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Primary Sclerosing CholangitisPhase 2NCT05642468

Safety and Tolerability of A3907 in Primary Sclerosing Cholangitis

A Phase 2 study testing Ritivixibat in people with Primary Sclerosing Cholangitis, run by Albireo, an Ipsen Company at 8 sites in 4 countries.

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
Search all trials

Trial summary

This study will test a drug called A3907 to see how safe and tolerated it is for treating people with Primary Sclerosing Cholangitis (PSC). 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?
Ritivixibat
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
18 participants
Where
8 sitesin 4 countries
Study dates
9 Jan 2023to 10 Jul 2025
Phase
Phase 2
Are there results?
Yes, posted in the registrySummary in the doctors' view

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

  • Adults between 18 and 75 years of age (inclusive)
  • Clinical diagnosis of large-duct PSC as evidenced by chronic cholestasis with evidence of more than 6 months duration with either a consistent magnetic resonance cholangiopancreatography (MRCP) or endoscopic retrograde cholangiopancreatography (ERCP) showing sclerosing cholangitis. and historical evidence of elevated alkaline phosphatase (ALP).
  • Willing to sign informed consent.
  • Women of childbearing potential (WOCBP) and males with female partners of childbearing potential must agree to use contraception. Women of nonchildbearing potential (WONCBP) have a confirmatory follicle-stimulating hormone [FSH] level ≥ 40 mIU/mL
  • Alkaline phosphatase Phosphatase (ALP) value > 1.5 × upper limit of normal (ULN) but ≤ 10 × ULNULN at Visit 1 (Screening Period). Before starting 12 weeks treatment variability of < 30% between ALP values at Visit 1 and Visit 2 must be confirmed. If variability is > 30 % a third ALP value may be obtained. If the third ALP value meets >1.5 × ULN but ≤ 10 × ULN the patient can start the 12-week treatment period.
  • Arms 1-3 Only: Total bilirubin < 1.5 × ULN (unless due to Gilberts Syndrome or hemolysis) and normal direct bilirubin.
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  • Aspartate aminotransferase (AST) and alanine aminotransferase (ALT) ≤ 5 × ULN
  • Serum bile acid level > ULN
  • Arms 1 - 3 Only: An MRCP or equivalent imaging modality performed within 6 months before the Screening Period that is consistent with PSC without a clinically relevant stricture.
  • Arm 4 Only: An MRCP or equivalent imaging modality performed within 6 months before the Screening. Period that is consistent with PSC with a clinically relevant stricture, or clinically relevant bile duct obstruction (see Inclusion # 12 for additional information).
  • Use of ursodeoxycholic acid (UDCA) with a total daily dose ≤ 23 mg/kg/day, or bile acid-binding resins are permitted, with a minimum of 3 months of stable treatment prior to the Screening Period, and expected to remain on a stable dose through the 12-week treatment period; or a minimum of 3 months off UDCA prior to the Screening Period if UDCA was recently discontinued.
  • If a patient has inflammatory bowel disease (IBD) with a minimum disease duration of 4 weeks, this diagnosis should be documented. Inflammatory bowel disease should be in clinical remission or mildly active according to Crohn's Disease Activity Index (CDAI), partial Mayo score for Crohn's Disease (CD) and ulcerative colitis (UC), respectively (i.e. patients with CDAI score < 220 and Mayo score < 5, respectively). Patients with IBD should have had a colonoscopy performed within one year prior to the Screening Period with results showing no evidence of dysplasia or cancer
  • Clinically stable for at least 3 months prior to the Screening Period.
  • Arm 4 Only: One stable clinically relevant biliary stricture of at least 4 weeks duration on contrast-enhanced MRI/MRCP with > 75% reduction of duct diameter in the common bile duct or hepatic duct without suspicion of cholangiocarcinoma (further established by imaging and stable CA 19-9 below ULN repeated twice over 1 month), or cholelithiasis. Subjects may have signs or symptoms of worsening obstructive cholestasis (increasing jaundice, nausea, anorexia, steatorrhea and worsening or new onset pruritus), deterioration of liver function (i.e. decreasing platelet count, increasing international normalized ratio [INR]) and/could be listed for liver transplantation due to their clinically relevant biliary stricture.
  • Arm 4 Only: MELD Score < 35

Who cannot join

  • Presence of documented secondary sclerosing cholangitis, small duct PSC, known or suspected overlapping clinical and histologic diagnosis of autoimmune hepatitis or other causes of chronic liver disease
  • Arm 1-3 Only: Biliary intervention within 3 months prior to study enrollment or planned.
  • Arm 4 Only: Planned Biliary intervention between the Screening Period and baseline.
  • Presence of alternative causes of chronic liver disease, including alcohol-associated liver disease, nonalcoholic steatohepatitis, primary biliary cholangitis, autoimmune hepatitis, or active hepatitis B or C.
  • IBD with uncontrolled moderate to severe activity and/or on treatment with any immunosuppressive, immunomodulator, or biologic agent for treatment of IBD (i.e. azathioprine, 6 mercaptopurine, tacrolimus, methotrexate, infliximab, adalimumab, golimumab, vedolizumab, ustekinumab, tofacitinib, ozanimod). Treatment with corticosteroids (including budesonide, budesonide MMX and beclomethasone) in the previous 4 weeks.
  • History of human immunodeficiency virus infection or any other known relevant infection (e.g. tuberculosis).
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  • History of ileectomy, colostomy or colectomy.
  • History of malignancy, including hepatocellular carcinoma and cholangiocarcinoma within the past 10 years, except for basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated.
  • Alpha-fetoprotein (AFP) > 20 ng/mL (at the Screening Visit) with 4-phase liver CT or MRI suggesting presence of liver cancer.
  • History of transplants, including liver transplantation, or currently on active transplantation list (Arms 1 3). Arm 4 may be on an active liver transplantation list.
  • Current or a history of hepatic decompensation events including, but not limited to ascites, encephalopathy, or history of esophageal variceal bleeding.
  • Known or suspected overlapping clinical and histologic diagnosis of autoimmune hepatitis.
  • Small duct PSC (evidence of PSC on historical liver histology, with normal bile ducts on cholangiography) without large duct PSC.
  • Liver cirrhosis as assessed by any of the following:
  • historical liver histology.
  • liver stiffness measurement, assessed by FibroScan (FibroScan value > 14.4 kPa), in addition to clinical assessment and biochemical markers at the discretion of the investigator.
  • signs and symptoms of hepatic decompensation (including, but not limited to, jaundice, ascites, variceal haemorrhage, and/or hepatic encephalopathy).
  • History of bacterial cholangitis within 60 days prior to the Screening Period, or if the patient is on antibiotics for prophylaxis of recurrent cholangitis.
  • Females who are pregnant, lactating, or breast feeding.
  • History of alcohol or substance abuse in the previous 2 years. Patients must agree to refrain from illicit drug (including marijuana) and alcohol use during the study.
  • Hypersensitivity to investigational medicinal product (A3907) and its excipients.
  • Presence of any contraindication for undergoing MRCP (e.g. pacemaker).
  • Any other condition or abnormality which, in the opinion of the investigator (or designee), may compromise the safety of the patient or interfere with the patient participating in, or completing the study.
  • Administration of medications that slow gastrointestinal motility.
  • Treatment with rifampicin.
  • Exposure to oral drugs that are strong inhibitors or inducers of CYP3A4 enzymes (e.g. grapefruit juice, ritonavir, itraconazole, ketoconazole, troleandomycin, rifampin, St John's wort, etc.) within 14 days prior to the Screening Period, or 5 half-lives of the drug, whichever is longer.
  • Exposure to oral drugs that are substrates of CYP3A4 enzymes (e.g. codeine, ciclosporin [cyclosporin], diazepam, etc.) during the study.
  • Treatment with vitamin D or fibrates, unless patient is on a stable dose ≥ 6 months prior to baseline.
  • Exposure to an investigational drug, biologic agent, or medical device within 30 days prior to the Screening Period, or 5 half-lives of the study agent, whichever is longer.
  • Platelet count < 150 000/mm3
  • Albumin level < 3.0 g/dL
  • INR > 1.3 (the patient may be treated with vitamin K intravenously, and if INR is ≤ 1.3 at resampling, the patient may be enrolled).
  • Medical conditions that may cause non-hepatic increases in ALP (e.g. Paget's disease). A GGT or ALP isoenzymes should be obtained for confirmation of biliary origin;
  • Glomerular filtration rate [GFR] < 60 mL/min/1.73 m2

Trial locations and contacts

8 sites in 4 countries.

8 sites in 4 countries

Italy3 sites

  • ASST Grande Ospedale Metropolitano Niguarda

    Milan

  • ASST di Monza - Azienda Ospedaliera San Gerardo

    Monza, 20900

  • Azienda Ospedale Università Padova

    Padova, 35128

Other trials in Italy

Poland3 sites

  • Centrum Medyczne INTER-MED

    Częstochowa

  • Uniwersyteckie Centrum Kliniczne im. Prof. Kornela Gibinskiego

    Katowice

  • ID Clinic Arkadiusz Pisula

    Mysłowice

Other trials in Poland

France1 site

  • Hopital Saint Antoine

    Paris, 75012

Other trials in France

Spain1 site

  • Hospital Clinic de Barcelona

    Barcelona, 8036

Other trials in Spain

About this study

Official title: An Open Label, Phase 2 Study to Evaluate the Effect of A3907 on Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics in Adults With Primary Sclerosing Cholangitis (PSC)

This study will test a drug called A3907 to see how safe and tolerated it is for treating people with Primary Sclerosing Cholangitis (PSC).

Read the detailed description

This study will test a drug called A3907 to see how safe and tolerated it is for treating people with Primary Sclerosing Cholangitis (PSC). Detailed Description: The primary goal of this study in participants with PSC with and without a Clinically Relevant Stricture (CRS) who are treated with A3907 is to assess the safety and tolerability of A3907 following repeat doses. Secondary goals include evaluation of the pharmacokinetic properties of A3907 (the study of how the body interacts with A3907 for the entire duration of exposure) and changes in safety parameters via laboratory testing such as liver enzymes, bile acid levels and markers of bile acid synthesis

Being tested
Ritivixibat
Keywords
PSC, Primary Sclerosing Cholangitis
Medical subject headings
Cholangitis, Sclerosing

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • RitivixibatDrug

    10mg tablet A3907 administered orally

Study groups in NCT05642468
Study groupRoleWhat they receive
10 mg (Arm 1)Experimental10mg tablet A3907 administered orally once daily for 12 weeks.
Receives: Drug: Ritivixibat
30 mg (Arm 2)Experimental30mg (3x10 mg tablets) A3907 administered orally once daily for 12 weeks.
Receives: Drug: Ritivixibat
30 mg BID (Arm 3)Experimental30mg (3x10 mg tablets) A3907 administered orally Bi-daily for 12 weeks.
Receives: Drug: Ritivixibat
30 mg BID for CRS (Arm 4)Experimental30mg (3x10 mg tablets) A3907 administered orally Bi-daily for 12 weeks.
Receives: Drug: Ritivixibat

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs)

    An adverse event (AE) was any untoward medical occurrence in an enrolled participant regardless of causal relationship with study drug. An SAE was defined as any untoward medical occurrence that, at any dose, resulted in death, was life-threatening, required inpatient hospitalization or prolongation of existing hospitalization, resulted in persistent or significant disability/incapacity, was a congenital anomaly/birth defect or an important medical event. A TEAE was any AE or worsening of an existing disease that occurred after the first dose of study drug and within the 14-day follow-up.

    Measured: From first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days

Secondary outcomes (7)

  1. Maximum Observed Plasma Concentration (Cmax) of A3907

    Blood samples were collected at specified timepoints to assess Cmax of A3907. The pharmacokinetic (PK) analysis was conducted after single dose (Day 0) and repeated dose administration (Day 98).

    Measured: Pre-dose and 0.5, 1, 1.5, 2, 4, 6, 8, 10 hours post-dose on Days 0 and 98

  2. Area Under the Plasma Concentration-time Curve From Time Zero to Time t (Time of Last Quantifiable Plasma Concentration) (AUC0-t) of A3907

    Blood samples were collected at specified timepoints to assess AUC0-t of A3907.

    Measured: Pre-dose and 0.5, 1, 1.5, 2, 4, 6, 8, 10 hours post-dose on Days 0 and 98

  3. Change From Baseline to Week 12 in Serum and Urine Individual and Total Bile Acid Levels

    Blood and urine samples were collected at specified timepoints to assess serum and urine individual and total bile acid levels. Baseline=average of all screening results and results on day of planned single dose if both results were available. Otherwise, baseline=last non-missing assessment prior to study drug administration of planned single dose. CA=Cholic Acid; LCA=lithocholic acid; DCA=deoxycholic acid; CDCA=chenodeoxycholic acid; UDCA= ursodeoxycholic acid; GCA=glycocholic acid; GLCA=glycolithocholic acid; GDCA=glycodeoxycholic acid; GCDCA=glycochenodeoxycholic acid; GUDCA= glycoursodeoxycholic acid; TCA=taurocholic acid; TCDCA=taurochenodeoxycholic acid; TDCA=taurodeoxycholic acid; TLCA=taurolithocholic acid; TUDCA=tauroursodeoxycholic acid; S=sulfate. Calculated total bile acids without all UDCA bile acids is the sum of all individual bile acids excluding all UDCAs (UDCA, GUDCA, TUDCA). Conjugated bile acids include GCA, GLCA, GDCA, GCDCA, GUDCA, TCA, TCDCA, TDCA, TLCA, TUDCA.

    Measured: Baseline (Day 1) and Week 12

  4. Change From Baseline to Week 12 in Liver Biochemical Tests (LBTs): Alanine Aminotransferase (ALT), Aspartate Aminotransferase (AST), Gamma-glutamyl Transferase (GGT) and Alkaline Phosphatase (ALP)

    Blood samples were collected at specified timepoints to assess LBTs. Baseline was defined as the average of all screening results and results on the day of the planned single dose if both results were available. Otherwise, baseline was defined as the last non-missing assessment prior to the study drug administration of planned single dose.

    Measured: Baseline (Day 1) and Week 12

  5. Change From Baseline to Week 12 in Liver Biochemical Tests: Total and Direct Bilirubin Levels

    Blood samples were collected at specified timepoints to assess LBTs. Baseline was defined as the average of all screening results and results on the day of the planned single dose if both results were available. Otherwise, baseline was defined as the last non-missing assessment prior to the study drug administration of planned single dose.

    Measured: Baseline (Day 1) and Week 12

  6. Change From Baseline to Week 12 in 7α-hydroxy-4-cholesten-3-one (C4)

    Blood samples were collected at specified timepoints to assess C4. Baseline was defined as the assessment performed on the day of the planned single dose.

    Measured: Baseline (Day 1) and Week 12

  7. Change From Baseline to Week 12 in Fibroblast Growth Factor 19 (FGF-19)

    Blood samples were collected at specified timepoints to assess FGF-19. Baseline was defined as the assessment performed on the day of the planned single dose.

    Measured: Baseline (Day 1) and Week 12

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted8 Dec 2022
  2. Study start9 Jan 2023
  3. Primary completion10 Jul 2025
  4. Study completion10 Jul 2025

Why it stopped: Due to Sponsor decision, not due to any safety concerns

Resources

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 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
18 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT05642468
Sponsor's protocol ID
A3907-002
Other IDs
2022-500790-14-00
Status last verified
Apr 2026
First submitted
23 Nov 2022
Last updated
4 May 2026
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
Yes: Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants. Any requests should be submitted to www.vivli.org for assessment by an independent scientific review board.

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Ritivixibat in people with Primary Sclerosing Cholangitis, run by Albireo, an Ipsen Company at 8 sites in 4 countries.

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 15 inclusion and 34 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: stopped early, as of the sponsor's last update on 4 May 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 8 sites in Italy, Poland, France, Spain. 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. 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 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

Yes. Results are posted in the registry (first posted 4 May 2026). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Number of Participants With Treatment-emergent Adverse Events (TEAEs) and Treatment-emergent Serious Adverse Events (TESAEs) (time frame: From first dose of study drug (Day 1) up to 14 days post last dose, approximately 112 days). 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 NCT05642468.

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 4 May 2026.

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