Primary Sclerosing CholangitisPhase 2PSCNCT03722576
Vidofludimus Calcium for Primary Sclerosing Cholangitis
A Phase 2 study testing Vidofludimus calcium in people with Primary Sclerosing Cholangitis, run by Elizabeth Carey at 3 sites in United States.
Trial summary
To examine the safety, tolerability, and efficacy of daily dosing with vidofludimus calcium over a 6-month period. 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?
- Vidofludimus calcium
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 3 sitesUnited States
- Study dates
- 17 Jun 2019to 30 Jun 2020
- 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
- Male or female subject age 18-75 years
- Diagnosis of PSC consistent with the guidelines published by the AASLD. All subjects must have an elevated serum ALP of at least 1.5 times upper limit of normal (ULN) at baseline plus cholangiographic evidence of PSC (MRI, endoscopic retrograde cholangiography, or direct cholangiography).
- Indirect bilirubin <1.2 times the ULN
- An ultrasound (or equivalent imaging modality) that excludes biliary obstruction and malignancy within 6 months of study enrollment
- PSC with or without inflammatory bowel disease, such as ulcerative colitis or Crohn's disease
- Must agree to comply with the study protocol and provide informed consent
Who cannot join
- Pregnancy, attempting to become pregnant, or breastfeeding
- Active hepatitis A or B infection
- Active hepatitis C infection (antibody positive); patients with a history of hepatitis C infection will be eligible for this study if they have undetectable levels of HCV RNA
- HIV/AIDS (per medical record or HIVAb/HIA antigen), tuberculosis, or positive interferon-gamma assay (IGRAs) for Mycobacterium tuberculosis
- Other cholestatic liver disease such as primary biliary cholangitis and cholestatic diseases of pregnancy
- Metabolic liver diseases such as Wilson's disease, Gilbert's syndrome or hemochromatosis
Show all 25Show fewer
- Serum uric acid levels at screening >1.2 ULN
- Inherited diseases of the liver such as α-1 antitrypsin deficiency
- Immunoglobulin G4-related cholangitis
- PSC with concomitant autoimmune hepatitis (AIH) and/or primary biliary cholangitis
- Secondary sclerosing cholangitis (SSC)
- Active acute ascending cholangitis requiring antibiotics
- CCA (malignant biliary stricture, neoplasm, and cytology/histopathology or positive fluorescence in situ hybridization (FISH) consistent with adenocarcinoma of the bile duct)
- A liver biopsy, if one has been previously obtained, which showed non-alcoholic steatohepatitis (NASH). Patients with suspected fatty liver by imaging will not be excluded.
- Presence of complications of advanced PSC such as hepatic encephalopathy, portal hypertension, hepato-renal syndrome, and hepato-pulmonary syndrome
- History of liver transplantation, anticipated need for liver transplantation within 12 months from randomization, a Model of End-stage Liver Disease (MELD) score of ≥15, or a Child Pugh score >6
- Ongoing alcohol abuse (>4 drinks per day for men, and >2 drinks per day for women)
- Moderate-to-severe renal impairment with a calculated creatinine clearance of <60mL/min
- Any other conditions or abnormalities that, in the opinion of the investigator, may compromise the safety of the subject or interfere with the subject participating in or completing the study
- Evidence of, or treatment for, C. difficile infection within 30 days before the initiation of the study drug
- Evidence of active C. difficile infection during the screening phase confirmed by a positive C. difficile toxin B
- Subjects who have been treated for intestinal pathogens other than C. difficile infection within 30 days prior to study drug initiation
- Received or plan to receive live vaccine within 30 days prior to, and through the end of the study
- Use of methotrexate at dose ≥17.5mg/week
- Rosuvastatin exceeding 10 mg daily
Trial locations and contacts
3 sites in 1 country.
United States3 sites
Mayo Clinic in Arizona
Phoenix, Arizona, 85259
Mayo Clinic in Rochester
Rochester, Minnesota, 55905
Arizona State University
Tempe, Arizona, 85281
About this study
Official title: Investigation of the Activity of Vidofludimus Calcium, a Novel, Orally Available, Small Molecule Inhibitor of Dihydroorotate Dehydrogenase, as a Treatment for Primary Sclerosing Cholangitis (PSC)
To examine the safety, tolerability, and efficacy of daily dosing with vidofludimus calcium over a 6-month period.
Read the detailed description
Investigators will assess the following:
- Changes on serum alkaline phosphatase levels at 3 \& 6 months.
- Changes in other liver biochemistries at 3 \& 6 months.
- Changes in IL-17 \&IFNγ levels at 6 weeks and 6 months.
- Conditions
- Primary Sclerosing Cholangitis
- Being tested
- Vidofludimus calcium
- Medical subject headings
- Cholangitis, Sclerosing
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Vidofludimus calciumDrug
Also called IMU-838
During the 6-month treatment period, subjects will receive 30 mg VC orally once daily. This will be preceded by a lead-in dosing period where subjects will receive 15 mg VC once daily for 1 week.
| Study group | Role | What they receive |
|---|---|---|
| Vidofludimus Calcium (VC) | Experimental | Daily dosing of VC over 6 months Receives: Drug: Vidofludimus calcium |
What is being measured
The primary outcome is the main question the study is designed to answer.
Subjects Who Experience a Positive Outcome as Measured by Combination of Serum Alkaline Phosphatase (ALP) and Aspartate Aminotransferase (AST) Levels.
The number of subjects who have both an ALP reduction from baseline to week 24 that is greater or equal to 25% and their AST increase from baseline is less than or equal to 33% at week 24. ALP measured as international units per liter (IU/L). AST measured as international units per liter (IU/L).
Measured: Baseline to 24 weeks
Secondary outcomes (4)
Abnormal Aspartate Aminotransferase (AST)
Number of subjects with abnormal (not within normal range) AST levels. AST is an enzyme found in high amounts in liver, heart, and muscle cells. This test is mainly done along with other tests such as alkaline phosphatase and bilirubin to diagnose and monitor liver disease. This test evaluates hepatocyte integrity, as serum levels of this enzyme rise in response to a variety of forms of injury to hepatic cells. The normal range is 5 to 40 U/L . Units: U/L
Measured: Baseline to 24 weeks
Abnormal Alanine Aminotransferase (ALT)
Number of subjects with abnormal (not within normal range) ALT levels. An enzyme normally present in liver and heart cells that is released into the bloodstream when the liver or heart is damaged. The blood ALT levels are elevated with liver damage (for example, from viral hepatitis) or with an insult to the heart (for example, from a heart attack). The normal range is 7 to 56 U/L. Units: U/L
Measured: 24 weeks
Abnormal Total Bilirubin
Number of subjects with abnormal (not within normal range) Total Bilirubin levels. Bilirubin is a yellowish pigment found in bile, a fluid made by the liver. A small amount of older red blood cells are replaced by new blood cells every day. Bilirubin is left after these older blood cells are removed. The liver helps break down bilirubin so that it can be removed from the body in the stool. The normal range for total bilirubin is 0.3 to 1.2 mg/dL Units: mg/dL
Measured: 24 weeks
Abnormal Direct Bilirubin
Number of subjects with abnormal (not within normal range) direct bilirubin levels. In the liver, bilirubin is changed into a form that your body can get rid of. This is called conjugated bilirubin or direct bilirubin. This bilirubin travels from the liver into the small intestine. A very small amount passes into your kidneys and is excreted in your urine. Normal range for direct bilirubin is 0.3 and 1.2 milligrams per deciliter (mg/dL). Units: mg/dL
Measured: 24 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted29 Oct 2018
- Study start17 Jun 2019
- Primary completion30 Jun 2020
- Study completion30 Jun 2020
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- 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 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Elizabeth Carey
- Sponsor type
- University, hospital or other
- Collaborators
- Arizona State University
- Responsible party
- Sponsor-investigator, Elizabeth Carey, Mayo Clinic
- Investigators
- Elizabeth Carey, MD, Principal investigator, Mayo Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03722576
- Sponsor's protocol ID
- IND140679
- Acronym
- PSC
- Status last verified
- Oct 2022
- First submitted
- 25 Oct 2018
- Last updated
- 17 Oct 2022
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Investigation of the Activity of Vidofludimus Calcium, a Novel, Orally Available, Small Molecule Inhibitor of Dihydroorotate Dehydrogenase, as a Treatment for Primary Sclerosing Cholangitis (PSC)
- ClinicalTrials.gov ID
- NCT03722576
- Acronym / sponsor ID
- PSC / IND140679
- Conditions
- Primary Sclerosing Cholangitis
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Elizabeth CareyUniversity, hospital or other
- Collaborators
- Arizona State University
- Study start
- 17 Jun 2019 (actual)
- Primary completion
- 30 Jun 2020 (actual)
- Study completion
- 30 Jun 2020 (actual)
- First posted
- 29 Oct 2018 (actual)
- Results
- Posted 2 Jun 2021
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
To examine the safety, tolerability, and efficacy of daily dosing with vidofludimus calcium over a 6-month period.
Detailed description
Investigators will assess the following:
- Changes on serum alkaline phosphatase levels at 3 \& 6 months.
- Changes in other liver biochemistries at 3 \& 6 months.
- Changes in IL-17 \&IFNγ levels at 6 weeks and 6 months.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Vidofludimus Calcium (VC) | Experimental | Drug: Vidofludimus calciumDaily dosing of VC over 6 months |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Male or female subject age 18-75 years
- Diagnosis of PSC consistent with the guidelines published by the AASLD. All subjects must have an elevated serum ALP of at least 1.5 times upper limit of normal (ULN) at baseline plus cholangiographic evidence of PSC (MRI, endoscopic retrograde cholangiography, or direct cholangiography).
- Indirect bilirubin <1.2 times the ULN
- An ultrasound (or equivalent imaging modality) that excludes biliary obstruction and malignancy within 6 months of study enrollment
- PSC with or without inflammatory bowel disease, such as ulcerative colitis or Crohn's disease
Key exclusion criteria (25 listed)
- Pregnancy, attempting to become pregnant, or breastfeeding
- Active hepatitis A or B infection
- Active hepatitis C infection (antibody positive); patients with a history of hepatitis C infection will be eligible for this study if they have undetectable levels of HCV RNA
- HIV/AIDS (per medical record or HIVAb/HIA antigen), tuberculosis, or positive interferon-gamma assay (IGRAs) for Mycobacterium tuberculosis
- Other cholestatic liver disease such as primary biliary cholangitis and cholestatic diseases of pregnancy
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 |
|---|---|---|
| Vidofludimus calciumAlso: IMU-838During the 6-month treatment period, subjects will receive 30 mg VC orally once daily. This will be preceded by a lead-in dosing period where subjects will receive 15 mg VC once daily for 1 week. | Drug | Vidofludimus Calcium (VC) |
Primary outcome
Subjects Who Experience a Positive Outcome as Measured by Combination of Serum Alkaline Phosphatase (ALP) and Aspartate Aminotransferase (AST) Levels.
Time frame: Baseline to 24 weeks
The number of subjects who have both an ALP reduction from baseline to week 24 that is greater or equal to 25% and their AST increase from baseline is less than or equal to 33% at week 24. ALP measured as international units per liter (IU/L). AST measured as international units per liter (IU/L).
4 secondary outcomes
Abnormal Aspartate Aminotransferase (AST)
Time frame: Baseline to 24 weeks
Abnormal Alanine Aminotransferase (ALT)
Time frame: 24 weeks
Abnormal Total Bilirubin
Time frame: 24 weeks
Abnormal Direct Bilirubin
Time frame: 24 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Oct 2022.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Mayo Clinic Clinical TrialsLink provided by the sponsor.
- 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.
Elizabeth Carey, MD principal investigator
Mayo Clinic
3 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Vidofludimus calcium in people with Primary Sclerosing Cholangitis, run by Elizabeth Carey at 3 sites in United States.
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 25 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: finished, as of the sponsor's last update on 17 Oct 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 3 sites in United States. 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 2 Jun 2021). 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?
Subjects Who Experience a Positive Outcome as Measured by Combination of Serum Alkaline Phosphatase (ALP) and Aspartate Aminotransferase (AST) Levels. (time frame: Baseline to 24 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 17 Oct 2022.