Clinical study comparing two doses of norucholic acid against placebo in the treatment of a liver disease called primary biliary cholangitis in patients without sufficient response to ursodeoxycholic acid

Phase 1

• Conditions: primary biliary cholangitis; MedDRA version: 21.0Level: PTClassification code 10080429Term: Primary biliary cholangitisSystem Organ Class: 10019805 - Hepatobiliary disorders; Therapeutic area: Diseases [C] - Digestive System Diseases [C06]

• Registration Number: EUCTR2021-001431-56-FR
• Lead Sponsor: Dr. Falk Pharma GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2022-04-06
• Last Update Posted: 2 September 2024

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 90

Inclusion Criteria

• Signed informed consent,
• Male or female patients = 18 and = 74 years at screening,
• PBC verified by at least 2 out of the following 3 criteria at screening:
- Chronic cholestatic disease of at least 12 months duration,
- Positive anti-mitochondrial antibody (AMA) titer or, if AMA negative or in low titer (< 1:80), presence of 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 available for review and compatible with the diagnosis of non-cirrhotic PBC,
• Ursodeoxycholic acid (UDCA) treatment for at least 12 months (with a stable dose for = 3 months) prior to screening,
• Women of childbearing potential agreeing to use a highly effective method of birth control during the entire duration of the trial and for 4 weeks following the last dose of trial treatment, defined as those which result in a low failure rate (i.e., less than 1% per year) when used consistently and correctly such as implants, injectables, combined oral contraceptive methods, some intrauterine devices (IUD), sexual abstinence, or vasectomized partner. Women of non-childbearing potential (surgically sterile [e.g., hysterectomy, bilateral salpingectomy, bilateral oophorectomy], or postmenopausal with at least two years without spontaneous menses) may be included. The investigator is responsible for determining whether the patient has adequate birth control for trial participation.

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 80
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 10

Exclusion Criteria

• History or presence of any other relevant concomitant liver diseases.
• Patients taking prohibited medications
• Liver cirrhosis. NOTE: Patients with compensated cirrhosis and a Child-Pugh Score <8 are allowed to participate,
• Any known relevant infectious disease (e.g., active tuberculosis, acquired immunodeficiency syndrome [AIDS]-defining diseases),
• Abnormal renal function (glomerular filtration rate estimated from cystatin C < 30 ml/min) at screening visit,
• Previous or concurrent cancer except cervical carcinoma in situ, treated basal cell carcinoma, or any cancer curatively treated < 3 years before screening,
• Existing or intended pregnancy or breast-feeding

Study & Design

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