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

ICTRP Studien-ID
ISRCTN58756615 (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 (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)
Digestive System (ICTRP)

Untersuchte Intervention
This is an open-label, single-arm study. To be able to take part, patients must have participated in the previous NUC-5/PSC study. If patients are eligible, they will be asked if they would like to enrol in the NUT-022/PSC study. They will receive the medication NCA at a dose of 1500 mg per day, taken as three tablets (containing 500 mg NCA each) orally daily. The patient will come to regular interim visits every 3 months. Participants will have a total of 8 visits over a period of up to 72 weeks. (ICTRP)

Studientyp
Interventional (ICTRP)

Studiendesign
Interventional non randomized (Safety, Efficacy) (ICTRP)

Ein-/Ausschlusskriterien
Inclusion criteria: 1. Signed informed consent.
2. Males or females = 18 years.
3. Patient has previously been diagnosed with PSC, has participated in the previous NUC 5/PSC trial and
3.1. has completed the DBE phase with Visit 22, or
3.2. 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.
4. 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 nonchildbearing potential may be included if surgically sterile or postmenopausal 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: 1. History or presence of chronic alcoholic consumption (daily consumption >30 g in men, >20 g in women)
2. Abnormal renal function at screening
3. Thyroid-stimulating hormone (TSH) >ULN at screening (elevated levels [4.2-10 U/mL] are acceptable if fT4 ismeasured and within the normal range).
4. 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.
5. Any active malignant disease
6. Known intolerance/hypersensitivity to study drug, or drugs of similar chemical structure or pharmacological profile
7. Well-founded doubt about the patients cooperation, e.g., because of addiction to alcohol or drugs.
8. Existing or intended pregnancy or breast-feeding.
9. 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
10. 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).
11. Patients who discontinued study participation in NUC-5/PSC due to an AE possibly caused by the study drug.
12. Liver Cirrhosis or any cirrhosis-related symptoms which in the opinion of the investigator may affect the patients safety.
13. Any known relevant infectious disease (e.g., active tuberculosis, AIDS defining diseases).

Primäre und sekundäre Endpunkte
1. Occurrence of Treatment emergent adverse events (TEAEs) is measured using patient records at the time of consent, screening phase, treatment phase, and during 4 weeks after EOT/withdrawal visit
2. Occurrence of Serious TEAEs is measured using patient records at the time of consent, screening phase, treatment phase, and during 4 weeks after EOT/withdrawal visit
3. Occurrence of Severe TEAEs is measured using patient records at the time of consent, screening phase, treatment phase, and during 4 weeks after EOT/withdrawal visit
4. Occurrence of Adverse Drug reactions (ADRs) is measured using patient records at the time of consent, screening phase, treatment phase, and during 4 weeks after EOT/withdrawal visit
5. Occurrence of Unexpected TEAEs is measured using patient records at the time of consent, screening phase, treatment phase, and during 4 weeks after EOT/withdrawal visit (ICTRP)

1. (Safety) Changes from baseline in vital signs (blood pressure, heart rate) and body weight � measured at baseline, v 2 to v7
2. (Safety) Changes from baseline in haematology, serum chemistry (other than efficacy variables) and urinalysis -measured at baseline, v 2 to v7
3. (Efficacy) Course of liver stiffness � measured at screening and at v7
4. (Efficacy) s-ALP in categories from baseline to EoT - measured at baseline, v 2 to v7 (ICTRP)

Registrierungsdatum
27.01.2025 (ICTRP)

Einschluss des ersten Teilnehmers
31.03.2025 (ICTRP)

Sekundäre Sponsoren
nicht verfügbar

Weitere Kontakte
nicht verfügbar

Sekundäre IDs
2024-514292-18, Nil known, NUT-022/PSC (ICTRP)

Angaben zur Verfügbarkeit von individuellen Teilnehmerdaten
YesStored in non-publicly available repository. The data collected will be transferred and stored in a pseudonymised form in the clinical data base that has restricted access to sponsor and its representatives. For analysis purposes, the participants will always be identified by their 5-digit participant number. The data will be stored for at least 25 years after the end or termination of the clinical trial. (ICTRP)

Weitere Informationen zur Studie
https://www.isrctn.com/ISRCTN58756615 (ICTRP)