Primary sclerosing cholangitis (PSC) Digestive System
Conditions
Interventions
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 en
Sponsors
Dr Falk Pharma (Germany)
Eligibility
Sex/Gender
All
Age
18 Years to 99 Years
Inclusion criteria
Inclusion criteria: Current inclusion criteria as of 23/05/2025: Male and female patients = 18 years, with PSC, who have participated in the NUC-5/PSC study. _____ Previous 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.
Exclusion criteria
Exclusion criteria: Current exclusion criteria as of 23/05/2025: Patients who discontinued the NUC-5/PSC study due to adverse drug reactions (side effects of the study drug) are not eligible. Other exclusion criteria include chronic alcohol consumption, advanced cirrhosis, liver transplantation, severe infections and other severe diseases _____ Previous 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 µU/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 patient’s compliance, or any disorder which in the opinion of the investigator may affect the patient’s 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 patient’s 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 patient’s safety. 13. Any known relevant infectious disease (e.g., active tuberculosis, AIDS defining diseases).
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Current primary outcome measure as of 23/05/2025: Occurrence of adverse events during the 18 months treatment period with NCA, and, in particular, · how serious and severe they are, · if they have a causal relationship with the study drug, · if they have been observed previously under the treatment with NCA, or if they are unexpected (“new”), and · if they lead to withdrawal of NCA. _____ Previous primary outcome measure: 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 | — |
Secondary
| Measure | Time frame |
|---|---|
| Current secondary outcome measures as of 23/05/2025: Safety: Change from the beginning of treatment (“baseline”) to the end of treatment · in vital signs (e.g. blood pressure, heart rate), and · in laboratory values (e.g. blood count, blood coagulation, thyroid hormone, kidney function parameters, blood coagulation, urine examination). Efficacy endpoints include: · course of liver stiffness from baseline to end of treatment, · course in alkaline phosphatase (ALP) and other liver enzymes from baseline to end of treatment (ALP is a liver enzyme particularly important for the evaluation of PSC), · occurrence of relevant strictures of the bile ducts during the treatment period, · course of pruritus (itchiness), · course of fatigue (tiredness), · occurrence of “clinical events” like cancer, transplantation, and cirrhosis-related events. _____ Previous secondary outcome measures: 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 | — |
Countries
Austria, Belgium, Denmark, France, Germany, Hungary, Netherlands, Norway, Poland, Sweden, Switzerland, United Kingdom
Outcome results
None listed