Detoxification of the Liver In PSC (Dolphin)

NCT ID: NCT05835505

Last Updated: 2025-11-06

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Clinical Phase

PHASE2

Total Enrollment

28 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-12-12

Study Completion Date

2026-12-01

Brief Summary

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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.

Detailed Description

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Conditions

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Primary Sclerosing Cholangitis

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

CROSSOVER

This a crossover study in which everyone will have a period of time on the active medication and a period of time on the placebo.
Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors
This is a double-blinded study.

Study Groups

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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.

Group Type ACTIVE_COMPARATOR

BRS201

Intervention Type DRUG

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 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.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

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.

Interventions

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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.

Intervention Type DRUG

Placebo

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.

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Brigham and Women's Hospital

OTHER

Sponsor Role lead

Responsible Party

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Joshua Korzenik

Director, Crohn's and Colitis Center

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Joshua A Korzenik, MD

Role: PRINCIPAL_INVESTIGATOR

Brigham and Womens Hospital

Locations

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Brigham and Women's Hospital

Chestnut Hill, Massachusetts, United States

Site Status RECRUITING

Countries

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United States

Central Contacts

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Sophie Mitchell, BS

Role: CONTACT

Phone: 5089363674

Email: [email protected]

Siani Ellis, BS

Role: CONTACT

Phone: 6177325500

Email: [email protected]

Facility Contacts

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Sophie A Mitchell, BS

Role: primary

Siani Ellis, BS

Role: backup

Other Identifiers

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2025P000196

Identifier Type: -

Identifier Source: org_study_id