Detoxification of the Liver In PSC (Dolphin)

Launched by BRIGHAM AND WOMEN'S HOSPITAL · Apr 18, 2023

Keywords

ClinConnect Summary

The Dolphin clinical trial is studying a new medication called BRS201 to see if it can help people with a condition called Primary Sclerosing Cholangitis (PSC), which affects the liver and bile ducts. If you decide to participate, the study will last for 8 weeks, during which you will take the study medication for 4 weeks and a placebo (a dummy pill) for another 4 weeks. This will help researchers understand how effective BRS201 is. You'll need to take the medication twice a day and keep a log of your doses. Throughout the study, you will attend 9 remote visits, where a nurse will come to your home to collect blood and stool samples for testing.

To be eligible for the trial, you should have been diagnosed with PSC for at least 6 months and meet certain health criteria, such as having specific liver enzyme levels. However, there are some health conditions that may disqualify you from participating, like needing a liver transplant soon or having other serious liver diseases. This study is open to all genders, and participants must be between the ages of 18 and 80. If you meet the eligibility criteria, this trial could be a valuable opportunity to contribute to research that may help improve treatment for PSC.

Gender

ALL

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