Ursodeoxycholic Acid (UDCA) for Hepatic Sarcoidosis

NCT03602976 | Terminated Phase 2 Results FDA Drug

• 1 other identifier: 828780
• Study Type: interventional
• Target: 7
• Locations: 1 country
• Sites: 1

Brief Summary

This study aims to (1) evaluate efficacy of UDCA in improving liver function and quality of life; (2) monitor safety, tolerability of UDCA, as well as progression of hepatic sarcoidosis and liver disease, in patients diagnosed with hepatic sarcoidosis. A minimum of 10 subjects will be followed for 12 months. For all subjects, initial 6 months will be observational; in subsequent 6 months, UDCA will be administered. Visits will occur every 3 months and involve routine blood collection.

Conditions

Hepatic Sarcoidosis, Elevated Alkaline Phosphatase

Keywords

hepatic sarcoidosis; elevated alp

Outcome Measures

Primary Outcomes (1)

• Alkaline Phosphatase (U/L) or Gamma Glutamyl Transferase (U/L) 6 Months After Initiation of Ursodeoxycholic Acid

  Alkaline Phosphatase (U/L) or Gamma Glutamyl Transferase (U/L) 6 months after initiation of ursodeoxycholic acid

  6 months after initiaiton of UDCA

Secondary Outcomes (4)

• Liver Stiffness

  Baseline and 6 months after initiation of UDCA

• Bilirubin

  Baseline and 6 months after initiation of UDCA

• AST

  Baseline and 6 months after initiation of UDCA

• ALT

  Baseline and 6 months after initiation of UDCA

Study Arms (2)

Observation

NO INTERVENTION

UDCA at Month 6

EXPERIMENTAL

Drug: Ursodeoxycholic Acid

Interventions

Ursodeoxycholic Acid DRUG

weight-based dosing

Also known as: UDCA, ursodiol

UDCA at Month 6

Eligibility Criteria

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

You may qualify if:

• Systemic sarcoidosis with evidence of liver involvement as denoted by any of the following:
• Elevated liver-specific alkaline phosphatase
• Granulomas on liver biopsy
• Hepatomegaly on imaging
• Portal Hypertension (via imaging or endoscopy)
• Stable dose of immunosuppressant, if taking (no dose variation for 6 months)
• If cirrhotic, absence of hepatocellular carcinoma as indicated by imaging within 6 months of screening

You may not qualify if:

• Female who is pregnant, planning to become pregnant during the study, or breastfeeding
• Clinically significant abnormalities, co-morbidities, or recent alcohol/drug abuse that make the subject an unsuitable candidate
• Concurrent liver disease including hepatitis B, hepatitis C, alcohol-related liver disease, autoimmune hepatitis, primary biliary cholangitis, primary sclerosing cholangitis
• Currently on UDCA
• Prior intolerance to UDCA
• Receipt of any investigational product within a time period equal to 10 half-lives of the product, or 6 weeks (whichever is longer), to study drug administration
• Current evidence of hepatic decompensation (variceal bleeding, hepatic encephalopathy, or ascites). In the event potential participant is post-transplant, no evidence of hepatic decompensation since transplantation

Sponsors & Collaborators

• Ethan Weinberg: lead
• American Association for the Study of Liver Diseases: collaborator
• Meridian Bioscience, Inc.: collaborator

Study Sites (1)

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

MeSH Terms

Interventions

Ursodeoxycholic Acid

Intervention Hierarchy (Ancestors)

Deoxycholic Acid; Cholic Acids; Bile Acids and Salts; Steroids; Fused-Ring Compounds; Polycyclic Compounds; Cholanes

Limitations and Caveats

Early termination due to poor recruitment in light of the COVID-19 pandemic, affecting data analysis.

Results Point of Contact

• Title: Dr. Ethan Weinberg
• Organization: University of Pennsylvania

ethan.weinberg@pennmedicine.upenn.edu

215-349-8222

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NON RANDOMIZED
• Masking: NONE
• Masking Details: All subjects will receive appropriately dosed UDCA after six months of observation
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Assistant Professor of Clinical Medicine

Model Details: Subjects will act as their own controls by undergoing a six-month initial observational period prior to initiating the six-month UDCA intervention

Study Record Dates

• First Submitted: July 19, 2018
• First Posted: July 27, 2018
• Study Start: August 20, 2018
• Primary Completion: January 12, 2022
• Study Completion: January 12, 2022
• Last Updated: June 7, 2023
• Results First Posted: June 7, 2023

Record last verified: 2023-06

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)