Doppelblinde, randomisierte, placebo-kontrollierte Phase III-Studie zum Vergleich von Norursodeoxycholsäure-Kapseln versus Placebo bei der Behandlung von primär sklerosierender Cholangitis

Product Name: Norursodeoxycholic acid
Pharmaceutical Form: Capsule
INN or Proposed INN: Norucholic acid
CAS Number: 99697-24-2
Current Sponsor code: NorUDCA
Other descriptive name: Norursodeoxycholic acid, NCA
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 250-
Pharmaceutical form of the placebo: Capsule
Route of administration of the placebo: Oral use

Gender:
Female: yes
Male: yes

Inclusion criteria:
1. Signed informed consent.
2. Males or females.
3. Verified PSC.
4. Liver biopsy available.
8. Women of child-bearing potential, i.e. fertile, following menarche and until becoming post-menopausal unless permanently sterile, who are sexually active have to apply a highly effective method of birth Control with a low failure rate (i.e., less than 1% per year) when used constantly and correctly such as combined (estrogen and progestogen
containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal or transdermal), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable or implantable), intrauterine device (IUD), intrauterine hormone-releasing system (IUS), bilateral tubal occlusion, vasectomized
partner, or sexual abstinence (only accepted as a highly effective contraceptive measure if it is the usual and preferred lifestyle of the patient), throughout the treatment period and for four weeks following the last dose of study drug. Hormonal methods other than levonorgestrel containing devices or medroxyprogesterone injections should be supplemented with use of a male condom. Women of non-childbearing potential may be included if surgically sterile or post-menopausal for at least 2 years. The investigator is responsible for determining whether the patient has this adequate birth control for study participation.

Inclusion Criteria for the open-label extension {OLE) phase:
1. Signed additional informed consent for OLE phase
2. DBE phase completed with Visit 22
Are the trial subjects under 18? yes
Number of subjects for this age range: 50
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 210
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 40

Exclusion criteria:
1. History or presence of other concomitant liver diseases including.
4. Secondary causes of Sclerosing Cholangitis
11. Total bilirubin > 4.0 mg/dL (> 68 ?mol/L) at screening or baseline.
13.Any known relevant infectious disease (e.g., active tuberculosis, AIDS
defining diseases). 14.Abnormal renal function 15. Thyroid-stimulating hormone (TSH) > ULN at screening (elevated levels [4.2-10 ?U/mL] are acceptable if fT4 is measured and within the normal range).
16. Any severe concomitant cardiovascular, renal, endocrine, or psychiatric disorder, which in the opinion of the investigator might have an influence on the patient's compliance or on the interpretation of the results, or patient with atrial fibrillation or any disorder which in the opinion of the investigator may affect the patient's safety.
17.Any active malignant disease.
18.Known intolerance/hypersensitivity to study drug, or drugs of similar chemical structure or pharmacological profile.
19.Well-founded doubt about the patient's cooperation.
20.Existing or intended pregnancy or breast-feeding.
21.Participation in another clinical trial within the last 30 days prior to screening visit.
22.Patients who have an absolute contraindication for liver biopsy.
23.Imprisoned persons, persons admitted to nursing homes, persons under legal guardianship, and persons not able to express their consent (e.g. due to mental impairment).