An Open-Label Study on the Treatment of Patients with Primary Sclerosing Cholangitis (PSC) with Norcholic Acid Tablets

Austria, Belgium, Denmark, France, Germany, Hungary, Netherlands, Norway, Poland, Sweden, Switzerland, United Kingdom (ICTRP)

ICTRP Studien-ID
CTIS2024-514292-18-00 (ICTRP)

Offizieller Titel (Genehmigt von der Ethikkommission)
An open-label study of patients with primary sclerosing cholangitis (PSC) treated with norucholic acid tablets (BASEC)

Wissenschaftlicher Titel
An Open-label study of patients with primary sclerosing cholangitis (PSC) treated with norucholic acid tablets - NUT-022/PSC (ICTRP)

Öffentlicher Titel
An Open-label study of patients with primary sclerosing cholangitis (PSC) treated with norucholic acid tablets (ICTRP)

Untersuchte Krankheit(en)
Primary Sclerosing Cholangitis (PSC)
MedDRA version: 20.1Level: LLTClassification code: 10036732Term: Primary sclerosing cholangitis Class: 10019805Therapeutic area: Diseases [C] - Digestive System Diseases [C06] (ICTRP)

Untersuchte Intervention
Product Name: NUT01, Product Code:PRD6821878, Pharmaceutical Form: FILM-COATED TABLET, Other descriptive name: , Strength: (ICTRP)

Studientyp
Interventional clinical trial of medicinal product (ICTRP)

Studiendesign
Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: no Number of treatment arms in the trial: (ICTRP)

Ein-/Ausschlusskriterien
Inclusion criteria: Signed informed consent., Males or females = 18 years., Patient has previously been diagnosed with PSC, has participated in the previous NUC 5/PSC trial and has completed the DBE phase with Visit 22, or has prematurely terminated the DBE phase before this trial has been started, or has prematurely terminated the DBE phase after this trial has been started, under the condition that the premature termination was due to lack of efficacy* *Lack of efficacy as defined in the NUC-5/PSC trial, Women of childbearing 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 treatment. 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. (ICTRP)

Exclusion criteria: History or presence of chronic alcoholic consumption (daily consumption > 30 g in men, > 20 g in women)., Abnormal renal function at screening, 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)., Any severe concomitant cardiovascular, renal, endocrine, or psychiatric disorder, which in the opinion of the investigator might have an influence on the patients compliance, or any disorder which in the opinion of the investigator may affect the patients safety., Any active malignant disease., Known intolerance/hypersensitivity to study drug, or drugs of similar chemical structure or pharmacological profile., Well-founded doubt about the patients cooperation, e.g., because of addiction to alcohol or drugs., Existing or intended pregnancy or breast-feeding., Participation in another clinical trial (other than the NUC-5/PSC trial) within the last 30 days prior to screening visit, simultaneous participation in another clinical trial, or previous enrolment in this trial and intake of Investigational Medicinal Product (IMP) within this trial, 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)., Patients who discontinued study participation in NUC-5/PSC due to an AE possibly caused by the study drug., Liver Cirrhosis or any cirrhosis-related symptoms which in the opinion of the investigator may affect the patients safety., Any known relevant infectious disease (e.g., active tuberculosis, AIDS defining diseases).

Primäre und sekundäre Endpunkte
Main Objective: To evaluate safety and tolerability of norucholic acid (NCA) film-coated tablets in the treatment of Primary Sclerosing Cholangitis (PSC).;Secondary Objective: To assess the efficacy of NCA in patients with PSC.;Primary end point(s): Occurrence of Treatment emergent adverse events (TEAEs), Occurrence of Serious TEAEs, Occurrence of Severe TEAEs, Occurrence of Adverse Drug reactions (ADRs), Occurrence of Unexpected TEAEs (ICTRP)

Secondary end point(s):(Safety) Changes from baseline in vital signs (blood pressure, heart rate) and body weight;Secondary end point(s):(Safety) Changes from baseline in hematology, serum chemistry (other than efficacy variables) and urinalysis;Secondary end point(s):(Efficacy) Course of liver stiffness;Secondary end point(s):(Efficacy) s-ALP in categories from baseline to EoT (ICTRP)

Registrierungsdatum
08.11.2024 (ICTRP)

Einschluss des ersten Teilnehmers
15.04.2025 (ICTRP)

Sekundäre Sponsoren
nicht verfügbar

Weitere Kontakte
Clinical Research and Development, zentrale@drfalkpharma.de, +4976115140, Dr. Falk Pharma GmbH (ICTRP)

Sekundäre IDs
nicht verfügbar

Angaben zur Verfügbarkeit von individuellen Teilnehmerdaten
NoIt is not yet decided which data will be made available. Neither can a time frame be indicated yet. Nevertheless, study results will be published independently from sharing other supportive information. (ICTRP)

Weitere Informationen zur Studie
https://euclinicaltrials.eu/search-for-clinical-trials/?lang=en&EUCT=2024-514292-18-00 (ICTRP)