Ensayos clínicos

Inclusion Criteria

• Male or female patients
• Age > 18 years 
• Able to provide written informed consent
• Diagnosis of Primary Sclerosing Cholangitis
• Scheduled for an ERCP as part of their clinical care
• Diagnosed with trisomy 7 on cytologic testing
• Willingness to utilize adequate contraception from screening to at least one month after the trial
  • If female, postmenopausal for at least 6 months or using contraceptive pill
  • For both females and males, surgically sterile, or using two forms of barrier contraception

Exclusion Criteria

• Cholangiocarcinoma, hepatocellular carcinoma, pancreatic adenocarcinoma or other malignancy ≤3 years of registration
• Other liver disease as determined by standard clinical, serological, imaging or histological criteria
• Secondary cause of sclerosing cholangitis (IgG cholangiopathy, autoimmune, post-surgical biliary stricture, radiation, human immunodeficiency syndrome)
• Cholestasis with a bilirubin of > 1.6 mg/dl (normal range 0.1 - 1.0 mg/dL)
• Decompensated cirrhosis, Child-Pugh Class B or C
• Child A cirrhosis with portal hypertension (i.e., splenomegaly, esophageal or gastric varices, or platelet count < 100,000/µl [normal range: 150 - 450 x 103/µL])
• Transaminase (AST [norm.: 8-48 U/L], ALT [norm.: 7-55 U/L]) elevation of more than three times the upper limit of the normal range
• Pregnancy
• Nursing mothers
• Uncontrolled intercurrent illness
• Concurrent administration of CYP3A modulators, Antiepileptics, Rifampin, St. Johns wort, Ketoconazole, protonpump-inhibitors
• Men or women unwilling to employ adequate contraception
• Abnormalities of the cornea by history
• Moderate diarrhea defined as defecation frequency of equal or more than 4/d for those with their colon, equal or more than 8/d for patients with a pouch, and high ostomy output with those with ostomy
• Known interstitial lung disease