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Vidofludimus Calcium for Primary Sclerosing Cholangitis (PSC)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT03722576
Recruitment Status : Completed
First Posted : October 29, 2018
Last Update Posted : November 25, 2020
Arizona State University
Information provided by (Responsible Party):
Elizabeth Carey, Mayo Clinic

Brief Summary:
To examine the safety, tolerability, and efficacy of daily dosing with vidofludimus calcium over a 6-month period.

Condition or disease Intervention/treatment Phase
Primary Sclerosing Cholangitis Drug: Vidofludimus calcium Phase 2

Detailed Description:

Investigators will assess the following:

  1. Changes on serum alkaline phosphatase levels at 3 & 6 months.
  2. Changes in other liver biochemistries at 3 & 6 months.
  3. Changes in IL-17 &IFNγ levels at 6 weeks and 6 months.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 14 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
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)
Actual Study Start Date : June 17, 2019
Actual Primary Completion Date : June 30, 2020
Actual Study Completion Date : June 30, 2020

Arm Intervention/treatment
Experimental: Vidofludimus Calcium
Active drug
Drug: Vidofludimus calcium
30 mg VC orally once daily
Other Name: IMU-838

Primary Outcome Measures :
  1. Changes on serum alkaline phosphatase levels at 3 and 6 months [ Time Frame: 3 - 6 months ]
    Determine if VC reduces serum alkaline phosphatase (ALP) in adults with PSC by comparing changes in levels of serum ALP after 3 and 6 months of VC treatment baseline levels (month 0).

Secondary Outcome Measures :
  1. Changes in other liver biochemistries levels at 3 and 6 months [ Time Frame: 3 - 6 months ]
    Compare other liver biochemistries at 3 and 6 months of VC treatment to baseline. Other biochemistries include: bilirubin, total & direct aspartate and alanine transaminases (AST & ALT).

  2. Changes in IL-17 and IFNγ levels at 6 weeks and months [ Time Frame: 6 weeks - 6 months ]
    Assess proinflammatory cytokine profile (IL-17 and IFNγ) at baseline, 6 weeks and 6 months

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Male or female subject age 18-75 years
  2. 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).
  3. Indirect bilirubin <1.2 times the ULN
  4. An ultrasound (or equivalent imaging modality) that excludes biliary obstruction and malignancy within 6 months of study enrollment
  5. PSC with or without inflammatory bowel disease, such as ulcerative colitis or Crohn's disease
  6. Must agree to comply with the study protocol and provide informed consent

Exclusion Criteria:

  1. Pregnancy, attempting to become pregnant, or breastfeeding
  2. Active hepatitis A or B infection
  3. 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
  4. HIV/AIDS (per medical record or HIVAb/HIA antigen), tuberculosis, or positive interferon-gamma assay (IGRAs) for Mycobacterium tuberculosis
  5. Other cholestatic liver disease such as primary biliary cholangitis and cholestatic diseases of pregnancy
  6. Metabolic liver diseases such as Wilson's disease, Gilbert's syndrome or hemochromatosis
  7. Serum uric acid levels at screening >1.2 ULN
  8. Inherited diseases of the liver such as α-1 antitrypsin deficiency
  9. Immunoglobulin G4-related cholangitis
  10. PSC with concomitant autoimmune hepatitis (AIH) and/or primary biliary cholangitis
  11. Secondary sclerosing cholangitis (SSC)
  12. Active acute ascending cholangitis requiring antibiotics
  13. CCA (malignant biliary stricture, neoplasm, and cytology/histopathology or positive fluorescence in situ hybridization (FISH) consistent with adenocarcinoma of the bile duct)
  14. 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.
  15. Presence of complications of advanced PSC such as hepatic encephalopathy, portal hypertension, hepato-renal syndrome, and hepato-pulmonary syndrome
  16. 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
  17. Ongoing alcohol abuse (>4 drinks per day for men, and >2 drinks per day for women)
  18. Moderate-to-severe renal impairment with a calculated creatinine clearance of <60mL/min
  19. 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
  20. Evidence of, or treatment for, C. difficile infection within 30 days before the initiation of the study drug
  21. Evidence of active C. difficile infection during the screening phase confirmed by a positive C. difficile toxin B
  22. Subjects who have been treated for intestinal pathogens other than C. difficile infection within 30 days prior to study drug initiation
  23. Received or plan to receive live vaccine within 30 days prior to, and through the end of the study
  24. Use of methotrexate at dose ≥17.5mg/week
  25. Rosuvastatin exceeding 10 mg daily

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT03722576

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United States, Arizona
Mayo Clinic in Arizona
Phoenix, Arizona, United States, 85259
Arizona State University
Tempe, Arizona, United States, 85281
United States, Minnesota
Mayo Clinic in Rochester
Rochester, Minnesota, United States, 55905
Sponsors and Collaborators
Elizabeth Carey
Arizona State University
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Principal Investigator: Elizabeth Carey, MD Mayo Clinic
Additional Information:
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Responsible Party: Elizabeth Carey, Principal Investigator, Mayo Clinic Identifier: NCT03722576    
Other Study ID Numbers: IND140679
First Posted: October 29, 2018    Key Record Dates
Last Update Posted: November 25, 2020
Last Verified: November 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Cholangitis, Sclerosing
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Calcium-Regulating Hormones and Agents
Physiological Effects of Drugs