Primary Sclerosing CholangitisPhase 3PiSCATINNCT04133792
Effect of Simvastatin on the Prognosis of Primary Primary Sclerosing Cholangitis (PSC)
A Phase 3 study testing Simvastatin 40mg in people with Primary Sclerosing Cholangitis, run by Annika Bergquist at 12 sites in Sweden.
Trial summary
This is a randomized, double-blind, placebo controlled multicenter study. A total of 700 patients will be included. After an updated powercalculation 560 was condidered enough The study will include patients with primary sclerosing cholangitis (PSC) for daily intake of 40 mg simvastatin/placebo for 5 years. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Primary Sclerosing Cholangitis
- What is being tested?
- Simvastatin 40mg
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 571 participants
- Where
- 12 sitesSweden
- Study dates
- 1 Oct 2020to 31 Mar 2030
- Phase
- Phase 3
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- PSC verified by cholangiography or liver biopsy, with or without irritable bowel disease (IBD).
- Men and women between ≥18 years and ≤75 years.
- Written informed consent.
- A magnetic resonance imaging (MRI) or Magnetic resonance cholangiopancreatography (MRCP) performed within 4 months prior to randomization.
- Colonoscopy performed within 24 months prior to randomization, if known IBD.
- For women of childbearing potential efficient contraceptive.
Who cannot join
- Subjects on waiting list for transplantation
- Transplanted subjects
- Previous variceal bleeding
- Previous hepatobiliary malignancy
- Subjects with secondary sclerosing cholangitis
- Intake of any type of statins within 3 months prior to randmization
- Known intolerance to simvastatin.
- Pregnancy or breastfeeding.
Trial locations and contacts
12 sites in 1 country.
12 sites in 1 country
Sweden12 sites
Sahlgrenska Universitetssjukhuset
Gothenburg, Västra Götaland County, 413 45
Sahlgrenska Universitetssjukhuset Östra
Gothenburg, 41685
Karlstads centralsjukhus
Karlstad, 652 30
Skaraborgs sjukhus
Lidköping
Universitetssjukhuset i Linköping
Linköping, Östergötland County, 581 85
Skåne Universitetssjukhus
Malmö, Skåne County, 222 42
Örebro Universitetssjukhus
Örebro, 701 85
Karolinska University Hospital
Stockholm, 141 57
Karolinska University Hospital Solna
Stockholm, 171 64
Danderyds sjukhus
Stockholm, 182 57
Norrlands Universitetssjukhus
Umeå, Västerbotten County, 907 37
Akademiska sjukhuset
Uppsala, 751 85
About this study
Official title: Effect of Simvastatin on the Prognosis of Primary Sclerosing Cholangitis (PSC); A Randomized, Double-blind, Placebo Controlled Multicenter Study
This is a randomized, double-blind, placebo controlled multicenter study.
A total of 700 patients will be included. After an updated powercalculation 560 was condidered enough
The study will include patients with primary sclerosing cholangitis (PSC) for daily intake of 40 mg simvastatin/placebo for 5 years. The aim is to study effect of prognosis of PSC by long term intake of simvastatin. Outcome measures are death, liver transplantation, cholangiocarcinoma or bleeding from esophageal varices.
Subjects will be randomized (1:1) between Simvastatin and placebo.
Read the detailed description
Please see published protocol
Long Term Effect of Simvastatin in Primary Sclerosing Cholangitits: A Placebo-Controlled, Double-Blind, Multicenter Phase III Study (Piscatin)
- Bergquist, H. U. Marschall, E. Nilsson, N. Nyhlin, M. Werner, A. Klein, et al.
British Journal of Gastroenterolgy 2022 Vol. 1 Pages 235-241.
- Conditions
- Primary Sclerosing Cholangitis
- Being tested
- Simvastatin 40mg, Placebo oral tablet
- Medical subject headings
- Cholangitis, Sclerosing
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Simvastatin 40mgDrug
40 mg orally daily for 5 years.
Placebo oral tabletDrug
40 mg orally daily for 5 years.
| Study group | Role | What they receive |
|---|---|---|
| Simvastatin | Experimental | Simvastatin 40 mg administered orally daily for 5 years. Receives: Drug: Simvastatin 40mg |
| Placebo | Placebo comparator | Placebo for Simvastatin 40 mg administered orally daily for 5 years. Receives: Drug: Placebo oral tablet |
What is being measured
The primary outcome is the main question the study is designed to answer.
Overall survival
Overall survival from time to randomization to death from any cause.
Measured: Time from the date of randomization to the date of death, assessed up to 5 years.
Listing for liver transplantation
Date the subject is getting registered on the waiting list for liver transplantation.
Measured: Time from the date of randomization to the date of listing for liver transplantation, assessed up to 5 years.
Time to first varices bleeding
Date of the subject's first varices bleeding according to hospital patient records.
Measured: Time from the date of randomization to the date of the first varices bleeding, assessed up to 5 years.
Time to diagnosis of cholangiocarcinoma, gall bladder cancer, or hepatocellular cancer.
Diagnosis of cancer of bile duct cancer or gall bladder, or hepatocellular cancer according to hospital patient records.
Measured: Time from the date of randomization to cancer diagnosis, assessed up to 5 years.
Secondary outcomes (10)
Effect on serum concentration of alkaline phosphatase (ALP).
Assessment of changes in the serum concentration of alkaline phosphatase.
Measured: Assessed yearly up to 5 years.
Effect on serum concentration of bilirubin
Assessment of changes in the serum concentration of bilirubin.
Measured: Assessed yearly up to 5 years.
Effect on the progress of PSC by liver failure measurement
Assessment of liver failure using Model for End Stage Liver Disease (MELD) Score (biochemical and clinical variables).
Measured: Assessed at every visit except the 3 months visit, up to 5 years.
Effect on the progress of PSC by liver failure measurement.
Assessment of liver failure using Child Pugh Score
Measured: Assessed at every visit except the 3 months visit, up to 5 years.
Effect on the progress of PSC assessed by cholangiography at MRI.
Progress assessed by cholangiography MRI
Measured: Assessed at inclusion and the 60 months visit.
Effect on the progress of PSC assessed by elastography
Assessment of fibrosis stage using elastography.
Measured: Assessed yearly up to 5 years.
Effect on the progress of PSC assessed by clinical symptoms
Assessment of symptoms including itching and bacterial cholangitis that requires treatment, ascites and encephalopathy.
Measured: Assessed yearly up to 5 years.
Effect on the progress of PSC assessed by measurement of biliary dysplasia
Biliary dysplasia from brush samples taken at endoscopic retrograde cholangiopancreatography (ERCP).
Measured: Assessed upon clinical indication, up to 5 years.
Effect on the development of colon cancer or colon dysplasia.
Development of colon cancer and/or colon dysplasia according to hospital patient records.
Measured: Assessed at 60 months.
Effect on the progress of PSC assessed by serum fibrosis markers
Fib-4, ELF (if funded)
Measured: Assessed yearly up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Oct 2019
- Study start1 Oct 2020
- Primary completion31 Mar 2028 (estimated)
- Study completion31 Mar 2030 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary Sclerosing CholangitisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 571 participants (actual)
Sponsor and investigators
- Lead sponsor
- Annika Bergquist
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor-investigator, Annika Bergquist, Karolinska University Hospital
- Investigators
- Annika Bergquist, MD PhD, Principal investigator, Karolinska University Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04133792
- Sponsor's protocol ID
- 2018-000814-39
- Other IDs
- 2018
- Acronym
- PiSCATIN
- Status last verified
- Aug 2025
- First submitted
- 17 Oct 2019
- Last updated
- 5 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Publications
Lindqvist C, Ingre M, Kechagias S, Nilsson E, Molinaro A, Rorsman F, Bergquist A. Dietary Habits of Individuals With Primary Sclerosing Cholangitis-Poor Fat-Soluble Vitamin Intake and Dietary Quality. Liver Int. 2025 Apr;45(4):e16182. doi: 10.1111/liv.16182. Epub 2024 Nov 27.
PubMed 39601354
Study at a glance
From the registry record, last updated by the sponsor on 5 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Effect of Simvastatin on the Prognosis of Primary Sclerosing Cholangitis (PSC); A Randomized, Double-blind, Placebo Controlled Multicenter Study
- ClinicalTrials.gov ID
- NCT04133792
- Acronym / sponsor ID
- PiSCATIN / 2018-000814-39
- Conditions
- Primary Sclerosing Cholangitis
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 571 participants (actual)
- Lead sponsor
- Annika BergquistUniversity, hospital or other
- Study start
- 1 Oct 2020 (actual)
- Primary completion
- 31 Mar 2028 (estimated)
- Study completion
- 31 Mar 2030 (estimated)
- First posted
- 21 Oct 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This is a randomized, double-blind, placebo controlled multicenter study.
A total of 700 patients will be included. After an updated powercalculation 560 was condidered enough
The study will include patients with primary sclerosing cholangitis (PSC) for daily intake of 40 mg simvastatin/placebo for 5 years. The aim is to study effect of prognosis of PSC by long term intake of simvastatin. Outcome measures are death, liver transplantation, cholangiocarcinoma or bleeding from esophageal varices.
Subjects will be randomized (1:1) between Simvastatin and placebo.
Detailed description
Please see published protocol
Long Term Effect of Simvastatin in Primary Sclerosing Cholangitits: A Placebo-Controlled, Double-Blind, Multicenter Phase III Study (Piscatin)
- Bergquist, H. U. Marschall, E. Nilsson, N. Nyhlin, M. Werner, A. Klein, et al.
British Journal of Gastroenterolgy 2022 Vol. 1 Pages 235-241.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Simvastatin | Experimental | Drug: Simvastatin 40mgSimvastatin 40 mg administered orally daily for 5 years. |
| Placebo | Placebo comparator | Drug: Placebo oral tabletPlacebo for Simvastatin 40 mg administered orally daily for 5 years. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- PSC verified by cholangiography or liver biopsy, with or without irritable bowel disease (IBD).
- Men and women between ≥18 years and ≤75 years.
- Written informed consent.
- A magnetic resonance imaging (MRI) or Magnetic resonance cholangiopancreatography (MRCP) performed within 4 months prior to randomization.
- Colonoscopy performed within 24 months prior to randomization, if known IBD.
Key exclusion criteria (8 listed)
- Subjects on waiting list for transplantation
- Transplanted subjects
- Previous variceal bleeding
- Previous hepatobiliary malignancy
- Subjects with secondary sclerosing cholangitis
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Simvastatin 40mg40 mg orally daily for 5 years. | Drug | Simvastatin |
| Placebo oral tablet40 mg orally daily for 5 years. | Drug | Placebo |
Primary outcome
Overall survival
Time frame: Time from the date of randomization to the date of death, assessed up to 5 years.
Overall survival from time to randomization to death from any cause.
Listing for liver transplantation
Time frame: Time from the date of randomization to the date of listing for liver transplantation, assessed up to 5 years.
Date the subject is getting registered on the waiting list for liver transplantation.
Time to first varices bleeding
Time frame: Time from the date of randomization to the date of the first varices bleeding, assessed up to 5 years.
Date of the subject's first varices bleeding according to hospital patient records.
Time to diagnosis of cholangiocarcinoma, gall bladder cancer, or hepatocellular cancer.
Time frame: Time from the date of randomization to cancer diagnosis, assessed up to 5 years.
Diagnosis of cancer of bile duct cancer or gall bladder, or hepatocellular cancer according to hospital patient records.
10 secondary outcomes
Effect on serum concentration of alkaline phosphatase (ALP).
Time frame: Assessed yearly up to 5 years.
Effect on serum concentration of bilirubin
Time frame: Assessed yearly up to 5 years.
Effect on the progress of PSC by liver failure measurement
Time frame: Assessed at every visit except the 3 months visit, up to 5 years.
Effect on the progress of PSC by liver failure measurement.
Time frame: Assessed at every visit except the 3 months visit, up to 5 years.
Effect on the progress of PSC assessed by cholangiography at MRI.
Time frame: Assessed at inclusion and the 60 months visit.
Effect on the progress of PSC assessed by elastography
Time frame: Assessed yearly up to 5 years.
Effect on the progress of PSC assessed by clinical symptoms
Time frame: Assessed yearly up to 5 years.
Effect on the progress of PSC assessed by measurement of biliary dysplasia
Time frame: Assessed upon clinical indication, up to 5 years.
Effect on the development of colon cancer or colon dysplasia.
Time frame: Assessed at 60 months.
Effect on the progress of PSC assessed by serum fibrosis markers
Time frame: Assessed yearly up to 5 years
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Sep 2025.
- Derived from this study: PubMed 39601354Lindqvist C, Ingre M, Kechagias S, Nilsson E, Molinaro A, Rorsman F, Bergquist A. Dietary Habits of Individuals With Primary Sclerosing Cholangitis-Poor Fat-Soluble Vitamin Intake and Dietary Quality. Liver Int. 2025 Apr;45(4):e16182. doi: 10.1111/liv.16182. Epub 2024 Nov 27.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Annika Bergquist, MD PhD principal investigator
Karolinska University Hospital
12 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Simvastatin 40mg in people with Primary Sclerosing Cholangitis, run by Annika Bergquist at 12 sites in Sweden.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 6 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: under way, not looking for participants, as of the sponsor's last update on 5 Sep 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in Sweden. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Overall survival (time frame: Time from the date of randomization to the date of death, assessed up to 5 years.). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT04133792, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Sep 2025.