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SNCTP000003254 | EUCTR2016-003367-19 | BASEC2018-01918

Doppelblinde, randomisierte, placebo-kontrollierte Phase III-Studie zum Vergleich von Norursodeoxycholsäure-Kapseln versus Placebo bei der Behandlung von primär sklerosierender Cholangitis

Base di dati: BASEC (Importata da 28.03.2024), WHO (Importata da 20.03.2024)
Cambiato: 19 gen 2024, 01:00
Categoria di malattie: Malattie dell'apparato digerente (non cancro)

Descrizione riassuntiva della sperimentazione (Fonte di dati: BASEC)

Das Ziel der klinischen Studie ist der Vergleich der Einnahme von Kapseln mit entweder 1500mg Norursodeoxycholsäure (norUDCA) oder von Placebo-Kapseln einmal täglich zur Behandlung von PSC.In dieser Studie werden die Patienten in zwei verschiedene Behandlungsgruppen eingeteilt. Dann werden die Ergebnisse beider Gruppen miteinander verglichen. Die Zuteilung zu den Behandlungsgruppen erfolgt mithilfe eines elektronischen Verfahrens vollständig nach dem Zufallsprinzip und vor der ersten Dosis der Studienmedikation. Die Behandlung ist doppelblind. Das bedeutet, dass weder Patienten noch Prüfarzt wissen, in welcher Behandlungsgruppe die Patienten sind. Wenn der Prüfarzt jedoch wissen muss, welche Behandlung ein Patient erhält, kann die „Verblindung“ aufgehoben werden. Um diese Doppelverblindung zu ermöglichen, werden Placebo-Kapseln verwendet, in denen sich kein Wirkstoff befindet, die aber genauso aussehen wie die Kapseln, die norUDCA enthalten. Die Patienten müssen daher ganz unabhängig davon, in welcher Behandlungsgruppe sie sind, jeden Tag dieselbe Anzahl an Kapseln einnehmen. Es ist geplant, insgesamt 300 Patienten mit PSC aus mehreren europäischen Ländern sowie Russland in die Studie aufzunehmen. Die Studie wird von der Dr. Falk Pharma GmbH aus Freiburg, Deutschland, finanziert. Ein Auftragsforschungsinstitut (die GKM Gesellschaft für Therapieforschung mbH aus München, Deutschland) unterstützt den Sponsor Dr. Falk Pharma GmbH bei allen praktischen Aspekten der Durchführung dieser Studie.

Malattie studiate(Fonte di dati: BASEC)

primär sklerosierende Cholangitis

Health conditions (Fonte di dati: WHO)

Primary Sclerosing Cholangitis (PSC) is a slowly progressing chronic cholestatic liver disease of assumed autoimmune, but finally unidentified etiology, characterized by a chronic inflammatory and fibro-obliterative destruction of extra-, and intrahepatic bile ducts. The disease is characterized by diffuse inflammation, fibrosis, and strictures of the intra- and/or extrahepatic bile ducts with an impaired biliary secretion of potentially aggressive bile fluid often leading to cirrhosis.
MedDRA version: 20.1Level: LLTClassification code 10036732Term: Primary sclerosing cholangitisSystem Organ Class: 100000004871;Therapeutic area: Diseases [C] - Digestive System Diseases [C06]

Malattia rara (Fonte di dati: BASEC)

No

Interventi esaminati (p. es. medicamento, terapia, campagna) (Fonte di dati: BASEC)

Norursodeoxycholsäure (norUDCA) ist ein neues Medikament, das sich derzeit in der Phase der Arzneimittelprüfung für die Behandlung von PSC befindet.

Interventions (Fonte di dati: WHO)


Product Name: Norursodeoxycholic acid
Pharmaceutical Form: Capsule
INN or Proposed INN: Norucholic acid
CAS Number: 99697-24-2
Current Sponsor code: NorUDCA
Other descriptive name: Norursodeoxycholic acid, NCA
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 250-
Pharmaceutical form of the placebo: Capsule
Route of administration of the placebo: Oral use

Criteri per la partecipazione alla sperimentazione (Fonte di dati: BASEC)

1. Unterschriebene Einwilligungserklärung.
2. Männer und Frauen
3. nachgewiesene primär sklerosierende Cholangitis (PSC)

Criteri di esclusione (Fonte di dati: BASEC)

1. Aus der Krankengeschichte bekannte oder bestehende andere gleichzeitige Lebererkrankungen
4. Sekundäre Ursachen für sklerosierende Cholangitis
11. Gesamtbilirubin > 4,0 mg/dl (> 68 µmol/l) beim Screening (Voruntersuchung) oder bei der Baseline-Visite.

Inclusion/Exclusion Criteria (Fonte di dati: WHO)

Gender:
Female: yes
Male: yes

Inclusion criteria:
1. Signed informed consent.
2. Males or females.
3. Verified PSC.
4. Liver biopsy available.
8. Women of child-bearing 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 drug. Hormonal methods other than levonorgestrel containing devices or medroxyprogesterone injections should be
supplemented with use of a male condom. 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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 50
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 210
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 40

Exclusion criteria:
1. History or presence of other concomitant liver diseases including.
4. Secondary causes of Sclerosing Cholangitis
11. Total bilirubin > 4.0 mg/dL (> 68 ?mol/L) at screening or baseline.
13.Any known relevant infectious disease (e.g., active tuberculosis, AIDS defining diseases).
14.Abnormal renal function
15. 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).
16. 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 on the interpretation of the results, or patient with atrial fibrillation or any disorder which in the opinion of the investigator may affect the patient's safety.
17.Any active malignant disease.
18.Known intolerance/hypersensitivity to study drug, or drugs of similar chemical structure or pharmacological profile.
19.Well-founded doubt about the patient's cooperation.
20.Existing or intended pregnancy or breast-feeding.
21.Participation in another clinical trial within the last 30 days prior to screening visit.
22.Patients who have an absolute contraindication for liver biopsy.
23.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).

Altri dati sulla sperimentazione nel registro primario dell’OMS

https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2016-003367-19

Altri dati sulla sperimentazione dalla banca dati dell’OMS (ICTRP)

https://trialsearch.who.int/Trial2.aspx?TrialID=EUCTR2016-003367-19
Altre informazioni sulla sperimentazione

Data di registrazione della sperimentazione

9 gen 2018

Inserimento del primo partecipante

30 mar 2018

Stato di reclutamento

Not Recruiting

Titolo scientifico (Fonte di dati: WHO)

Double-blind, randomized, placebo-controlled, phase III study comparing norursodeoxycholic acid capsules with placebo in the treatment of primary sclerosing cholangitis

Tipo di sperimentazione (Fonte di dati: WHO)

Interventional clinical trial of medicinal product

Disegno della sperimentazione (Fonte di dati: WHO)

Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2

Fase (Fonte di dati: WHO)

Human pharmacology (Phase I): noTherapeutic exploratory (Phase II): noTherapeutic confirmatory - (Phase III): yesTherapeutic use (Phase IV): no

Punti finali primari (Fonte di dati: WHO)

Main Objective: To show the superiority of norursodeoxycholic acid (norUDCA) compared to placebo in the treatment of Primary Sclerosing Cholangitis (PSC) with regard to prevention of disease progression.;Secondary Objective: To study safety and tolerability (Adverse Events, laboratory parameters) of norUDCA,
To assess quality of life.
;Primary end point(s): Partial normalization of s-ALP and no worsening of disease stage.

;Timepoint(s) of evaluation of this end point: Visit 2 and Visit 14

Punti finali secondari (Fonte di dati: WHO)

Secondary end point(s): Secondary endpoints with regard to liver stiffness, fibrosis stage, liver histology, s-ALP levels, dominant strictures, quality of life;Timepoint(s) of evaluation of this end point: week 96

Contatto per informazioni (Fonte di dati: WHO)

Dr. Falk Pharma GmbH

Risultati della sperimentazione (Fonte di dati: WHO)

Sintesi dei risultati

Double-blind, randomized, placebo-controlled, phase III study comparing norursodeoxycholic acid capsules with placebo in the treatment of primary sclerosing cholangitis

Collegamento ai risultati nel registro primario

ancora nessuna informazione disponibile

Informazioni sulla disponibilità dei dati dei singoli partecipanti

ancora nessuna informazione disponibile

Siti di esecuzione della sperimentazione

Siti di esecuzione in Svizzera (Fonte di dati: BASEC)

Basilea, Berna, Losanna, Lugano, San Gallo, Zurigo

Paesi di esecuzione (Fonte di dati: WHO)

Austria, Belgium, Czech Republic, Czechia, Denmark, Finland, France, Germany, Hungary, Ireland, Italy, Lithuania, Netherlands, Norway, Poland, Russian Federation, Spain, Sweden, Switzerland, United Kingdom

Contatto per maggiori informazioni sulla sperimentazione

Dati della persona di contatto in Svizzera (Fonte di dati: BASEC)

Dr. Falk Pharma GmbH, Department of Clinical Research
+49 761 1514 0
zentrale@drfalkpharma.de

Contatto per informazioni generali (Fonte di dati: WHO)

Department of Clinical Research
Leinenweberstrasse 5
Dr. Falk Pharma GmbH
004976115140
zentrale@drfalkpharma.de

Contatto per informazioni scientifiche (Fonte di dati: WHO)

Department of Clinical Research
Leinenweberstrasse 5
Dr. Falk Pharma GmbH
004976115140
zentrale@drfalkpharma.de

Autorizzazione da parte della commissione d’etica (Fonte di dati: BASEC)

Nome della commissione d’etica che rilascia l’autorizzazione (nel caso di studi multicentrici solo la commissione direttiva)

Kantonale Ethikkommission Zürich

Data di autorizzazione da parte della commissione d’etica

31.01.2019

Altri numeri di identificazione delle sperimentazioni

Numero di identificazione della sperimentazione della commissione d’etica (BASEC-ID) (Fonte di dati: BASEC)

2018-01918

Secondary ID (Fonte di dati: WHO)

NUC-5/PSC
2016-003367-19-DE
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