Recruiting

Phase 2

BRS201Detoxification of the Liver In PSC (Dolphin)

Sponsor:

Brigham and Women's Hospital

Code:

NCT05835505

Conditions

Primary Sclerosing Cholangitis

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

Interventions

BRS201

Placebo

Study Details

Brief 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.

Conditions

Primary Sclerosing Cholangitis

Study ID

NCT05835505

Start date

Dec 12, 2023

Status verified date

Jul, 2026

Completion date

Jun 1, 2027

Anticipated

Primary completion date

May 1, 2027

Anticipated

Eligibility Criteria

Sex: All

Age: 18 - 70+

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

28 participants

Anticipated

Allocation

Randomized

Intervention Model

Crossover

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

active comparator: BRS201 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 5g 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.

placebo comparator: Placebo Arm

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 5g 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.

Interventions

BRS201

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.

Placebo

Primary outcome measure

Alkaline Phosphatase (ALP) Test Result [ Time Frame: 4 weeks ]

Central Contacts and Locations

Central contacts

Sophie Mitchell, BS

5089363674 smitchell22@bwh.harvard.edu

Siani Ellis, BS

6177325500 pscresearch@bwh.harvard.edu

Locations

Brigham and Women's Hospital

Recruiting

Chestnut Hill, Massachusetts, United States, 02467

Contacts

Sophie A Mitchell, BS

508-936-3674 smitchell22@bwh.harvard.edu

Siani Ellis, BS

4699806602 sellis13@bwh.harvard.edu

Principal Investigator:

Joshua Korzenik, MD

More Information

Sponsor

Brigham and Women's Hospital

Last update posted

Jul 21, 2026

Last verified

Jul, 2026

Trial information was received from ClinicalTrials.gov and was last updated on 2026-09-10. This information was provided to ClinicalTrials.gov by Brigham and Women's Hospital on 2026-07-21.