RecruitingPhase 2NCT05835505

Detoxification of the Liver In PSC (Dolphin)

Detoxification of the Liver in Primary Sclerosing Cholangitis

Sponsor

Brigham and Women's Hospital

Enrollment

28 participants

Start Date

Dec 12, 2023

Study Type

INTERVENTIONAL

Conditions

Primary Sclerosing Cholangitis

Summary

This study is a clinical trial being done to investigate the efficacy of drug BRS201 (hydroxocobalamin) as a treatment in patients with primary sclerosing cholangitis. Participation in this study will take 8 weeks long and the study is structured as a cross-over study in which participants will take the study drug for 4 weeks and a placebo drug for 4 weeks in a randomized order in the form of an oral medication. Participation may also involve receiving an IV dose of the medication. The study will require participants to attend 9 study visits, all of which will be remote. Participation will involve taking an oral medication twice daily, tracking the medication in a log, and getting blood drawn and giving a stool sample for a few lab tests throughout the study. For the lab tests, a research nurse will visit the participant in-home for the convenience of the participant.

Eligibility

Min Age: 18 YearsMax Age: 75 Years

Inclusion Criteria3

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 Criteria19

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 Check eligibility here: https://redcap.link/Checkmyeligibility

Interested in this trial?

Get notified about updates and connect with the research team.

Interventions

DRUGBRS201

Groups 1, 2, 3, and 4 will all contain 7 subjects each, with each subject receiving active study drug and placebo in a randomized order; half will receive the placebo first for four weeks followed by active treatment for four weeks, while the other half will receive active treatment for four weeks followed by placebo for four weeks.

DRUGPlacebo