Detoxification of the Liver In PSC (Dolphin) (DOLPHIN)
Primary Sclerosing Cholangitis
About this trial
This is an interventional treatment trial for Primary Sclerosing Cholangitis
Eligibility Criteria
Inclusion Criteria: A diagnosis of PSC for at least 6 months based upon cholangiography (ERCP or MRCP) demonstrating intrahepatic and/or extrahepatic biliary strictures, beading or irregularity consistent with PSC. ALP > 1.5 times the upper limit of normal (ULN) at screening. Subject must either be on a stable dose of ursodeoxycholic acid for > 6 months prior to screening or have been discontinued > 4 weeks prior to screening (enrollment of patients who are on UDCA will be limited to 60% of all enrolled patients). Exclusion Criteria: Anticipated need for liver transplant within one year as determined by Mayo PSC risk score Evidence of decompensated liver disease such as variceal bleeding, ascites, or hepatic encephalopathy. Evidence of advanced liver disease including MELD score > 10, bilirubin > 3.0, platelet count < 100,000; or INR > 1.4 Concomitant chronic liver disease including alcohol related liver disease, chronic hepatitis B or C infection, haemochromatosis, Wilson's disease, alpha1-antitrypsin deficiency, non-alcoholic steatohepatitis, autoimmune hepatitis, or primary biliary cholangitis Secondary causes of sclerosing cholangitis Patients who have a confirmed malignancy or cancer within 5 years except non-melanoma skin cancers Treatment with any investigational agents, within two months or 5 half-lives of the investigational product, whichever is longer. Active illicit drug or more than moderate alcohol consumption. Evidence of bacterial cholangitis within 6 months of enrollment In patients with Ulcerative Colitis, or, if Crohn's disease, a need for additional therapy at time of screening. Chronic kidney injury (eGFR < 60) Pregnancy or lactation Uncontrolled hypertension with a systolic BP > 140 and a systolic BP > 90 Prohibited medications: current use of vitamin C and prednisone Patients with a history or risk of cardiovascular conditions, including arrhythmia, long QT syndrome, congestive heart failure, stroke, or coronary artery disease Patients with a history of kidney stones Congenital or acquired immunodeficiencies Other comorbidities including: diabetes mellitus, systemic lupus An episode of acute cholangitis within 4 weeks of screening
Sites / Locations
Arms of the Study
Arm 1
Arm 2
Active Comparator
Placebo Comparator
Interventional Arm
Placebo Arm
In Group 1 of the study, subjects will take oral study drug at 1.2g twice daily, PO (2.4g daily) for 4 weeks. In Group 2 of the study, subjects will take oral study drug at 2g twice daily, PO (4g daily) for 4 weeks. In Group 3 of the study, subjects will take receive a one-time 4g IV dose of the study drug using the FDA-approved IV product followed by the oral study drug at 2g twice daily, PO (4g daily) for 4 weeks. In Group 4 of the study, subjects will repeat the previous conditions of the group that proves to be the most effective.
In Group 1 of the study, subjects will take oral placebo at 1.2g twice daily, PO (2.4g daily) for 4 weeks. In Group 2 of the study, subjects will take oral placebo at 2g twice daily, PO (4g daily) for 4 weeks. In Group 3 of the study, subjects will take receive a one-time 4g IV dose of the study drug using the FDA-approved IV product followed by the oral placebo at 2g twice daily, PO (4g daily) for 4 weeks. In Group 4 of the study, subjects will repeat the previous conditions of the group that proves to be the most effective.