Mirum VLX-301 (Primary Sclerosing Cholangitis)

Purpose of this Study

We are doing this study to find out if an experimental drug called volixibat (the study drug) is a safe and effective option for itching that is caused by primary sclerosing cholangitis (PSC).

Who Can Participate?

Eligibility

Adults ages 18+ who:
  • Are diagnosed with PSC and have itching associated with the disease
  • Have not made any changes to medications that are being used to treat PSC and/or itching for at least 6 weeks
For more information about this study, contact the study team at adele.gaskin@duke.edu.

Age Range

18-110

Sex/Genders

Male (cisgender)
Female (cisgender)
Non-binary or gender fluid
Transgender male
Transgender female
Looking for Healthy Participants
No

What is Involved?

Description

If you choose to join this study, you will get a random assignment (by chance) to 1 of the following 3 study groups:
  • You will take a 20 mg dose of the study drug by mouth twice each day; OR
  • You will take a 80 mg dose of the study drug by mouth twice each day; OR
  • You will take a placebo (inactive substance with no drug in it, "sugar pill") by mouth twice each day
You will take the study drug or placebo for 28 weeks. If you complete the study, you might have the option to enroll in a long-term extension study and continue taking the study drug for at least 2 years. If you got the placebo originally, you will get a 20 mg dose of the study drug in the extension period. Participants who got the study drug originally will continue to take the same dose they were assigned unless the study sponsor determines that one dose is preferable to the other, then everyone in the extension study will take the dose found to be preferable.

Locations

Duke University Hospital

Visit Timing

Weekdays

Compensation

Yes

Spanish Materials Available

No

Study Details

Full Title

A Randomized Double-Blind Placebo-Controlled Study to Evaluate the Efficacy and Safety of Volixibat in the Treatment of Cholestatic Pruritus in Patients with Primary Sclerosing Cholangitis (VISTAS)

Principal Investigator

Julius
Wilder

Protocol Number

PRO00107038

NCT ID

NCT04663308

Phase

II

Enrollment Status

Open to Enrollment
ClinicalTrials.gov