primary biliary cholangitis MedDRA version: 27.0 Level: PT Classification code 10080429 Term: Primary biliary cholangitis System Organ Class: 10019805 - Hepatobiliary disorders
Conditions
Interventions
Product Name: Norucholic acid (NCA) 500 mg
Product Code: NCA 500mg
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Norucholic acid
CAS Number: 9697-24-2
Current Sponsor code: NCA
Other de
Sponsors
Dr. Falk Pharma GmbH
Eligibility
Sex/Gender
All
Inclusion criteria
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 (=65 years) yes F.1.3.1 Number of subjects for this age range 10
Exclusion criteria
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the efficacy of two doses of norucholic acid vs. placebo for the treatment of primary biliary cholangitis (PBC) in patients with an inadequate response to ursodeoxycholic acid (UDCA).;Secondary Objective: To identify efficacious norucholic acid dose(s) for the treatment of PBC for further evaluation in phase III, To study safety and tolerability (adverse events, laboratory parameters) of norucholic acid.;Primary end point(s): Mean relative change (%) in ALP between the baseline visit and the EOT visit (Last Observation Carried Forward, LOCF).;Timepoint(s) of evaluation of this end point: End of Treatment, 12 weeks after baseline | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): 1. Proportions of patients with at least 10%, at least 20%, and at least 40% reduction in ALP between baseline and End of treatment (LOCF), 2. Proportion of patients with normalisation of ALP (< ULN) at any visit during the treatment phase, 3. Proportion of patients with bilirubin levels <0.6x ULN at any visit during the treatment phase, 4. Proportion of patients with partial normalisation of ALP (< 1.5x ULN) at any visit during the treatment phase, 5. ALP at each trial visit (screening to follow-up), 6. Absolute and relative changes (%) of ALP from baseline to each visit up to End of treatment (LOCF), and from End of treatment to the follow-up visit, 7. Gamma-glutamyltransferase (?-GT), AST, ALT, albumin, platelet count and total and conjugated bilirubin levels at each trial visit (screening to follow-up), 8. Absolute and relative changes (%) of ?-GT, AST, ALT, albumin, platelet count and total and conjugated bilirubin levels from baseline to each visit up to End of treatment (LOCF), and from End of treatment to the follow-up visit. ;Timepoint(s) of evaluation of this end point: 1. End of Treatment, 12 weeks after baseline 2. any visit during the treatment phase 3. any visit during the treatment phase 4. any visit during the treatment phase 5. at each trial visit 6. End of treatment and follow-up visit 7. at each trial visit 8. End of treatment and follow-up visit | — |
Countries
Austria, Belgium, Denmark, Finland, France, Germany, Netherlands, Norway, Poland, Switzerland, United Kingdom
Contacts
Public ContactDept. of Clinic. Res. & Development
Dr. Falk Pharma GmbH
Outcome results
None listed