Phase 2
N=18
Vidofludimus Calcium for Primary Sclerosing Cholangitis
Primary Sclerosing Cholangitis
Bottom Line
View on ClinicalTrials.gov: NCT03722576 ↗Enrolled (actual)
18
Serious AEs
0.0%
Results posted
Jun 2021
Primary outcome: Primary: Subjects Who Experience a Positive Outcome as Measured by Combination of Serum Alkaline Phosphatase (ALP) and Aspartate Aminotransferase (AST) Levels. — 3 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Vidofludimus calcium (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Elizabeth Carey
- Primary completion
- Jun 2020
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Subjects Who Experience a Positive Outcome as Measured by Combination of Serum Alkaline Phosphatase (ALP) and Aspartate Aminotransferase (AST) Levels. |
3 | — |
| SECONDARY Abnormal Aspartate Aminotransferase (AST) |
8 | — |
| SECONDARY Abnormal Alanine Aminotransferase (ALT) |
8 | — |
| SECONDARY Abnormal Total Bilirubin |
3 | — |
| SECONDARY Abnormal Direct Bilirubin |
4 | — |
Summary
To examine the safety, tolerability, and efficacy of daily dosing with vidofludimus calcium over a 6-month period.
Eligibility Criteria
Inclusion Criteria
- 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 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
Data sourced from ClinicalTrials.gov (NCT03722576). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.