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

Purpose

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.

Condition

  • Primary Sclerosing Cholangitis

Eligibility

Eligible Ages
Over 12 Years
Eligible Genders
All
Accepts Healthy Volunteers
No

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

Study Design

Phase
Phase 2
Study Type
Interventional
Allocation
Randomized
Intervention Model
Parallel Assignment
Primary Purpose
Treatment
Masking
Double (Participant, Investigator)

Arm Groups

ArmDescriptionAssigned Intervention
Experimental
Part 1 Arm 1: Volixibat 20mg 		Participants randomized to this arm will receive volixibat 20mg twice daily.
  • Drug: Volixibat
    Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.
    Other names:
    • SHP626
Experimental
Part 1 Arm 2: Volixibat 80mg 		Participants randomized to this arm will receive volixibat 80mg twice daily.
  • Drug: Volixibat
    Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.
    Other names:
    • SHP626
Placebo Comparator
Part 1 Arm 3: Placebo 		Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
  • Drug: Placebo
    Capsules matched to study drug minus active substance
Experimental
Part 2 Arm 1: Volixibat Selected Dose 20mg 		Participants randomized to this arm will receive volixibat 20mg twice daily.
  • Drug: Volixibat
    Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor.
    Other names:
    • SHP626
Placebo Comparator
Part 2 Arm 2: Placebo 		Participants in this arm will receive capsules matched to the study drug minus the active volixibat substance, twice daily.
  • Drug: Placebo
    Capsules matched to study drug minus active substance

Recruiting Locations

MedStar Georgetown University Hospital
Washington, District of Columbia 20007
Contact:
Albert Lee
202-877-2383
Albert.c.lee@medstar.net

More Details

Status
Recruiting
Sponsor
Mirum Pharmaceuticals, Inc.

Study Contact

Clinical Trials Mirum
+16506674085
Clinicaltrials@mirumpharma.com