Clinical trial • Phase III • Immunology|Gastroenterology|Rare Disease

seladelpar for Primary biliary cholangitis

Phase III trial of seladelpar for Primary biliary cholangitis. open-label, none/not specified-controlled. 272 participants.

Overview

Trial Therapeutic Area
Immunology|Gastroenterology|Rare Disease
Trial Disease
Primary biliary cholangitis
Trial Stage
Phase III
Drug Modality
Small molecule
Orphan Drug
Yes

Key dates

Initial CTIS Submission Date
29-05-2024
First CTIS Authorization Date
11-07-2024

Trial design

open-label, none/not specified-controlled Phase III trial in Netherlands, Hungary, Austria and others.

Open Label
Yes
Comparator
None/Not specified
Target Sample Size
272

Eligibility

Recruits 272 The trial record indicates vulnerable population selection (isVulnerablePopulationSelected = true). Subjects must provide written informed consent: "Must have given informed consent (signed and dated)". Multiple informed consent forms are provided (main ICF, pregnant-partner/newborn ICFs, genomic ICF, optional research ICFs) in country-specific languages. No explicit mention of assent procedures for minors is provided in the available documents..

Pregnancy Exclusion
For females, pregnancy or breast-feeding
Vulnerable Population
The trial record indicates vulnerable population selection (isVulnerablePopulationSelected = true). Subjects must provide written informed consent: "Must have given informed consent (signed and dated)". Multiple informed consent forms are provided (main ICF, pregnant-partner/newborn ICFs, genomic ICF, optional research ICFs) in country-specific languages. No explicit mention of assent procedures for minors is provided in the available documents.

Inclusion criteria

  • {"criterion_text":"-Must have given informed consent (signed and dated)"}
  • {"criterion_text":"-Participated in a prior PBC study with seladelpar (e.g., including CB8025-21629, CB8025-31735, or CB8025-31731), current PBC studies (CB8025-32048 or CB8025-21838), or completed a future PBC study with seladelpar that allows rollover into CB8025-31731-RE, and meet eligibility criteria for the current study."}
  • {"criterion_text":"-Females of reproductive potential must use at least one barrier contraceptive and a second effective birth control method during the study and for at least 90 days after the last dose. Male subjects who are sexually active with female partners of reproductive potential must use barrier contraception and their female partners must use a second effective birth control method during the study and for at least 90 days after the last dose"}

Exclusion criteria

  • {"criterion_text":"-Exclusion criteria are only applicable for subjects with a study drug interruption greater than 4 weeks prior to Day 1 of this study and for subjects who participated in CB8025-21838 irrespective of seladelpar interruption 1. Treatment-related AE leading to study drug discontinuation in a previous PBC study with seladelpar"}
  • {"criterion_text":"-Known history of alpha-1-antitrypsin deficiency"}
  • {"criterion_text":"-Known history of chronic viral hepatitis"}
  • {"criterion_text":"-For females, pregnancy or breast-feeding"}
  • {"criterion_text":"-Use of colchicine, methotrexate, azathioprine or long-term use of systemic steroids (e.g. prednisone, prednisolone, budesonide) (>2 weeks) within 2 months prior to Screening. See the concomitant medication section for additional medications that may be excluded."}
  • {"criterion_text":"-Current use of fibrates or use of fibrates within 3 months prior to Screening"}
  • {"criterion_text":"-Current use of obeticholic acid or use of obeticholic acid within 3 months prior to Screening"}
  • {"criterion_text":"-Use of an experimental or unapproved treatment for PBC within 3 months prior to Screening"}
  • {"criterion_text":"-History of malignancy diagnosed or treated, actively or within 2 years, or active evaluation for malignancy; localized treatment of squamous or non-invasive basal cell skin cancers and cervical carcinoma in-situ is allowed if appropriately treated prior to Screening"}
  • {"criterion_text":"-Treatment with any other investigational therapy or medical device within 30 days or within 5 half-lives, whatever is longer, prior to Screening"}
  • {"criterion_text":"-Any other condition(s) that would compromise the safety of the subject or compromise the quality of the clinical study as judged by the Investigator"}
  • {"criterion_text":"-A medical condition, other than PBC, that in the Investigator's opinion would preclude full participation in the study or confound its results (e.g., cancer, any active infection)"}
  • {"criterion_text":"-Immunosuppressant therapies (e.g., cyclosporine, tacrolimus, antiTNF or other immunosuppressive biologics)"}
  • {"criterion_text":"-Other medications that effect liver or GI functions such as absorption of medications may be prohibited and should be discussed with the medical monitor on a case-by-case basis"}
  • {"criterion_text":"-Positive for: a. Hepatitis B, defined as the presence of hepatitis B surface antigen b. Hepatitis C, defined as the presence of hepatitis C virus ribonucleic acid c. Human immunodeficiency virus (HIV) antibody"}
  • {"criterion_text":"-Active COVID-19 infection during Screening."}
  • {"criterion_text":"-AST or ALT above 3 × the upper limit of normal (ULN)"}
  • {"criterion_text":"-Total bilirubin above 2 × ULN"}
  • {"criterion_text":"-MELD score ≥ 12. For subjects on anticoagulation medication, evaluation of the baseline INR, in concert with any current dose adjustments in anti-coagulant medications, will be taken into account when calculating this score. This will be done in consultation with the medical monitor."}
  • {"criterion_text":"-Evidence of advanced PBC as defined by the Rotterdam criteria (albumin below 1 × lower limit of normal AND total bilirubin above 1 × ULN)"}
  • {"criterion_text":"-Estimated glomerular filtration rate ≤ 45 mL/min/1.73 m2 (calculated by Modification of Diet in Renal Disease formula)"}
  • {"criterion_text":"-Auto-immune hepatitis"}
  • {"criterion_text":"-Primary sclerosing cholangitis"}

Endpoints

Primary endpoints

  • {"endpoint_text":"-Treatment-emergent AEs (National Cancer Institute [NCI] Common Terminology Criteria for Adverse Events [CTCAE] Version 5.0), biochemistry and hematology results are collected","definition_or_measurement_approach":"Treatment-emergent adverse events collected and graded using NCI CTCAE Version 5.0; collection of biochemistry and hematology laboratory results."}

Secondary endpoints

  • {"endpoint_text":"-Occurrence of the following adjudicated PBC clinical outcomes: -Overall death -Liver transplantation -MELD score ≥ 15 for at least 2 consecutive visits -Ascites requiring treatment -Hospitalization for new onset or recurrence, of any: -Variceal bleeding, Hepatic encephalopathy, Spontaneous bacterial peritonitis","definition_or_measurement_approach":"Adjudicated clinical PBC outcomes tracked including all-cause mortality, liver transplantation, MELD ≥15 on ≥2 consecutive visits, clinically significant ascites, and hospitalizations for variceal bleeding, hepatic encephalopathy, or spontaneous bacterial peritonitis (definitions and adjudication described in protocol)."}
  • {"endpoint_text":"-Biochemical markers: -Response on composite of ALP and total bilirubin -Proportion of subjects with normalization of ALP -Relative and absolute changes of the following: -Alkaline phosphatase (ALP) -Aspartate aminotransferase (AST) -Alanine aminotransferase (ALT) -Gamma-glutamyl transferase (GGT) -Bilirubin (total, direct, indirect)","definition_or_measurement_approach":"Biochemical response assessed by composite ALP and total bilirubin criteria; proportion with ALP normalization; relative and absolute changes in listed liver enzymes and bilirubin measured by standard laboratory assays."}
  • {"endpoint_text":"-Change from Baseline in pruritus NRS","definition_or_measurement_approach":"Pruritus measured using the Numerical Rating Scale (NRS); change from baseline score calculated."}

Recruitment

Registry Or Advocacy Recruitment
True — Site-and-Patient-advocacy contact lists are provided (document: 'Site-and-Patient-advocacy_Contact-List-for-ICF_AT_clean_Public').
Digital Remote Recruitment
True — digital/remote methods documented include email communication (Scout Email Communication), eCOA/ePRO handheld and electronic patient-reported outcome tools (multiple 'ePRO'/'eCOA' documents), and Direct-to-Patient logistics.
Planned Sample Size
272
Recruitment Window Months
59
Consent Approach
Informed consent is required from each subject ("Must have given informed consent (signed and dated)"). Subjects sign the main ICF prior to any study procedures; additional specific consent forms are provided for pregnant participants, pregnant partners/newborn follow-up, genomic research, optional sub-studies and optional procedures. Country-specific ICFs are provided in multiple languages (examples in the record include: English, Dutch, Hungarian, Greek, French, Polish, Spanish, German, Italian, Czech, Romanian). There is no explicit mention of assent procedures for minors in the available documents.

Methods

  • Direct to Patient (DTP) distribution and home delivery (documented via third party MARKEN Germany GmbH with duty 'Direct to Patient (DTP)')
  • Email outreach via Scout Clinical (documents: 'Scout Email Communication'; third party 'Scout Clinical' involved) for patient communication and recruitment support
  • Primary care / PCP patient-enrolment letters (document: 'ASSURE_PCP-Patient-Enrollment-Letter') and patient welcome/visit reminder cards (documents: 'Patient Welcome Letter', 'Patient-Visit-Reminder-Card')
  • On-site/site-based recruitment through participating hospitals and hepatology/gastroenterology clinics (multiple site documents and ICFs per country)

Geography

Total Number Of Sites
29
Total Number Of Participants
272

Netherlands

Earliest CTIS Part Ii Submission Date
21-06-2024
Latest Decision Or Authorization Date
11-07-2024
Processing Time Days
20
Number Of Sites
1
Number Of Participants
2

Sites

Site Name
Stichting Radboud universitair medisch centrum
Department Name
MDL
Principal Investigator Name
Eric Tjwa
Principal Investigator Email
Eric.Tjwa@radboudumc.nl
Contact Person Name
Eric Tjwa
Contact Person Email
Eric.Tjwa@radboudumc.nl

Hungary

Earliest CTIS Part Ii Submission Date
22-05-2024
Latest Decision Or Authorization Date
15-07-2024
Processing Time Days
54
Number Of Sites
2
Number Of Participants
3

Sites

Site Name
Somogy Varmegyei Kaposi Mor Oktato Korhaz
Principal Investigator Name
Attila Haragh
Principal Investigator Email
haragatt@pazmanykabel.hu
Contact Person Name
Attila Haragh
Contact Person Email
haragatt@pazmanykabel.hu
Site Name
Semmelweis University
Department Name
I. sz. Sebeszeti es Intervencios Gasztroenterologiai Klinika
Principal Investigator Name
Krisztina Hagymasi
Principal Investigator Email
hagymasi.krisztina@med.semmelweis-univ.hu
Contact Person Name
Krisztina Hagymasi

Austria

Earliest CTIS Part Ii Submission Date
01-07-2024
Latest Decision Or Authorization Date
16-07-2024
Processing Time Days
15
Number Of Sites
1
Number Of Participants
1

Sites

Site Name
Klinikum Wels-Grieskirchen GmbH
Department Name
Department of Internal Medicine I, Klinikum Wels-Grieskirchen
Principal Investigator Name
Harald Hofer
Principal Investigator Email
harald.hofer@klinikum-wegr.at
Contact Person Name
Harald Hofer
Contact Person Email
harald.hofer@klinikum-wegr.at

Poland

Earliest CTIS Part Ii Submission Date
22-05-2024
Latest Decision Or Authorization Date
15-07-2024
Processing Time Days
54
Number Of Sites
2
Number Of Participants
11

Sites

Site Name
Uniwersyteckie Centrum Kliniczne Im. Prof. K. Gibinskiego Slaskiego Uniwersytetu Medycznego W Katowicach
Department Name
Oddział Gastroenterologii i Hepatologii
Principal Investigator Name
Agata Bacik
Principal Investigator Email
agatapl@poczta.fm
Contact Person Name
Agata Bacik
Contact Person Email
agatapl@poczta.fm
Site Name
ID Clinic Arkadiusz Pisula
Department Name
Investigator
Principal Investigator Name
Ewa Janczewska
Principal Investigator Email
e.janczewska@poczta.fm
Contact Person Name
Ewa Janczewska
Contact Person Email
e.janczewska@poczta.fm

Spain

Earliest CTIS Part Ii Submission Date
23-05-2024
Latest Decision Or Authorization Date
15-07-2024
Processing Time Days
53
Number Of Sites
7
Number Of Participants
13

Sites

Site Name
Hospital Universitario 12 De Octubre
Department Name
Hepatology Department
Principal Investigator Name
Elena Gomez Dominguez
Principal Investigator Email
elenagodo@hotmail.com
Contact Person Name
Elena Gomez Dominguez
Contact Person Email
elenagodo@hotmail.com
Site Name
Hospital Universitari Vall D Hebron
Department Name
Liver Unit-Internla Medicine department
Principal Investigator Name
Ares Villagrasa Vilella
Principal Investigator Email
aresaurora.villagrasa@vallhebron.cat
Contact Person Name
Ares Villagrasa Vilella
Site Name
Hospital Germans Trias I Pujol
Department Name
Servicio de Gastroenterología.
Principal Investigator Name
Rosa Maria Morillas
Principal Investigator Email
rmorillas.germanstrias@gencat.cat
Contact Person Name
Rosa Maria Morillas
Site Name
Hospital Universitario La Paz
Department Name
Servicio de Digestivo
Principal Investigator Name
Pilar Castillo Grau
Principal Investigator Email
pcastillograu@gmail.com
Contact Person Name
Pilar Castillo Grau
Contact Person Email
pcastillograu@gmail.com
Site Name
Hospital Universitario Marques De Valdecilla
Department Name
Gastroenteroloy and Hepatology department
Principal Investigator Name
Alvaro Diaz Gonzalez
Principal Investigator Email
alvaro.diaz@scsalud.es
Contact Person Name
Alvaro Diaz Gonzalez
Contact Person Email
alvaro.diaz@scsalud.es
Site Name
Hospital Universitario Virgen De La Victoria
Department Name
Gastroenterology and Hepatology Department
Principal Investigator Name
Raul Jesus Andrade Bellido
Principal Investigator Email
andrade@uma.es
Contact Person Name
Raul Jesus Andrade Bellido
Contact Person Email
andrade@uma.es
Site Name
Hospital Clinic De Barcelona
Department Name
Liver Unit
Principal Investigator Name
Maria Carlota Londoño Hurtado
Principal Investigator Email
mlondono@clinic.cat
Contact Person Name
Maria Carlota Londoño Hurtado
Contact Person Email
mlondono@clinic.cat

Belgium

Earliest CTIS Part Ii Submission Date
23-05-2024
Latest Decision Or Authorization Date
11-07-2024
Processing Time Days
49
Number Of Sites
2
Number Of Participants
6

Sites

Site Name
UZ Leuven
Department Name
Gastroenterology & Hepatology
Principal Investigator Name
Jef Verbeek
Principal Investigator Email
jef.verbeek@uzleuven.be
Contact Person Name
Jef Verbeek
Contact Person Email
jef.verbeek@uzleuven.be
Site Name
Universitair Ziekenhuis Gent
Department Name
Hepatology & Gastroenterology
Principal Investigator Name
Xavier Verhelst
Principal Investigator Email
xavier.verhelst@uzgent.be
Contact Person Name
Xavier Verhelst
Contact Person Email
xavier.verhelst@uzgent.be

France

Earliest CTIS Part Ii Submission Date
22-05-2024
Latest Decision Or Authorization Date
16-07-2024
Processing Time Days
55
Number Of Sites
2
Number Of Participants
3

Sites

Site Name
Assistance Publique Hopitaux De Paris
Department Name
HepatoGastroenterology Department
Principal Investigator Name
Christophe CORPECHOT
Principal Investigator Email
christophe.corpechot@aphp.fr
Contact Person Name
Christophe CORPECHOT
Contact Person Email
christophe.corpechot@aphp.fr
Site Name
Hospices Civils De Lyon
Department Name
HepatoGastroenterology Department
Principal Investigator Name
Domitille Erard
Principal Investigator Email
domitille.erard@chu-lyon.fr
Contact Person Name
Domitille Erard
Contact Person Email
domitille.erard@chu-lyon.fr

Greece

Earliest CTIS Part Ii Submission Date
23-05-2024
Latest Decision Or Authorization Date
29-07-2024
Processing Time Days
67
Number Of Sites
1
Number Of Participants
5

Sites

Site Name
General University Hospital Of Larissa
Department Name
Department of Medicine and Reasearch Laboratory of Internal Medicine
Principal Investigator Name
George Dalekos
Principal Investigator Email
georgedalekos@gmail.com
Contact Person Name
George Dalekos
Contact Person Email
georgedalekos@gmail.com

Czechia

Earliest CTIS Part Ii Submission Date
24-05-2024
Latest Decision Or Authorization Date
15-07-2024
Processing Time Days
52
Number Of Sites
1
Number Of Participants
1

Sites

Site Name
Fakultni Nemocnice Ostrava
Department Name
Interní a kardiologická klinika - Oddělení gastroenterologie, hepatologie a pankreatologie
Principal Investigator Name
Adam Vašura
Principal Investigator Email
adam.vasura@fno.cz
Contact Person Name
Adam Vašura
Contact Person Email
adam.vasura@fno.cz

Italy

Earliest CTIS Part Ii Submission Date
24-05-2024
Latest Decision Or Authorization Date
16-07-2024
Processing Time Days
53
Number Of Sites
3
Number Of Participants
7

Sites

Site Name
Azienda Ospedaliera Universitaria Policlinico Paolo Giaccone
Department Name
Sezione Di Endocrinologia - Dipartimento Di Oncologia Sperimentale Ed Applicazioni Cliniche (dosac)
Principal Investigator Name
Vincenza Calvaruso
Principal Investigator Email
vincenza.calvaruso@unipa.it
Contact Person Name
Vincenza Calvaruso
Contact Person Email
vincenza.calvaruso@unipa.it
Site Name
Azienda Ospedaliero Universitaria Di Modena
Department Name
Medicina Interna metabolica
Principal Investigator Name
Alessia Cavicchioli
Principal Investigator Email
alessia.cavicchioli.1990@gmail.com
Contact Person Name
Alessia Cavicchioli
Site Name
Fondazione IRCCS San Gerardo Dei Tintori
Department Name
Gastroenterologia
Principal Investigator Name
Pietro Invernizzi
Principal Investigator Email
pietro.invernizzi@unimib.it
Contact Person Name
Pietro Invernizzi
Contact Person Email
pietro.invernizzi@unimib.it

Romania

Earliest CTIS Part Ii Submission Date
22-05-2024
Latest Decision Or Authorization Date
16-07-2024
Processing Time Days
55
Number Of Sites
1
Number Of Participants
3

Sites

Site Name
Institutul Clinic Fundeni
Department Name
Gastroenterology III
Principal Investigator Name
Liliana-Simona Gheorghe
Principal Investigator Email
drlgheorghe@gmail.com
Contact Person Name
Liliana-Simona Gheorghe
Contact Person Email
drlgheorghe@gmail.com

Germany

Earliest CTIS Part Ii Submission Date
01-07-2024
Latest Decision Or Authorization Date
15-07-2024
Processing Time Days
14
Number Of Sites
6
Number Of Participants
12

Sites

Site Name
Charite Universitaetsmedizin Berlin KöR
Department Name
Medical Polyclinic
Principal Investigator Name
Ulrike Morgera
Principal Investigator Email
ulrike.morgera@charite.de
Contact Person Name
Ulrike Morgera
Contact Person Email
ulrike.morgera@charite.de
Site Name
ifi-Medizin GmbH
Department Name
Haus L
Principal Investigator Name
Peter Buggisch
Principal Investigator Email
Peter.Buggisch@amedes-group.com
Contact Person Name
Peter Buggisch
Site Name
Gastroenterologische Gemeinschaftspraxis Herne
Principal Investigator Name
Matthias Hinz
Principal Investigator Email
hinz@gastro-praxis-herne.de
Contact Person Name
Matthias Hinz
Contact Person Email
hinz@gastro-praxis-herne.de
Site Name
Universitaetsklinikum Tuebingen AöR
Department Name
Medizinische Klinik, Innere Medizin 1
Principal Investigator Name
Christoph Berg
Principal Investigator Email
christoph.berg@med.uni-tuebingen.de
Contact Person Name
Christoph Berg
Site Name
Universitaetsklinikum Erlangen AöR
Department Name
Gastroenterology, Pneumology and Endocrinology
Principal Investigator Name
Peter Dietrich
Principal Investigator Email
peter.dietrich@uk-erlangen.de
Contact Person Name
Peter Dietrich
Contact Person Email
peter.dietrich@uk-erlangen.de
Site Name
Gastroenterologic Hepatologic Center Kiel (Gastroenterologisch-Hepatologisches MVZ Kiel GmbH)
Department Name
Gastroenterologic Hepatologic Center Kiel
Principal Investigator Name
Holger Hinrichsen
Contact Person Name
Holger Hinrichsen

Sponsor

Primary sponsor

Full Name
Gilead Sciences Inc.
Organisation Type
Pharmaceutical company
Country Of Registered Address
United States

Contract research organisations

Name
PPD Global Ltd.
Responsibilities
codes: 1,5
Name
PPD Development LP
Responsibilities
codes: 1,10,11,12,14,2,5,8,9

Third parties

  • {"country":"United States","full_name":"Medidata Solutions Inc.","duties_or_roles":"EDC/RAVE and Patient Profiles/CSA","organisation_type":"Non-Pharmaceutical company"}
  • {"country":"United States","full_name":"Metabolon Inc.","duties_or_roles":"Biomarkers, exploratory measures, Sample receiving","organisation_type":"Pharmaceutical company"}
  • {"country":"Greece","full_name":"PPD Global Ltd.","duties_or_roles":"codes: 1,5","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Arup Laboratories Inc.","duties_or_roles":"Exploratory measures","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"FMD K And L Inc.","duties_or_roles":"Biostatistics","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Bioagilytix Labs LLC","duties_or_roles":"Analytical chemistry, Sample receiving","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Certara USA Inc.","duties_or_roles":"Pharmacology Analysis","organisation_type":"Pharmaceutical company"}
  • {"country":"Switzerland","full_name":"United BioSource (Suisse) S.A.","duties_or_roles":"Safety Database","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Pharma Start LLC","duties_or_roles":"Home Health Care","organisation_type":"Pharmaceutical company"}
  • {"country":"Germany","full_name":"MARKEN Germany GmbH","duties_or_roles":"Direct to Patient (DTP)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"PPD Development LP","duties_or_roles":"codes: 1,10,11,12,14,2,5,8,9","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Eresearchtechnology Inc.","duties_or_roles":"ePatient Reported Outcomes (ePRO)","organisation_type":"Pharmaceutical company"}
  • {"country":"United States","full_name":"Quest Diagnostics Nichols Institute Inc.","duties_or_roles":"Serology/Endocrinology","organisation_type":"Pharmaceutical company"}
  • {"country":"Belgium","full_name":"MEDPACE LABORATORIES","duties_or_roles":"Clinical chemistry, Clinical haematology, Serology/Endocrinology, Immunology","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Scout Clinical","duties_or_roles":"Patient Travel and Reimbursement","organisation_type":"Hospital/Clinic/Other health care facility"}
  • {"country":"United States","full_name":"Medpace Reference Laboratories LLC","duties_or_roles":"Clinical chemistry, Clinical haematology, Analytical chemistry","organisation_type":"Laboratory/Research/Testing facility"}
  • {"country":"United States","full_name":"Neogenomics Laboratories Inc.","duties_or_roles":"Biopsy slide preparation and staining","organisation_type":"Laboratory/Research/Testing facility"}

Investigational products

Investigational Product Name
MBX-8025 10 mg capsule
Active Substance
seladelpar
Modality
Small molecule
Routes Of Administration
ORAL
Route
ORAL
Authorisation Status
Authorised
Orphan Designation
Yes
Frequency
once daily
Maximum Dose
10 mg
Investigational Product Name
MBX-8025 5 mg capsule
Active Substance
seladelpar
Modality
Small molecule
Routes Of Administration
ORAL
Route
ORAL
Authorisation Status
Authorised
Orphan Designation
Yes
Frequency
once daily
Maximum Dose
10 mg

Related trials

Other published trials that may interest you.