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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Mirum Pharmaceuticals Inc.

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Digestive System Diseases [C06]

Trial duration

10 Jun 2022 → ongoing

Decision date (initial)

2024-03-19

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

External identifiers

EU CT number

2023-505764-11-00

EudraCT number

2020-003027-41

ClinicalTrials.gov

NCT04663308

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Pharmacokinetic, Therapy, Efficacy, Pharmacodynamic, Safety

To evaluate the efficacy of volixibat versus placebo for the treatment of pruritus (as measured by the change in the Adult ItchRO tool) in participants with PSC.

Secondary objectives 4

1. To evaluate the efficacy of volixibat versus placebo on the improvement of markers of cholestasis
2. To evaluate safety and tolerability of volixibat versus placebo in participants with PSC-associated pruritus.
3. To evaluate the efficacy of volixibat versus placebo for the treatment of pruritus on the basis of proportion of participants achieving a reduction of ≥2 points in the weekly averaged daily Adult ItchRO score from baseline through Week 24
4. To evaluate quality of life in participants with PSC-associated pruritus treated with volixibat versus placebo.

Conditions and MedDRA coding

Pruritus associated with Primary Sclerosing Cholangitis (PSC)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 6

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. Age ≥12 years for eligible regions; otherwise ≥18 years
3. Have a confirmed PSC diagnosis prior to Visit 2 consistent with the guidelines for the diagnosis and management of PSC published by the AASLD
4. Average daily Adult ItchRO score ≥1 during the 2 consecutive weeks leading to Visit 2
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 7

1. Chronic pruritus of other etiology other than that may interfere with the severity assessment of PSC-related pruritus
2. Evidence or clinical suspicion of decompensated cirrhosis, or a history of decompensation events.
3. Bile duct stent or percutaneous bile duct drain placement, or balloon dilatation procedure of a stricture within 12 weeks of Screening
4. History of ileostomy or small bowel surgery/resection or other surgeries that may have disrupted the enterohepatic circulation.
5. Evidence, history, or suspicion of other liver and GI-related diseases and malignancies.
6. History of liver transplantation
7. 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

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Mean change in the Adult ItchRO comparing baseline with the average of the weekly averaged worst daily itch scores.

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 2

Placebo 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Mirum Pharmaceuticals Inc.

Sponsor organisation

Mirum Pharmaceuticals Inc.

Address

950 Tower Lane Suite 300

City

Foster City

Postcode

94404-2171

Country

United States

Scientific contact point

Organisation

Mirum Pharmaceuticals Inc.

Contact name

Mirum Clinical Lead

Public contact point

Organisation

Mirum Pharmaceuticals Inc.

Contact name

Mirum Clinical Lead

Third parties 16

Locations

6 EU/EEA countries · 27 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 214 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

14 submissions — initial application plus substantial / non-substantial modifications