The primary and secondary objectives and endpoints will be evaluated in participants with PSC who have a baseline score of ≥4 on the Adult Itch-Reported Outcome (ItchRO), as assessed during the single-blind, placebo run-in period during the core study.
Volixibat is an experimental treatment, meaning that it has not yet been approved by the US Food and Drug Administration or any worldwide regulatory body. Volixibat is a medicine that lowers circulating bile acid levels that are believed to lead to the itching in patients with PSC. There are no other medicines approved to treat itching in patients with PSC. This is the first time volixibat will be used in patients with PSC. In the first part of this study, called the “Core Study,” the study medicine will be compared with a placebo for a period of 28 weeks. A placebo is a capsule that looks exactly like the study medicine but does not contain any active ingredient. At any time during this part of study, you may be given volixibat or placebo. Whether you receive volixibat or placebo will be decided randomly (by chance, like flipping a coin/rolling a die). In the Core Study, between 50% and 67% of participants will receive volixibat. To make this study fair, for part of the study, you and the study doctor will not be told which medicine or placebo you will receive. This is called “blinding.” In case of an emergency, the study doctor will be able to look up the medicine or placebo you are taking/receiving at any time. In the next part of this study, called the “Open-Label Extension,” all eligible participants who choose to continue will receive volixibat for up to 2 years.More
Type of Participant and Disease Characteristics 3. Have a confirmed PSC diagnosis consistent with the guidelines for the diagnosis and management of PSC published by the AASLD (refer to protocol) a. Large duct PSC: diagnosis confirmed by prior abnormal cholangiography in the absence of alternative etiologies. b. Small duct PSC: diagnosis confirmed by prior consistent liver biochemistry changes and consistent liver histology, as well as evidence of an established diagnosis of concomitant IBD. 4. Qualified pruritus reflected by an average daily Adult ItchRO score ≥4 overall, during the screening period 5. Completion of ≥80% of daily Adult ItchRO assessments during the screening period 6. Participants treated with UDCA will be allowed to enroll if they meet one of the following criteria: a. Total daily dose of ≤23 mg/kg/day and a minimum of 24 weeks of stable treatment prior to screening OR b. A minimum of 12 weeks off treatment before screening if UDCA was recently discontinued 7. Systemic antipruritic therapies (such as but not limited to rifampin/rifampicin, opiate-receptor antagonists, or cholestyramine) are allowed if participants meet one of the following criteria: a. A minimum of 12 weeks of stable treatment prior to screening OR b. A minimum of 4 weeks off treatment before screening if recently discontinued 8. For participants with concomitant IBD: a. Colonoscopy (if participant has a colon) or other appropriate endoscopic procedure performed within 12 to 18 months of Visit 1 confirming no dysplasia or colorectal cancer b. Participants with UC must either be in remission or have mild disease. Remission is defined as a partial Mayo IBD score of <2 with no individual subscore exceeding 1. Mild disease is defined as a partial Mayo IBD score 2–4 with no individual subscore exceeding 2 points at screening through Day 1 c. Participants with CD must be in remission as defined by a Crohn's Disease Activity Index <150 at screening through Day 1 d. Participants with IBD who are on stable treatment of medications as listed in the protocol for at least 12 weeks are allowed
University of Colorado Hospital
Lisa Forman, MD
Protocol Number: 20-2820
More information available at ClinicalTrials.gov: NCT04663308
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