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

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Dr. Falk Pharma GmbH

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Digestive System Diseases [C06]

Trial duration

24 Mar 2025 → ongoing

Decision date (initial)

2025-03-21

Transition trial

No

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Dr. Falk Pharma GmbH

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Therapy

To evaluate safety and tolerability of norucholic acid (NCA) film-coated tablets in the treatment of Primary Sclerosing Cholangitis (PSC).

Secondary objectives 1

1. To assess the efficacy of NCA in patients with PSC.

Conditions and MedDRA coding

Primary Sclerosing Cholangitis (PSC)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

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 • 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
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 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.

Exclusion criteria 13

1. History or presence of chronic alcoholic consumption (daily consumption > 30 g in men, > 20 g in women).
2. Known intolerance/hypersensitivity to study drug, or drugs of similar chemical structure or pharmacological profile.
3. Well-founded doubt about the patient’s cooperation, e.g., because of addiction to alcohol or drugs.
4. Existing or intended pregnancy or breast-feeding.
5. 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
6. 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).
7. Patients who discontinued study participation in NUC-5/PSC due to an AE possibly caused by the study drug.
8. Liver Cirrhosis or any cirrhosis-related symptoms which in the opinion of the investigator may affect the patient’s safety.
9. Any known relevant infectious disease (e.g., active tuberculosis, AIDS defining diseases).
10. Abnormal renal function at screening
11. Thyroid-stimulating hormone (TSH) > ULN at screening (elevated levels [4.2-10 µU/mL] are acceptable if fT4 is measured and within the normal range).
12. 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.
13. Any active malignant disease.

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 5

1. Occurrence of Treatment emergent adverse events (TEAEs)
2. Occurrence of Serious TEAEs
3. Occurrence of Severe TEAEs
4. Occurrence of Adverse Drug reactions (ADRs)
5. Occurrence of Unexpected TEAEs

Secondary endpoints 4

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

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Dr. Falk Pharma GmbH

Sponsor organisation

Dr. Falk Pharma GmbH

Address

Leinenweberstrasse 5, Hochdorf Hochdorf

City

Freiburg Im Breisgau

Postcode

79108

Country

Germany

Scientific contact point

Organisation

Dr. Falk Pharma GmbH

Contact name

CTIS – Scientific Request

Public contact point

Organisation

Dr. Falk Pharma GmbH

Contact name

Clinical Research and Development

Third parties 4

Locations

10 EU/EEA countries · 22 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 62 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

2 submissions — initial application plus substantial / non-substantial modifications