Detoxification of the Liver In PSC (Dolphin)

Description

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.

Conditions

Primary Sclerosing Cholangitis

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Study Overview

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Study Details

Study overview

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.

Detoxification of the Liver in Primary Sclerosing Cholangitis

Detoxification of the Liver In PSC (Dolphin)

Condition
Primary Sclerosing Cholangitis
Intervention / Treatment

-

Contacts and Locations

Chestnut Hill

Brigham and Women's Hospital, Chestnut Hill, Massachusetts, United States, 02467

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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).
  • * 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

Ages Eligible for Study

18 Years to 75 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Brigham and Women's Hospital,

Joshua A Korzenik, MD, PRINCIPAL_INVESTIGATOR, Brigham and Womens Hospital

Study Record Dates

2025-12-01