAs) for Mycobacterium tuberculosis
- •Other cholestatic liver disease such as primary biliary cholangitis and cholestatic diseases of pregnancy
- •Metabolic liver diseases such as Wilson's disease, Gilbert's syndrome or hemochromatosis
- •Serum uric acid levels at screening >1.2 ULN
- •Inherited diseases of the liver such as α-1 antitrypsin deficiency
- •Immunoglobulin G4-related cholangitis
- •PSC with concomitant autoimmune hepatitis (AIH) and/or primary biliary cholangitis
- •Secondary sclerosing cholangitis (SSC)
- •Active acute ascending cholangitis requiring antibiotics
- •CCA (malignant biliary stricture, neoplasm, and cytology/histopathology or positive fluorescence in situ hybridization (FISH) consistent with adenocarcinoma of the bile duct)
- •A liver biopsy, if one has been previously obtained, which showed non-alcoholic steatohepatitis (NASH). Patients with suspected fatty liver by imaging will not be excluded.
- •Presence of complications of advanced PSC such as hepatic encephalopathy, portal hypertension, hepato-renal syndrome, and hepato-pulmonary syndrome
- •History of liver transplantation, anticipated need for liver transplantation within 12 months from randomization, a Model of End-stage Liver Disease (MELD) score of ≥15, or a Child Pugh score >6
- •Ongoing alcohol abuse (>4 drinks per day for men, and >2 drinks per day for women)
- •Moderate-to-severe renal impairment with a calculated creatinine clearance of <60mL/min
- •Any other conditions or abnormalities that, in the opinion of the investigator, may compromise the safety of the subject or interfere with the subject participating in or completing the study
- •Evidence of, or treatment for, C. difficile infection within 30 days before the initiation of the study drug
- •Evidence of active C. difficile infection during the screening phase confirmed by a positive C. difficile toxin B
- •Subjects who have been treated for intestinal pathogens other than C. difficile infection within 30 days prior to study drug initiation
- •Received or plan to receive live vaccine within 30 days prior to, and through the end of the study
- •Use of methotrexate at dose ≥17.5mg/week
- •Rosuvastatin exceeding 10 mg daily
研究组 & 干预措施
Vidofludimus Calcium (VC)
Experimental
Daily dosing of VC over 6 months
干预措施: Vidofludimus calcium (Drug)
结局指标
主要结局
Subjects Who Experience a Positive Outcome as Measured by Combination of Serum Alkaline Phosphatase (ALP) and Aspartate Aminotransferase (AST) Levels.
时间窗: Baseline to 24 weeks
The number of subjects who have both an ALP reduction from baseline to week 24 that is greater or equal to 25% and their AST increase from baseline is less than or equal to 33% at week 24. ALP measured as international units per liter (IU/L). AST measured as international units per liter (IU/L).
次要结局
- Abnormal Total Bilirubin(24 weeks)
- Abnormal Alanine Aminotransferase (ALT)(24 weeks)
- Abnormal Aspartate Aminotransferase (AST)(Baseline to 24 weeks)
- Abnormal Direct Bilirubin(24 weeks)
研究者
Elizabeth Carey
Principal Investigator
Mayo Clinic
研究点 (3)
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