A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Sclerosing Cholangitis (PSC) (VISTAS)

Mirum Pharmaceuticals

Status and phase: Enrolling; Phase 2

Conditions: Primary Sclerosing Cholangitis

Treatments: Drug: Placebo; Drug: Volixibat

Study type: Interventional

Funder types: Industry

Identifiers

NCT04663308

2020-003027-41 (EudraCT Number)

VLX-301

2023-505764-11-00 (Other Identifier)

Details and patient eligibility

About

The purpose of this clinical research study is to learn more about the use of the study medicine, volixibat, for the treatment of pruritus (itching) associated with Primary Sclerosing Cholangitis (PSC), and to assess the possible impact on the disease progression of PSC.

Enrollment: 200 estimated patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Provide freely signed informed consent and assent (as applicable) and be willing to comply with all study visits and requirements through end of study, including the follow-up period.
2. Subjects aged ≥12 years for eligible regions; otherwise ≥18 years
3. Confirmed diagnosis of large duct or small duct PSC based on American Association for the Study of Liver Disease (AASLD) guidelines.
4. Pruritus associated with PSC as assessed by Adult ItchRO.
5. Ursodeoxycholic acid (UDCA) and anti-pruritic medication use will be allowed if meeting additional criteria.
6. Concomitant Inflammatory Bowel Disease (IBD) is allowed if meeting additional criteria.

Exclusion criteria

1. Pruritus associated with an etiology other than PSC
2. Evidence or clinical suspicion of decompensated cirrhosis, or a history of decompensation events
3. History of ileostomy or small bowel surgery/resection or other surgeries that may have disrupted the enterohepatic circulation
4. Evidence, history, or suspicion of other liver disease; PSC patients with AIH are not excluded.
5. Bile duct stent or percutaneous bile duct drain placement, or balloon dilatation procedure of a stricture within 12 weeks of Screening
6. Exceeding pre-defined biochemical values for alanine aminotransferase/aspartate aminotransferase (ALT/AST), estimated glomerular filtration rate (eGFR),serum creatinine (sCr), platelet count, international normalized ratio (INR) and total bilirubin
7. History of liver transplantation

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Double Blind

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200 participants in 5 patient groups, including a placebo group

Part 1 Arm 1: Volixibat 20mg

Experimental group

Description:

Participants randomized to this arm will receive volixibat 20mg twice daily.

Treatment:

Drug: Volixibat

Part 1 Arm 2: Volixibat 80mg

Experimental group

Description:

Participants randomized to this arm will receive volixibat 80mg twice daily.

Treatment:

Drug: Volixibat

Part 1 Arm 3: Placebo

Placebo Comparator group

Description:

Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.

Treatment:

Drug: Placebo

Part 2 Arm 1: Volixibat Selected Dose 20mg

Experimental group

Description:

Participants randomized to this arm will receive volixibat 20mg twice daily.

Treatment:

Drug: Volixibat

Part 2 Arm 2: Placebo

Placebo Comparator group

Description:

Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.

Treatment:

Drug: Placebo