Recruiting NCT05050136
A Study to Evaluate Efficacy and Safety of an Investigational Drug Named Volixibat in Patients With Itching Caused by Primary Biliary Cholangitis
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: Volixibat, Placebo.
- Who it may be relevant to
- Registry conditions: Primary Biliary Cholangitis, PBC. Basic parameters: from 18 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States, Argentina, Belgium, Brazil, Canada +10
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
Unsure about the terms? Read our patient guide →
Official 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 Biliary Cholangitis
Overview
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 Biliary Cholangitis (PBC), and to assess the possible impact on the disease progression of PBC.
Interventions
- Drug Volixibat
Oral capsules, administered twice daily. Volixibat is an Ileal Bile Acid Transporter (IBAT) inhibitor. - Drug Placebo
Capsules matched to study drug without the active pharmaceutical ingredient
Primary outcome measures
- Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire [Time frame: Baseline to week 28]
Secondary outcome measures (8)
- Proportion of participants with itch response using the Adult ItchRO [Time frame: Baseline to week 28]
- Incidence of adverse events [Time frame: Baseline to week 28]
- Changes in alkaline phosphatase [Time frame: Baseline to week 28]
- Changes in total bilirubin [Time frame: Baseline to week 28]
- Changes in serum bile acid levels [Time frame: Baseline to week 28]
- Change in Primary Biliary Cholangitis Quality of Life Measure (PBC-40) [Time frame: Baseline to week 28]
- Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) fatigue questionnaire [Time frame: Baseline to week 28]
- Change in Patient-Reported Outcomes Measurement Information System (PROMIS®) sleep disturbance questionnaire [Time frame: Baseline to week 28]
Eligibility criteria
Inclusion criteria
- Provide signed informed consent at the screening visit as well as comply with all study visits and requirements through the end of the study
- Male or female, age ≥18 years at the screening visit
- Confirmed diagnosis of PBC in line with the AASLD guidelines
- UDCA, anti-pruritic medication and PPAR agonist use will be allowed if meeting additional criteria
- Qualified pruritus associated with PBC as assessed by Adult ItchRO
Exclusion criteria
- Pruritus associated with an etiology other than PBC
- Evidence or clinical suspicion of decompensated cirrhosis or a history of decompensation events
- Current symptomatic cholelithiasis or inflammatory gallbladder disease
- History of small bowel surgery/resection impacting the terminal ileum that may disrupt the enterohepatic circulation.
- Evidence, history, or suspicion of other liver diseases; PBC patients with AIH are not excluded
- History of Liver transplantation
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- Randomized
- Model
- Parallel assignment
- Masking
- Double blind
- Primary purpose
- Treatment
Study locations
United States · 44 centers
- Southern California Research Center — Coronado
- Cedars Sinai Medical Center — Los Angeles
- University of California Los Angeles — Los Angeles
- UC Davis — Sacramento
- University of Colorado — Aurora
- Yale University Digestive Diseases — New Haven
- Covenant Metabolic Specialists — Fort Myers
- UF Hepatology Research at CTRB — Gainesville
- … and 36 more centers
Italy · 10 centers
Center list to be confirmed — check the primary protocol.
Germany · 9 centers
- Klinikum Chemnitz gGmbH — Chemnitz
- … and 8 more centers
Israel · 9 centers
Center list to be confirmed — check the primary protocol.
Canada · 8 centers
- Gordon and Leslie Diamond Health Care Centre, Division of Gastroenterology — Vancouver
- (G.I.R.I.) GI Research Institute Foundation — Vancouver
- McMaster University Medical Center — Hamilton
- University Hospital, LHSC — London
- Toronto General Hospital — Toronto
- McGill University Health Centre — Montreal
- University of Alberta — Edmonton
- CRCHUM (Centre de Recherche du Centre Hospitalier de l'Université de Montréal) — Montreal
Brazil · 7 centers
- Centro de Pesquisa Clínica da Bahiana Saúde — Salvador
- Hospital Das Clínicas Da Ufg — Goiânia
- Centro de Pesquisas Clínicas do HC/UFMG-EBSERH — Belo Horizonte
- Hospital de Clínicas de Porto Alegre — Porto Alegre
- Hospital Universitário Clementino Fraga Filho - Secretaria de Hepatologia — Rio de Janeiro
- Instituto de Educação, Pesquisa e Gestão em Saúde — Rio de Janeiro
- Fundação Oswaldo Ramos — São Paulo
China · 7 centers
- Mengchao Hepatobiliary Hospital of Fujian Medical University — Fuzhou
- Union Hospital Tongji Medical College Huazhong University of Science and Technology — Wuhan
- The First Hospital of Jilin University — Changchun
- The First Affiliated Hospital, Zhejiang University School of Medicine — Hangzhou
- Beijing Youan Hospital, Capital Medical University — Beijing
- Beijing Friendship Hospital, Capital Medical University — Beijing
- Sir Run Run Shaw Hospital -Zhejiang University School of Medicine — Hangzhou
United Kingdom · 7 centers
Center list to be confirmed — check the primary protocol.
France · 6 centers
- CHU Grenoble - Hôpital MICHALLON — Grenoble
- Hopital Claude Huriez-Digestive system disease clinic — Lille
- CHU de NICE- Hospital L'Archet 2 — Nice
- Hospital Saint Antoine — Paris
- Nouvel Hospital Civil — Strasbourg
- Hospital Paul Brousse- APHP — Villejuif
Spain · 6 centers
Center list to be confirmed — check the primary protocol.
Japan · 5 centers
Center list to be confirmed — check the primary protocol.
Argentina · 4 centers
- Hospital de Alta Complejidad En Red El Cruce — San Juan Bautista
- Hospital Provincial Del Centenario — Rosario
- Glenny Corp S.A — CABA
- Clínica de Nefrología, Urología y Enfermedades Cardiovasculares — Santa Fe
Belgium · 3 centers
- Universitair Ziekenhuis Antwerpe UZA — Antwerp
- Cliniques Universitaires Saint-Luc — Brussels
- UZ Gent — Ghent
Netherlands · 3 centers
Center list to be confirmed — check the primary protocol.
Switzerland · 2 centers
Center list to be confirmed — check the primary protocol.
Publications
- Klatzkow H, Bhavsar-Burke I, Pearson M, Wentworth BJ. Primary Biliary Cholangitis in 2025: A New Frontier. Am J Gastroenterol. 2025 Dec 1;120(12):2746-2749. doi: 10.14309/ajg.0000000000003559. Epub 2025 May 29. No abstract available. PMID 40439725
Identifiers
NCT: NCT05050136 · VLX-601