Vidofludimus Calcium for Primary Sclerosing Cholangitis

NCT ID: NCT03722576

Last Updated: 2022-10-17

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 18 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2019-06-17
• Study Completion Date: 2020-06-30

Brief Summary

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:

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.

Conditions

Primary Sclerosing Cholangitis

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Vidofludimus Calcium (VC)

Daily dosing of VC over 6 months

Group Type: EXPERIMENTAL

Vidofludimus calcium

Intervention Type: DRUG

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.

Interventions

Vidofludimus calcium

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.

Intervention Type: DRUG

Other Intervention Names

IMU-838

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Arizona State University

OTHER

Sponsor Role: collaborator

Elizabeth Carey

OTHER

Sponsor Role: lead

Responsible Party

Elizabeth Carey

Principal Investigator

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Elizabeth Carey, MD

Role: PRINCIPAL_INVESTIGATOR

Mayo Clinic

Locations

Mayo Clinic in Arizona

Phoenix, Arizona, United States

Arizona State University

Tempe, Arizona, United States

Mayo Clinic in Rochester

Rochester, Minnesota, United States

Countries

United States

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Related Links

https://www.mayo.edu/research/clinical-trials

Mayo Clinic Clinical Trials

Other Identifiers

IND140679

Identifier Type: -

Identifier Source: org_study_id