A clinical trial where neither the doctor, patient or sponsor know whether aplacebo or active medicine is being given to the patient with cirrhosis to see if the medicine is safe in the treatment of that disease and to see how study drug is absorbed, distributed through the body and excreted out of the body.

Phase 1

• Conditions: Primary Biliary Cholangitis and Moderate to Severe Hepatic Impairment; Therapeutic area: Diseases [C] - Digestive System Diseases [C06]

• Registration Number: EUCTR2017-001762-13-EE
• Lead Sponsor: Intercept Pharmaceuticals, Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2018-10-15
• Study Completion: 2021-07-09
• Last Update Posted: 8 August 2022

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 22

Inclusion Criteria

1. A definite or probable diagnosis of PBC (consistent with American Association for the Study of Liver Diseases [AASLD] and European Association for the Study of the Liver [EASL] Practice Guidelines, defined as having =2 of the following 3 diagnostic factors:
• History of elevated ALP levels for at least 6 months
• Positive antimitochondrial antibody (AMA) titer or if AMA negative or low titer (=1:80), PBC specific antibodies (anti-GP210 and/or anti-SP100) and/or antibodies against the major M2 components (PDC E2, 2-oxo-glutaric acid dehydrogenase complex)
• Liver biopsy consistent with PBC (collected at any time prior to Screening)
2. Evidence of cirrhosis including at least one of the following:
• Biopsy results consistent with PBC Stage 4
• Liver stiffness as assessed by TE Value =16.9kPa
• Clinical evidence in the absence of acute liver failure consistent with cirrhosis including: gastroesophageal varices, ascites, radiological evidence of cirrhosis (nodular liver or enlargement of portal vein and splenomegaly)
• Combined low platelet count (<140 000/mm3) with
- persistent decrease in serum albumin, or
- elevation in prothrombin time/INR (not due to antithrombotic agent use), or
- elevated bilirubin (2× ULN)
3. Satisfy the criteria of the modified CP classification for hepatic impairment during Screening:
• Moderate: CP-B (Scores 7 to 9) or
• Severe: CP-C (Scores 10 to12)
4. MELD score of 6 to 24 at Screening
5. Taking UDCA for at least 12 months (stable dose for =3 months) prior to Day 1, or unable to tolerate or unresponsive to UDCA (no UDCA for =3 months)

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 31
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 19

Exclusion Criteria

1. Non-cirrhotic or cirrhotic CP-A (Mild; Score 5 to 6)
2. History of liver transplant or organ transplant
3. History of alcohol or drug abuse within 12 months prior to Screening
4. Current hepatic encephalopathy (as defined by a West Haven score of =2)
5. History or presence of other concomitant liver diseases including:
• Hepatitis C virus infection and RNA positive
• Active hepatitis B infection; however, patients who have seroconverted (hepatitis B surface antigen and hepatitis B e antigen negative) may be included in this study after consultation with the Medical Monitor
• Primary sclerosing cholangitis
• Alcoholic liver disease
• Definite autoimmune liver disease or overlap hepatitis
• Gilbert’s Syndrome
6. In the opinion of the Investigator, fluctuating or rapidly deteriorating hepatic function prior to randomization

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified