Treatment for liver detoxification in patients with primary sclerosing cholangitis
Detoxification of the Liver in Primary Sclerosing Cholangitis
This study is testing a new oral medication called BRS201 to see if it can improve liver function and overall health in people with primary sclerosing cholangitis.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 28 (estimated) |
| Ages | 18 Years to 75 Years |
| Sex | All |
| Sponsor | Brigham and Women's Hospital Academic / other |
| Drugs / interventions | prednisone |
| Locations | 1 site (Chestnut Hill, Massachusetts) |
| Trial ID | NCT05835505 on ClinicalTrials.gov |
What this trial studies
This clinical trial investigates the efficacy of BRS201 (hydroxocobalamin) in treating patients with primary sclerosing cholangitis (PSC). The study is designed as a cross-over trial where participants will receive the study drug for 4 weeks followed by a placebo for another 4 weeks, with randomization in the order of administration. Participants will take the medication orally twice daily and will have 9 remote study visits, including in-home lab tests conducted by a research nurse. The trial aims to assess the drug's impact on liver function and overall health in PSC patients.
Who should consider this trial
Good fit: Ideal candidates are individuals diagnosed with primary sclerosing cholangitis for at least 6 months and meeting specific liver function criteria.
Not a fit: Patients who are anticipated to need a liver transplant within one year or have advanced liver disease may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could improve liver function and quality of life for patients with primary sclerosing cholangitis.
How similar studies have performed: Other studies have explored treatments for PSC, but the specific approach of using BRS201 is novel and untested.
Eligibility criteria
Show full inclusion / exclusion 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 Check eligibility here: https://redcap.link/Checkmyeligibility
Where this trial is running
Chestnut Hill, Massachusetts
- Brigham and Women's Hospital — Chestnut Hill, Massachusetts, United States (Recruiting)
Study contacts
- Principal investigator: Joshua A Korzenik, MD — Brigham and Womens Hospital
- Study coordinator: Sophie Mitchell, BS
- Email: smitchell22@bwh.harvard.edu
- Phone: 5089363674
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.