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NCT03633227 | SNCTP000003721

Eine placebokontrollierte Studie zur Beurteilung der Verteilung und Abbau, sowie Sicherheit von Obeticholsäure bei Patienten mit primär biliäre Zirrhose und mäßiger bis schwerer Leberfunktionsstörung

Base di dati: BASEC (Importata da 24.05.2020), WHO (Importata da 22.03.2020)
Cambiato: 07.04.2020
Categoria di malattie: Anderes

Descrizione riassuntiva della sperimentazione (Fonte di dati: BASEC)

In dieser Studie wird die primär biliäre Cholangitis (auch bekannt als primär biliäre Zirrhose oder PBC) untersucht. Wir führen diese Studie durch, um zusätzliche Informationen über Obeticholsäure und ihre Wirkung auf Ihre PBC-Erkrankung zu gewinnen, wenn diese gemeinsam mit Ursodeoxycholsäure verabreicht wird und der Sponsor möchte den klinischen Nutzen für Patienten bestätigen. Darüber hinaus bewertet der Sponsor die Sicherheit des Arzneimittels sowie die Verteilung und den Abbau des Studienmedikaments. Dies wird Pharmakokinetik genannt.

Malattie studiate (Fonte di dati: BASEC)

primär biliäre Zirrhose und mäßige bis schwerer Leberfunktionsstörung

Health conditions (Fonte di dati: WHO)

Liver Cirrhosis, Biliary

Malattia rara (Fonte di dati: BASEC)

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

Der Zweck dieser Studie besteht darin, herauszufinden, wie das Prüfpräparat, im Körper absorbiert und verteilt wird und wie das Prüfpräparat aus dem Körper ausgeschieden wird. Dies wird als Pharmakokinetik bezeichnet. Zudem wird auch die Sicherheit des Prüfpräparats untersuchen.

Interventions (Fonte di dati: WHO)

Drug: Obeticholic Acid (OCA);Drug: Placebo

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

1. Eine definitive oder wahrscheinliche Diagnose von primärer biliäre Cholangitis
2. Hinweise auf eine Zirrhose
3. Leberfunktionsstörungen während der Voruntersuchung

Criteri di esclusione (Fonte di dati: BASEC)

1. Nicht-zirrhotische oder zirrhotische biliäre Cholangitis
2. Lebertransplantation oder Organtransplantation in der Krankengeschichte
3. Alkohol- oder Drogenmissbrauch innerhalb von 12 Monaten vor der Voruntersuchung

Inclusion/Exclusion Criteria (Fonte di dati: WHO)


Inclusion Criteria:

1. A definite or probable diagnosis of PBC (consistent with American Association for the
Study of Liver Diseases [AASLD] and European Association for the Study of the Liver
[EASL] Practice Guidelines [Lindor 2009, EASL 2009]), defined as having =2 of the
following 3 diagnostic factors:

- History of elevated ALP levels for at least 6 months

- Positive antimitochondrial antibody (AMA) titer or if AMA negative or low titer
(=1:80), PBC specific antibodies (anti-GP210 and/or anti-SP100) and/or antibodies
against the major M2 components (PDC-E2, 2-oxo-glutaric acid dehydrogenase
complex)

- Liver biopsy consistent with PBC (collected at any time prior to Screening)

2. Evidence of cirrhosis including at least one of the following:

- Biopsy results consistent with PBC Stage 4

- Liver stiffness as assessed by Transient Elastography (TE) Median Value =16.9kPa

- Clinical evidence in the absence of acute liver failure consistent with cirrhosis
including: gastroesophageal varices, ascites, radiological evidence of cirrhosis
(nodular liver or enlargement of portal vein and splenomegaly)

- Combined low platelet count (<140 000/mm3) with

- persistent decrease in serum albumin, or

- elevation in prothrombin time /INR (not due to antithrombotic agent use), or

- elevated bilirubin (2× ULN)

3. Satisfy the criteria of the modified CP classification for hepatic impairment during
Screening:

- Moderate: CP-B (Scores 7 to 9) or

- Severe: CP-C (Scores 10 to 12)

4. MELD score of 6 to 24 at Screening

5. Taking UDCA for at least 12 months (stable dose for =3 months) prior to Day 1, or
unable to tolerate or unresponsive to UDCA (no UDCA for =3 months)

Exclusion Criteria:

1. Non-cirrhotic or cirrhotic CP-A (Mild; Score 5 to 6)

2. History of liver transplant or organ transplant

3. History of alcohol or drug abuse within 12 months prior to Screening

4. Hepatic encephalopathy (as defined by a West Haven score of =2 [AASLD, EASL 2014])

5. History or presence of other concomitant liver diseases including:

- Hepatitis C virus infection and RNA positive

- Active hepatitis B infection; however, patients who have seroconverted (hepatitis
B surface antigen and hepatitis B e antigen negative) may be included in this
study after consultation with the medical monitor

- Primary sclerosing cholangitis

- Alcoholic liver disease

- Definite autoimmune liver disease or overlap hepatitis

- Gilbert's Syndrome

6. In the opinion of the Investigator, fluctuating or rapidly deteriorating hepatic
function prior to randomization

Altri dati sulla sperimentazione nel registro primario dell’OMS

https://clinicaltrials.gov/show/NCT03633227

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

http://apps.who.int/trialsearch/Trial2.aspx?TrialID=NCT03633227

Altre informazioni sulla sperimentazione

Data di registrazione della sperimentazione

08.04.2018

Inserimento del primo partecipante

22.06.2018

Stato di reclutamento

Recruiting

Titolo scientifico (Fonte di dati: WHO)

A Phase 4, Double-Blind, Randomized, Placebo-Controlled Study Evaluating the Pharmacokinetics and Safety of Obeticholic Acid in Patients With Primary Biliary Cholangitis and Moderate to Severe Hepatic Impairment

Tipo di sperimentazione (Fonte di dati: WHO)

Interventional

Disegno della sperimentazione (Fonte di dati: WHO)

Allocation: Randomized. Intervention model: Parallel Assignment. Primary purpose: Treatment. Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor).

Fase (Fonte di dati: WHO)

Phase 4

Punti finali primari (Fonte di dati: WHO)

Evaluate maximum concentration (Cmax) of OCA, its conjugates and total OCA (sum of OCA and its conjugates);Evaluate area under the concentration curve versus time curve from 0 to 24 hours (AUC 0-24) of OCA, its conjugates and total OCA;Evaluate safety and tolerability as assessed by the incidence of treatment emergent adverse events and serious treatment emergent adverse events comparing OCA to placebo

Punti finali secondari (Fonte di dati: WHO)

Evaluate the effect of OCA treatment compared to placebo on the model of end-stage liver disease (MELD) and its components;Evaluate the effect of OCA treatment compared to placebo on Child-Pugh score and its components;Evaluate the effect of OCA treatment compared to placebo on total bilirubin (mg/dL) and direct bilirubin (mg/dL);Evaluate the effect of OCA treatment compared to placebo on alkaline phosphatase (U/L), alanine aminotransferase (U/L), aspartate transaminase (U/L), and gamma glutamyl transaminase (U/L);Evaluate the effect of OCA treatment compared to placebo on platelets (109/L);Evaluate the effect of OCA treatment compared to placebo on fibroblast growth factor-19 (pg/mL);. Evaluate the effect of OCA treatment compared to placebo on 7a hydroxy-4-cholesten-3-one (ng/mL);Evaluate the effect of OCA treatment compared to placebo on plasma bile acids (µmol/L)

Contatto per informazioni (Fonte di dati: WHO)

Please refer to primary and secondary sponsors

Siti di esecuzione della sperimentazione

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

Bern

Paesi di esecuzione (Fonte di dati: WHO)

Argentina, Australia, Belgium, Brazil, Estonia, Germany, Hungary, Italy, Lithuania, Spain, United States

Contatto per maggiori informazioni sulla sperimentazione

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

Beat Weiss
+41-31-543 01 19
b.weiss@weiss-company.ch

Contatto per informazioni generali (Fonte di dati: WHO)

Christian Weyer, M.D.;Judith Romano
Intercept Pharmaceuticals
+1(646) 757-2335
judith.romano@interceptpharma.com

Contatto per informazioni scientifiche (Fonte di dati: WHO)

Christian Weyer, M.D.;Judith Romano
Intercept Pharmaceuticals
+1(646) 757-2335
judith.romano@interceptpharma.com

Responsabile della sperimentazione

Sponsor principale (Fonte di dati: WHO)

Intercept Pharmaceuticals

Altri numeri di identificazione delle sperimentazioni

BASEC ID (Fonte di dati: BASEC)

2019-00181

Secondary ID (Fonte di dati: WHO)

747-401