Undersøgelse af OCA i kombination med BZF, der evaluerer effektivitet, sikkerhed og tolerabilitet hos patienter med PBC
17. juni 2026 opdateret af: Intercept Pharmaceuticals
Et fase 2a, dobbeltblindt, randomiseret, aktivt kontrolleret, parallelt gruppestudie, der evaluerer effektiviteten, sikkerheden og tolerabiliteten af bezafibrat administreret i kombination med obeticholsyre hos forsøgspersoner med primær biliær kolangitis
Undersøgelse for at bestemme effekten af forsøgslægemidlet bezafibrat (BZF) alene og i kombination med undersøgelseslægemidlet obeticholsyre (også kendt som OCA) hos patienter med primær biliær kolangitis (også kendt som PBC).
Studieoversigt
Status
Afsluttet
Betingelser
Undersøgelsestype
Interventionel
Tilmelding (Faktiske)
72
Fase
- Fase 2
Kontakter og lokationer
Dette afsnit indeholder kontaktoplysninger for dem, der udfører undersøgelsen, og oplysninger om, hvor denne undersøgelse udføres.
Studiesteder
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Buenos Aires, Argentina
- Hospital Italiano de Buenos Aires
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Pilar, Argentina
- Hospital Universitario Austral
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Rosario, Argentina
- Hospital Provincial del Centenario
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Buenos Aires
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La Plata, Buenos Aires, Argentina
- Hospital Italiano La Plata
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Ramos Mejía, Buenos Aires, Argentina
- DIM Clinical Privada
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Alberta
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Edmonton, Alberta, Canada, T6G 287
- The Northern Alberta Clinical Trials and Research Centre
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British Columbia
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Vancouver, British Columbia, Canada, V6Z 2K5
- Pacific Gastroenterology Associates
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Quebec
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Montreal, Quebec, Canada
- University of Montreal
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Alabama
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Birmingham, Alabama, Forenede Stater, 35233
- University of Alabama at Birmingham
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California
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Coronado, California, Forenede Stater, 92118
- Southern California Gastrointestinal (GI) and Liver Centers (SCLC) - Coronado
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Mission Hills, California, Forenede Stater, 91345
- Facey Medical Group
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Florida
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Miami, Florida, Forenede Stater, 33136
- Schiff Center for Liver Diseases / University of Miami
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Tampa, Florida, Forenede Stater, 33606
- Tampa General Medical Group
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Georgia
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Atlanta, Georgia, Forenede Stater, 30309
- Piedmont Atlanta Hospital
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Illinois
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Maywood, Illinois, Forenede Stater, 60459
- Loyola University Medical Center
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Louisiana
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New Orleans, Louisiana, Forenede Stater, 70121
- Ochsner Medical Center
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Massachusetts
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Boston, Massachusetts, Forenede Stater, 02215
- Beth Israel Deaconess Medical Center Harvard Liver Research Center
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New York
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New York, New York, Forenede Stater, 10016-6402
- NYU Langone Medical Center
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North Carolina
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Raleigh, North Carolina, Forenede Stater, 27607
- Wake Endoscopy Center
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Ohio
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Cleveland, Ohio, Forenede Stater, 44106
- University Hospitals Cleveland Medical Center
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Tennessee
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Cordova, Tennessee, Forenede Stater, 38018
- Gastro One
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Johnson City, Tennessee, Forenede Stater, 37604
- Gastrointestinal Associates of Northeast Tennessee
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Texas
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Dallas, Texas, Forenede Stater, 75203
- Methodist Clinical Research Institute (CRI)
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Houston, Texas, Forenede Stater, 77030-2717
- Houston Methodist Cancer Center
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San Antonio, Texas, Forenede Stater, 78215
- American Research Corporation at the Texas Liver Institute
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Bologna, Italien
- Azienda Ospedaliero-Universitaria di Bologna Policlinico S. Orsola-Malpighi
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Deltagelseskriterier
Forskere leder efter personer, der passer til en bestemt beskrivelse, kaldet berettigelseskriterier. Nogle eksempler på disse kriterier er en persons generelle helbredstilstand eller tidligere behandlinger.
Berettigelseskriterier
Aldre berettiget til at studere
18 år og ældre (Voksen, Ældre voksen)
Tager imod sunde frivillige
Ingen
Beskrivelse
Inklusionskriterier:
- En sikker eller sandsynlig diagnose af PBC
- Kvalificerende ALP og/eller bilirubin leverbiokemiske værdier
- Tager ursodeoxycholsyre (UDCA) i mindst 12 måneder eller ingen UDCA i 3 måneder før dag 1
Ekskluderingskriterier:
- Anamnese eller tilstedeværelse af andre samtidige leversygdomme
- Tilstedeværelse af kliniske komplikationer af PBC
- Historik eller tilstedeværelse af dekompenserende begivenheder
- Nuværende eller historie med galdeblæresygdom
- Hvis kvinde, kendt graviditet eller har en positiv uringraviditetstest (bekræftet af en positiv serumgraviditetstest), eller ammende
- Behandling med kommercielt tilgængelig OCA eller deltagelse i en tidligere undersøgelse, der involverer OCA eller andre FXR-agonister eller peroxisomproliferatoraktiverede receptor (PPAR)-agonister inden for 3 måneder før screening
- Behandling med kommercielt tilgængelige fibrater eller deltagelse i en tidligere undersøgelse, der involverer fibrat inden for 3 måneder før screening
Studieplan
Dette afsnit indeholder detaljer om studieplanen, herunder hvordan undersøgelsen er designet, og hvad undersøgelsen måler.
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: Randomiseret
- Interventionel model: Parallel tildeling
- Maskning: Firedobbelt
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
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Aktiv komparator: Dobbeltblind (DB) fasebehandling A: BZF 100 milligram (mg) tablet med øjeblikkelig frigivelse (IR)
Hver deltager vil tage én OCA placebotablet, én BZF 100 mg IR-tablet og én BZF placebotablet dagligt.
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En tablet obeticholsyre placebotablet én gang dagligt
En tablet af bezafibrate placebotablet én gang dagligt
En tablet bezafibrat 100 mg IR én gang dagligt
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Aktiv komparator: Dobbeltblind (DB) fasebehandling B: BZF 400 mg IR-tablet
Hver deltager vil tage en OCA placebotablet og to BZF 200 mg IR-tabletter (for at opnå en dosis på 400 mg) dagligt.
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En tablet obeticholsyre placebotablet én gang dagligt
To tabletter bezafibrat 200 mg IR én gang dagligt til BZF 400 mg IR
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Eksperimentel: Dobbeltblind (DB) fasebehandling C: OCA 5 mg + BZF 100 mg IR
Hver deltager vil tage en OCA 5 mg tablet, en BZF 100 mg IR tablet og en BZF placebotablet dagligt.
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En tablet af bezafibrate placebotablet én gang dagligt
En tablet bezafibrat 100 mg IR én gang dagligt
En tablet obeticholsyre 5 mg tablet én gang dagligt.
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Eksperimentel: Dobbeltblind (DB) fasebehandling D: OCA 5 mg + BZF 400 mg IR
Hver deltager vil tage en OCA 5 mg tablet og to BZF 200 mg IR tabletter (for at opnå 400 mg dosis) dagligt.
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To tabletter bezafibrat 200 mg IR én gang dagligt til BZF 400 mg IR
En tablet obeticholsyre 5 mg tablet én gang dagligt.
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Eksperimentel: Long Term Safety Extension (LTSE) Fasebehandling D af DB-fasen: OCA 5 mg + BZF 400 mg IR
Hver deltager vil tage en OCA 5 mg tablet og to BZF 200 mg IR tabletter (for at opnå 400 mg dosis) dagligt.
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To tabletter bezafibrat 200 mg IR én gang dagligt til BZF 400 mg IR
En tablet obeticholsyre 5 mg tablet én gang dagligt.
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Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
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Change From Baseline in Alkaline Phosphatase (ALP)
Tidsramme: Baseline to Week 12
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Serum samples were collected at scheduled visits during the double-blind treatment period.
Changes in ALP were evaluated using a mixed-effects repeated-measures model (MMRM) to assess the effects of treatment groups over time.
Baseline was defined as the mean of all available evaluations prior to the first administration of investigational product.
Change from Baseline was calculated as post Baseline value minus Baseline value.
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Baseline to Week 12
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Sekundære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
|---|---|---|
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Percentage of Participants With Response Rates of ≥10%, ≥20%, ≥30% and ≥40% Reduction From Baseline in ALP
Tidsramme: At Week 12
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Responders were defined as participants achieving a ≥10%, ≥20%, ≥30%, or ≥40% reduction from baseline in serum ALP at week 12 during the DB treatment period with non-responder imputation applied.
Baseline ALP was defined as the last evaluations prior to the first administration of investigational product.
The percentage of responders were summarized by treatment group for each response threshold and compared using a Cochran Mantel Haenszel test stratified by the randomization stratification factor.
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At Week 12
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Normalization Rates of ALP at Week 12
Tidsramme: At Week 12
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Percentage of participants achieving a response in serum ALP at week 12 during the DB treatment period was assessed using non-responder imputation.
Analyses of ALP normalization rates were performed using a Cochran-Mantel-Haenszel test stratified by the randomization stratification factor.
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At Week 12
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Normalization Rates of Biochemical Disease Markers
Tidsramme: At Week 12
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Percentage of participants achieving a response in biochemical disease markers including ALP, alanine amino transferase (ALT), aspartate amino transferase (AST), gamma-glutamyl transferase (GGT), total and conjugated bilirubin and lipid panel (cholesterol, high density lipoprotein [HDL] and low density lipoprotein [LDL]) at Week 12 during the DB treatment period has been presented.
Analyses of response rates and normalization rates were performed using a Cochran-Mantel-Haenszel test stratified by the randomization stratification factor.
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At Week 12
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Change From Baseline in in GGT, ALT and AST Levels
Tidsramme: Baseline and At Week 12
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Blood samples were collected at indicated timepoint and Change from Baseline in GGT, ALT and AST were analyzed using the MMRM model and results were summarized in standard International System of Units (SI) by treatment group using descriptive statistics at baseline and at each on-study evaluation.
Baseline was defined as the mean of all available evaluations prior to the first administration of investigational product.
Change from baseline was calculated as post Baseline value minus Baseline value.
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Baseline and At Week 12
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Change From Baseline in Total and Conjugated Bilirubin
Tidsramme: Baseline and At Week 12
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Blood samples were collected at indicated timepoints and the changes in total and conjugated bilirubin were evaluated using MMRM to assess the effects of treatment groups over time.
Baseline was defined as the mean of all available evaluations prior to the first administration of investigational product.
Change from baseline was calculated as post Baseline value minus Baseline value.
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Baseline and At Week 12
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Change From Baseline in Lipid Panel
Tidsramme: Baseline and At Week 12
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Blood samples were collected at indicated timepoints and the changes in lipid panel including cholesterol, HDL and LDL were evaluated using MMRM to assess the effects of treatment groups over time.
Baseline was defined as the mean of all available evaluations prior to the first administration of investigational product.
Change from baseline was calculated as post Baseline value minus Baseline value.
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Baseline and At Week 12
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Change From Baseline of the Plasma Value of 7 Alpha (α) Hydroxy 4 Cholesten-3 One (C4)
Tidsramme: Baseline and At Week 12
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Blood samples were collected at indicated timepoints for the assessment of C4.
Baseline was defined as the mean of all available evaluations prior to the first administration of investigational product.
Change from baseline was calculated as post Baseline value minus Baseline value.
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Baseline and At Week 12
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Number of Participants With Clinically Significant Changes From Baseline in Bile Acids
Tidsramme: At Week 12
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Blood samples were collected for the assessment of bile acids.
Baseline was defined as the mean of all available evaluations prior to the first administration of investigational product.
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At Week 12
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Samarbejdspartnere og efterforskere
Det er her, du vil finde personer og organisationer, der er involveret i denne undersøgelse.
Sponsor
Efterforskere
- Studieleder: Lynda Szczech, M.D., Intercept Pharmaceuticals, Inc
Datoer for undersøgelser
Disse datoer sporer fremskridtene for indsendelser af undersøgelsesrekord og resumeresultater til ClinicalTrials.gov. Studieregistreringer og rapporterede resultater gennemgås af National Library of Medicine (NLM) for at sikre, at de opfylder specifikke kvalitetskontrolstandarder, før de offentliggøres på den offentlige hjemmeside.
Studer store datoer
Studiestart (Faktiske)
21. marts 2022
Primær færdiggørelse (Faktiske)
1. september 2025
Studieafslutning (Faktiske)
1. september 2025
Datoer for studieregistrering
Først indsendt
28. januar 2022
Først indsendt, der opfyldte QC-kriterier
11. februar 2022
Først opslået (Faktiske)
15. februar 2022
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
16. juli 2026
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
17. juni 2026
Sidst verificeret
1. juni 2026
Mere information
Begreber relateret til denne undersøgelse
Yderligere relevante MeSH-vilkår
- Patologiske processer
- Sygdomme i fordøjelsessystemet
- Galdevejssygdomme
- Leversygdomme
- Galdevejssygdomme
- Kolestase, intrahepatisk
- Kolestase
- Levercirrhose
- Patologiske tilstande, tegn og symptomer
- Fibrose
- Levercirrhose, galdevejr
- Organiske kemikalier
- Ethers
- Kulbrinter
- Kulbrinter, cyklisk
- Syrer, acyklisk
- Carboxylsyrer
- Kulbrinter, aromatisk
- Amider
- Fenoler
- Benzenderivater
- Butyrater
- Syrer, carbocykliske
- Benzoates
- Phenylethere
- Benzamider
- Fibrinsyrer
- Isobutyrater
- Chlorobenzoates
- Bezafibrate
- Obetikolsyre
Andre undersøgelses-id-numre
- 747-214
Lægemiddel- og udstyrsoplysninger, undersøgelsesdokumenter
Studerer et amerikansk FDA-reguleret lægemiddelprodukt
Ja
Studerer et amerikansk FDA-reguleret enhedsprodukt
Ingen
Disse oplysninger blev hentet direkte fra webstedet clinicaltrials.gov uden ændringer. Hvis du har nogen anmodninger om at ændre, fjerne eller opdatere dine undersøgelsesoplysninger, bedes du kontakte register@clinicaltrials.gov. Så snart en ændring er implementeret på clinicaltrials.gov, vil denne også blive opdateret automatisk på vores hjemmeside .