Clinical trial comparing the efficacy and safety of Rhudex granules with placebo in the treatment of the liver disease Primary Biliary Cholangitis
- Conditions
- Primary biliary cholangitisMedDRA version: 20.0Level: SOCClassification code 10019805Term: Hepatobiliary disordersSystem Organ Class: 10019805 - Hepatobiliary disordersTherapeutic area: Diseases [C] - Immune System Diseases [C20]
- Registration Number
- EUCTR2020-001961-34-IT
- Lead Sponsor
- DR. FALK PHARMA GMBH
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 136
- Patient is able to understand the information on the trial and has signed the informed consent form,
- Male or female patients = 18 and < 74 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,
- 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
- 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 < 30 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 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>• Relative change (%) in Alkaline phosphatase (ALP) from baseline to End Of Trial (EoT).<br><br>Safety Endpoints:<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.;Timepoint(s) of evaluation of this end point: Throughout the study
- Secondary Outcome Measures
Name Time Method