Vidofludimus Calcium for Primary Sclerosing Cholangitis

NCT03722576 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: IND140679
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 3

Brief Summary

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

Conditions

Primary Sclerosing Cholangitis

Outcome Measures

Primary Outcomes (1)

• 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).

  Baseline to 24 weeks

Secondary Outcomes (4)

• Abnormal Aspartate Aminotransferase (AST)

  Baseline to 24 weeks

• Abnormal Alanine Aminotransferase (ALT)

  24 weeks

• Abnormal Total Bilirubin

  24 weeks

• Abnormal Direct Bilirubin

  24 weeks

Study Arms (1)

Vidofludimus Calcium (VC)

EXPERIMENTAL

Daily dosing of VC over 6 months

Drug: Vidofludimus calcium

Interventions

Vidofludimus calcium 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.

Also known as: IMU-838

Vidofludimus Calcium (VC)

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Elizabeth Carey: lead
• Arizona State University: collaborator

Study Sites (3)

Mayo Clinic in Arizona

Phoenix, Arizona, 85259, United States

Location

Arizona State University

Tempe, Arizona, 85281, United States

Location

Mayo Clinic in Rochester

Rochester, Minnesota, 55905, United States

Location

Related Links

• Mayo Clinic Clinical Trials

MeSH Terms

Conditions

Cholangitis, Sclerosing

Condition Hierarchy (Ancestors)

Cholangitis; Bile Duct Diseases; Biliary Tract Diseases; Digestive System Diseases

Results Point of Contact

• Title: Elizabeth J. Carey, M.D.
• Organization: Mayo Clinic

Carey.Elizabeth@mayo.edu

480-342-1094

Study Officials

• Elizabeth Carey, MD

  Mayo Clinic

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Principal Investigator

Study Record Dates

• First Submitted: October 25, 2018
• First Posted: October 29, 2018
• Study Start: June 17, 2019
• Primary Completion: June 30, 2020
• Study Completion: June 30, 2020
• Last Updated: October 17, 2022
• Results First Posted: June 2, 2021

Record last verified: 2022-10

Data Sharing

• IPD Sharing: Will not share

Locations

US (3)