Clinical trial comparing the efficacy and safety of Rhudex granules with placebo in the treatment of the liver disease Primary Biliary Cholangitis

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] - Immune System Diseases [C20]

• Registration Number: EUCTR2020-001961-34-SK
• Lead Sponsor: Dr. Falk Pharma GmbH

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-10-02
• Last Update Posted: 5 July 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 136

Inclusion Criteria

- Patient is able to understand the information on the trial and has signed the informed consent form,

- Male or female patients = 18 and < 75 years,

- PBC verified by at least 2 out of the following 3 criteria (consistent with EASL practice guidelines [2017]):
• Chronic cholestatic disease (e.g. elevated serum ALP) of at least 6 months duration,
• Positive AMA titer or presence of PBC-specific antibodies,
• Liver biopsy compatible with the diagnosis of non-cirrhotic PBC,

- UDCA treatment for at least 6 months (with a stable dose for = 3 months of at least 12 mg/kg/day) prior to baseline and no foreseen changes of the dosing regimen throughout trial participation,

- Inadequate response to UDCA treatment defined by serum ALP levels between 1.5x and 10x the upper limit of normal (ULN) at screening,

- Women of childbearing potential agree to use during the entire duration of the trial and until 4 weeks following the last dose of trial treatment a highly effective method of birth control.
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 106
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 30

Exclusion Criteria

- History or presence of other relevant concomitant liver diseases

- Liver cirrhosis

- History or presence of hepatic decompensation (e.g. variceal bleeding hepatic encephalopathy or poorly controlled ascites),

- Serum albumin less than 3.2 g/dL, at screening,

- 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 < 60 mL/min) at screening visit,

- Thyroid-stimulating hormone (TSH) > ULN at screening (elevated levels [4.2-10 µU/mL] are acceptable if free thyroxine 4 (fT4) is measured and within the normal range),

- Current history of significant alcohol consumption (> 30 g/day in men, > 20 g/day in women on average) for a period of more than 3 consecutive months within 1 year prior to screening,

- Any illness or medical conditions that are unstable or could jeopardise the safety of the patient and his/her compliance in the trial or might interfere with the trial results,

- Previous and concurrent HIV infection,

- Previous or concurrent cancer except cervical carcinoma in situ, treated basal cell carcinoma, or any cancer curatively treated < 3 years before trial entry,

- Existing or intended pregnancy or breast-feeding.

Study & Design

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

Primary Outcome Measures

Name	Time	Method
Main Objective: To evaluate the efficacy of 3 doses of RhuDex vs placebo for the treatment of PBC in patients with an inadequate response to UDCA.;Secondary Objective: - To identify efficacious RhuDex dose(s) for the treatment of PBC for further evaluation in phase III<br>- To study safety and tolerability of RhuDex;Primary end point(s): Primary Efficacy Endpoint:<br><br>• Relative change (%) in Alkaline phosphatase (ALP) from baseline to End Of Trial (EoT).<br><br>---<br><br>Safety Endpoints:<br><br>• Adverse Events,<br>• Vital signs (blood pressure, heart rate), body temperature, body weight and BMI,<br>• Haematology, serum chemistry, urinalysis, coagulation,<br>• ECG parameters (12 leads),<br>• Patient’s tolerability of the IMP.<br>;Timepoint(s) of evaluation of this end point: Throughout the study

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): Secondary Efficacy Endpoints:<br><br>- ALP at each trial visit (screening to follow-up),<br><br>- Absolute and relative changes (%) of ALP from baseline to each visit up to EOT, and from EOT to the follow-up visit,<br><br>- ?-GT, AST, ALT, and total and conjugated bilirubin levels at each trial visit (screening to follow-up),<br><br>- Absolute and relative changes (%) of ?-GT, AST, ALT and total and conjugated bilirubin levels from baseline to each visit up to EOT, and from EOT to the follow-up visit.<br>;Timepoint(s) of evaluation of this end point: Throughout the study