Clinical trial • Gastroenterology|Rare Disease

NORUCHOLIC ACID for Primary sclerosing cholangitis

Clinical trial of NORUCHOLIC ACID for Primary sclerosing cholangitis. open-label. 25 participants.

Overview

Trial Therapeutic Area
Gastroenterology|Rare Disease
Trial Disease
Primary sclerosing cholangitis
Drug Modality
Small molecule
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
08-11-2024
First CTIS Authorization Date
10-03-2025

Trial design

open-label trial in Germany, France, Sweden and others.

Open Label
Yes
Target Sample Size
25
Trial Duration For Participant
72

Eligibility

Recruits 25 The protocol excludes 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). Signed informed consent from the participant is required; only adults (≥18 years) are eligible, so assent for minors is not applicable..

Pregnancy Exclusion
Existing or intended pregnancy or breast-feeding.
Vulnerable Population
The protocol excludes 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). Signed informed consent from the participant is required; only adults (≥18 years) are eligible, so assent for minors is not applicable.

Inclusion criteria

  • {"criterion_text":"- Signed informed consent.\n- Males or females ≥ 18 years.\n- Patient has previously been diagnosed with PSC, has participated in the previous NUC 5/PSC trial and •\thas completed the DBE phase with Visit 22, or •\thas prematurely terminated the DBE phase before this trial has been started, or •\thas 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\n- 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

  • {"criterion_text":"- History or presence of chronic alcoholic consumption (daily consumption > 30 g in men, > 20 g in women).\n- Abnormal renal function at screening\n- 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).\n- 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.\n- Any active malignant disease.\n- Known intolerance/hypersensitivity to study drug, or drugs of similar chemical structure or pharmacological profile.\n- Well-founded doubt about the patient’s cooperation, e.g., because of addiction to alcohol or drugs.\n- Existing or intended pregnancy or breast-feeding.\n- 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\n- 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).\n- Patients who discontinued study participation in NUC-5/PSC due to an AE possibly caused by the study drug.\n- Liver Cirrhosis or any cirrhosis-related symptoms which in the opinion of the investigator may affect the patient’s safety.\n- Any known relevant infectious disease (e.g., active tuberculosis, AIDS defining diseases)."}

Endpoints

Primary endpoints

  • {"endpoint_text":"- Occurrence of Treatment emergent adverse events (TEAEs)","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Occurrence of Serious TEAEs","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Occurrence of Severe TEAEs","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Occurrence of Adverse Drug reactions (ADRs)","definition_or_measurement_approach":""}
  • {"endpoint_text":"- Occurrence of Unexpected TEAEs","definition_or_measurement_approach":""}

Secondary endpoints

  • {"endpoint_text":"- (Safety) Changes from baseline in vital signs (blood pressure, heart rate) and body weight","definition_or_measurement_approach":""}
  • {"endpoint_text":"- (Safety) Changes from baseline in hematology, serum chemistry (other than efficacy variables) and urinalysis","definition_or_measurement_approach":""}
  • {"endpoint_text":"- (Efficacy) Course of liver stiffness","definition_or_measurement_approach":""}
  • {"endpoint_text":"- (Efficacy) s-ALP in categories from baseline to EoT","definition_or_measurement_approach":""}

Recruitment

Planned Sample Size
25
Recruitment Window Months
32
Consent Approach
Signed informed consent from the participant is required. Only adults (≥18 years) may participate; therefore no assent for minors is planned. Country-specific subject information and informed consent forms are provided; ICF and patient-facing documents are available in multiple languages (examples present in the dossier include German, English, French, Dutch, Polish, Hungarian, Swedish, Norwegian, Danish). Persons under legal guardianship and those unable to express consent are excluded.

Geography

Total Number Of Sites
22
Total Number Of Participants
79

Germany

Earliest CTIS Part Ii Submission Date
26-02-2025
Latest Decision Or Authorization Date
16-07-2025
Processing Time Days
140
Number Of Sites
11
Number Of Participants
40

Sites

Site Name
Medical Center - University Of Freiburg
Department Name
Klinik für Innere Medizin II, Gastroenterologie, Hepatologie, Infektiologie & Endokrinologie
Contact Person Name
Tobias Böttler
Site Name
Medizinische Hochschule Hannover
Department Name
Gastroenterologie, Hepatologie, Infektiologie, Endokrinologie
Contact Person Name
Heiner Wedemeyer
Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Hepatologie und Gastroenterologie
Contact Person Name
Münevver Demir
Contact Person Email
muenevver.demir@charite.de
Site Name
Universitaetsklinikum Essen AöR
Department Name
Klinik für Gastroenterologie, Hepatologie und Transplantationsmedizin
Contact Person Name
Julia Kälsch
Contact Person Email
julia.kaelsch@uk-essen.de
Site Name
Universitaetsklinikum Erlangen AöR
Department Name
Medizinische Klinik I
Contact Person Name
Peter Dietrich
Contact Person Email
peter.dietrich@uk-erlangen.de
Site Name
Goethe University Frankfurt
Department Name
Medizinische Klinik I
Contact Person Name
Stefan Zeuzem
Contact Person Email
Zeuzem@em.uni-frankfurt.de
Site Name
University Medical Center Hamburg-Eppendorf
Department Name
Hepatologische Studienambulanz
Contact Person Name
Christoph Schramm
Contact Person Email
c.schramm@uke.de
Site Name
Universitaetsklinikum Heidelberg AöR
Department Name
Medizinische Klinik IV
Contact Person Name
Michael Dill
Site Name
Universitaetsklinikum Tuebingen AöR
Department Name
Medical Clinic Department I
Contact Person Name
Christoph Berg
Site Name
Universitaetsklinikum Leipzig AöR
Department Name
Klinik und Poliklinik für Onkologie, Gastroenterologie, Hepatologie Pneumologie / Sektion
Contact Person Name
Thomas Berg
Site Name
Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz KöR
Department Name
Medizinische Klinik und Poliklinik
Contact Person Name
Christian Labenz

France

Earliest CTIS Part Ii Submission Date
27-01-2025
Latest Decision Or Authorization Date
16-07-2025
Processing Time Days
170
Number Of Sites
2
Number Of Participants
5

Sites

Site Name
Assistance Publique Hopitaux De Paris
Department Name
hépatologie
Contact Person Name
Olivier Chazouilleres
Contact Person Email
olivier.chazouilleres@aphp.fr
Site Name
Centre Hospitalier Universitaire De Toulouse
Department Name
hépatologie
Contact Person Name
Christophe Bureau
Contact Person Email
bureau.c@chu-toulouse.fr

Sweden

Earliest CTIS Part Ii Submission Date
22-11-2024
Latest Decision Or Authorization Date
21-07-2025
Processing Time Days
241
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
Karolinska University Hospital
Department Name
Forskningen Övre Buk
Contact Person Name
Annika Bergquist
Contact Person Email
annika.bergquist@ki.se

Austria

Earliest CTIS Part Ii Submission Date
27-02-2025
Latest Decision Or Authorization Date
22-07-2025
Processing Time Days
145
Number Of Sites
2
Number Of Participants
11

Sites

Site Name
Medical University Of Graz
Department Name
Department of Internal Medicine, Division of Gastroenterology and Hepatology
Contact Person Name
Peter Fickert
Contact Person Email
peter.fickert@medunigraz.at
Site Name
Medical University Of Vienna
Department Name
Klin. Abt. für Gastroenterologie und Hepatologie
Contact Person Name
Michael Trauner

Netherlands

Earliest CTIS Part Ii Submission Date
21-02-2025
Latest Decision Or Authorization Date
25-08-2025
Processing Time Days
185
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
Amsterdam UMC Stichting
Department Name
Department of Gastroenterology and Hepatology
Contact Person Name
Joost P.H. Drenth
Contact Person Email
j.p.h.drenth@amsterdamumc.nl

Norway

Earliest CTIS Part Ii Submission Date
17-02-2025
Latest Decision Or Authorization Date
17-07-2025
Processing Time Days
150
Number Of Sites
1
Number Of Participants
5

Sites

Site Name
Akershus University Hospital
Department Name
Gastro
Contact Person Name
Kristin Kaasen Jorgensen

Hungary

Earliest CTIS Part Ii Submission Date
31-01-2025
Latest Decision Or Authorization Date
18-07-2025
Processing Time Days
168
Number Of Sites
1
Number Of Participants
3

Sites

Site Name
University Of Debrecen
Department Name
Gasztroenterológiai Klinika
Contact Person Name
István Tornai
Contact Person Email
itornai@med.unideb.hu

Denmark

Earliest CTIS Part Ii Submission Date
04-03-2025
Latest Decision Or Authorization Date
17-07-2025
Processing Time Days
135
Number Of Sites
1
Number Of Participants
7

Sites

Site Name
Aarhus University Hospital
Department Name
Hepato-Gastroenterology
Contact Person Name
Gerda Elisabeth Villadsen

Belgium

Earliest CTIS Part Ii Submission Date
18-02-2025
Latest Decision Or Authorization Date
22-07-2025
Processing Time Days
154
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
UZ Leuven
Department Name
Gastroenterology and Hepatology
Contact Person Name
Hannah van Malenstein

Poland

Earliest CTIS Part Ii Submission Date
14-02-2025
Latest Decision Or Authorization Date
23-07-2025
Processing Time Days
159
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
ID Clinic Arkadiusz Pisula
Department Name
ID Clinic
Contact Person Name
Ewa Janczewska
Contact Person Email
idclinic@idclinic.eu

Sponsor

Primary sponsor

Full Name
Dr. Falk Pharma GmbH
Organisation Type
Pharmaceutical company
Country Of Registered Address
Germany

Third parties

  • {"country":"Germany","full_name":"A&M Labor fuer Analytik und Metabolismusforschung Service GmbH","duties_or_roles":"archiving of blood samples for future trial-related scientific investigation; code:15; code:4","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"Germany","full_name":"GKM Gesellschaft fuer Therapieforschung mbH","duties_or_roles":"roles codes:1,10,11,12,15 (Statistical Planning),5,6","organisation_type":"Pharmaceutical company"}
  • {"country":"Germany","full_name":"allphamed Pharbil Arzneimittel GmbH","duties_or_roles":"Drug supplier (code:15)","organisation_type":"Pharmaceutical company"}
  • {"country":"Germany","full_name":"Labor Dr. Spranger","duties_or_roles":"archiving of blood samples for future trial-related scientific investigation; code:15; code:4","organisation_type":"Laboratory/Research/Testing facility"}

Investigational products

Investigational Product Name
NUT01
Active Substance
NORUCHOLIC ACID
Modality
Small molecule
Routes Of Administration
ORAL USE
Route
oral
Orphan Designation
Yes
Maximum Dose
1500 mg per day

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