Detoxification of the Liver In PSC (Dolphin)
ConditionPrimary Sclerosing Cholangitis
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-75
SponsorBrigham and Women's Hospital
About this trial
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 criteria
This trial does not accept healthy volunteersQualifiers
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).
Disqualifiers
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
Trial design
Design model
Crossover
Treatments tested in this trial
BRS201
DrugGroups 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
DrugGroups 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.
Treatment groups
28 Participants
are divided into 2 treatment groupsGroup A: BRS201 ArmActive comparator 1 intervention
Group B: Placebo ArmPlacebo comparator 1 intervention
Trial outcomes
Primary outcomes
1
Alkaline Phosphatase (ALP) Test Result
The primary endpoint for this study is the capacity of BRS201 to normalize alkaline phosphatase levels from baseline (week 0) compared to end of active treatment (4 weeks).
Time frame
4 weeksSponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov