Primary Biliary Cirrhosis (PBC)Phase 2NCT02609048
Study to Evaluate the Effects of Two Doses of Seladelpar (MBX-8025) in Subjects With Primary Biliary Cirrhosis (PBC)
A Phase 2 study testing Experimental: Seladelpar 50 mg and Experimental: Seladelpar / MBX-8025 200 mg in people with Primary Biliary Cirrhosis (PBC), run by Gilead Sciences at 49 sites in 5 countries.
Trial summary
The primary objective of this study is to evaluate the effect of seladelpar (MBX-8025) on alkaline phosphatase (AP) levels in participants with primary biliary cirrhosis (PBC). Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Primary Biliary Cirrhosis (PBC)
- What is being tested?
- Experimental: Seladelpar 50 mg and Experimental: Seladelpar / MBX-8025 200 mg
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 41 participants
- Where
- 49 sitesin 5 countries
- Study dates
- 4 Nov 2015to 1 Jul 2016
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Must have given written informed consent (signed and dated) and any authorizations required by local law
- 18 to 75 years old (inclusive)
- Male or female with a diagnosis of PBC, by at least two of the following criteria:
- History of AP above upper limit of normal (ULN) for at least six months
- Positive Anti-Mitochondrial Antibodies (AMA) titers (>1/40 on immunofluorescence or M2 positive by enzyme linked immunosorbent assay (ELISA) or positive PBC-specific antinuclear antibodies
- Documented liver biopsy result consistent with PBC
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- On a stable and recommended dose of UDCA for the past twelve months
- AP ≥ 1.67 × ULN
- For females of reproductive potential, use of at least one barrier contraceptive and a second effective birth control method during the study and for at least two weeks after the last dose. For male subjects, use of appropriate contraception (e.g., condoms), so their female partners of reproductive potential do not become pregnant during the study and for at least two weeks after the last dose
Who cannot join
- 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 on active treatment)
- Aspartate Aminotransferase (AST) or Alanine Aminotransferase (ALT) > 3 × ULN
- Total bilirubin > 2 × ULN
- Auto-immune hepatitis
- Primary sclerosing cholangitis
- Known history of alpha-1-Antitrypsin deficiency
Show all 15Show fewer
- Known history of chronic viral hepatitis
- Creatine kinase above ULN
- Serum creatinine above ULN
- For females, pregnancy or breast-feeding
- Use of colchicine, methotrexate, azathioprine, or systemic steroids in the two months preceding screening
- Current use of fibrates, including fenofibrates, or simvastatin
- Use of an experimental treatment for PBC
- Use of experimental or unapproved immunosuppressant
- 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
Trial locations and contacts
49 sites in 5 countries.
49 sites in 5 countries
United States21 sites
Norman Gitlin, MD
Atlanta, Georgia, 30308
Digestive Helathcare of Georgia
Atlanta, Georgia, 30309
Consultants for Clinical Research
Cincinnati, Ohio, 45249
Henry Ford Health System
Detroit, Michigan, 48202
University of Florida
Gainesville, Florida, 32610
CHI St. Luke's Health Baylor College of Medicine Medical Center - Advanced Liver Therapies
Houston, Texas, 77030
Indiana University Hospital - Clinical Research Center
Indianapolis, Indiana, 46202
Kansas City Gastroenterology and Hepatology
Kansas City, Missouri, 64131
Pinnacle Clinical Research
Live Oak, Texas, 78233
North Shore-Long Island Jewish Health System / Division of Gastroenterology
Manhasset, New York, 11030
University of Miami, Center for Liver Diseases
Miami, Florida, 33136
NYU Langone Medical Center
New York, New York, 10016
Icahn School of Medicine at Mount Sinai - The Mount Sinai Medical Center
New York, New York, 10029
Bon Secours St. Mary's Hospital of Richmond
Newport News, Virginia, 23602
Digestive and Liver Disease Specialists
Norfolk, Virginia, 23502
University of Nebraska Medical Center / The Nebraska Medical Center
Omaha, Nebraska, 68198
Mayo Clinic of Arizona
Phoenix, Arizona, 85054
University of California, Davis Medical Center
Sacramento, California, 95817
American Research Corporation at Texas Liver Institute
San Antonio, Texas, 78215
St. Louis University School of Medicine
St Louis, Missouri, 63110
Gastroenterology Associates of Western Michigan, PLC, d.b.a. West Michigan Clinical Research
Wyoming, Michigan, 49519
Germany15 sites
Charite Universitatsmedizin Berlin - Campus Mitte
Berlin, 10117
Leber- und Studienzentrum am Checkpoint
Berlin, 10969
Universitatsklinikum Bonn
Bonn, 53127
Universitatsklinikum Carl Gustav Carus an der TU Dresden
Dresden, 01307
University Hospital Erlangen
Erlangen, 91054
Universitatsklinikum Essen, Zentrum fur Innere Medizin
Essen, 45157
Ifi-Studien und Projekte GmbH, A.d. Asklepios Klinik St. Georg
Hamburg, 20099
Universitatsklinikum Hamburg-Eppendorf MARTINISTRASSE 52
Hamburg, 20246
Med. Hochschule Hannover, Klinik fur Gastroenterologie
Hanover, 30625
Medizinische Universitatsklinik
Heidelberg, 69120
Gastroenterologische Gemeinschaftspraxis Herne
Herne, 44623
UKSH, Campus Kiel, Klinik fur Allgemeine Innere Medizin 1
Kiel, 24105
UKSH, Campus Kiel
Kiel, 24105
Universitatsklinikum Leipzig AOR
Leipzig, 04103
Universitatsmedizin der Johannes Gutenberg - Universitat Mainz
Mainz, 55131
United Kingdom6 sites
University Hospital Birmingham NHS Foundation Trust
Birmingham, B15 2GW
Addenbrooke Hospital
Cambridge, CB2 0QQ
Hull and East Yorkshire NHS Trust
Hull, HU3 2jZ
The Newcastle upon Tyne Hospitals NHS Foundation Trust
Newcastle upon Tyne, NE1 4LP
Nottingham University Hospitals NHS Trust
Nottingham, NG7 2UH
Derriford Hospital
Plymouth, England, PL6 8DH
Poland5 sites
Wojewodzki Szpital Obserwacyjno-Zakazny im. Tadeusza w Bydgoszczy
Bydgoszcz, Kuyavian-Pomeranian Voivodeship, 85-030
SP CSK im. Prof. K. Gibinskiego SUM w Katowicach
Katowice, Silesian Voivodeship, 40-752
SPZOZ Szpital Uniwersytecki w Krakowie
Krakow, Malopolski, 31-531
ID Clinic Arkadiusz Pisula
Mysłowice, Silesian Voivodeship, 41-400
Centrum Onkologii-Instytut Im. Marii Sklodowskiej-Curie
Warsaw, Masovian Voivodeship, 02-781
Canada2 sites
University of Calgary Liver Unit (Heritage Medical Research Clinic)
Calgary, Alberta, T2N 4Z6
Toronto General Hospital
Toronto, Ontario, M5G 2C4
About this study
Official title: A 12-week, Double-blind, Randomized, Placebo-controlled, Phase 2 Study, to Evaluate the Effects of Two Doses of MBX-8025 in Subjects With Primary Biliary Cirrhosis (PBC) and an Inadequate Response to Ursodeoxycholic Acid (UDCA)
The primary objective of this study is to evaluate the effect of seladelpar (MBX-8025) on alkaline phosphatase (AP) levels in participants with primary biliary cirrhosis (PBC).
Read the detailed description
Primary:
To evaluate the effect of MBX-8025 on Alkaline Phosphatase (AP) levels
Secondary:
To evaluate the safety and tolerability of MBX-8025 in subjects with Primary Biliary Cirrhosis (PBC) To evaluate the effects of MBX-8025 on Primary Biliary Cirrhosis (PBC) response criteria To evaluate the effects of MBX-8025 on other markers of liver function, lipids, pruritus and Quality of Life (QoL)
- Conditions
- Primary Biliary Cirrhosis (PBC)
- Being tested
- Placebo Comparator, Experimental: Seladelpar 50 mg, Experimental: Seladelpar / MBX-8025 200 mg
- Keywords
- PBC
- Medical subject headings
- Liver Cirrhosis, Biliary
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Placebo ComparatorDrug
Also called Placebo
Placebo Capsule
Experimental: Seladelpar 50 mgDrug
Also called MBX-8025, Livdelzi®
Seladelpar 50 mg capsule
Experimental: Seladelpar / MBX-8025 200 mgDrug
Also called MBX-8025, Livdelzi®
Seladelpar 100 mg capsules
| Study group | Role | What they receive |
|---|---|---|
| Placebo Dose | Placebo comparator | Participants received 2 placebo-to-match (PTM) seladelpar capsules, orally, once daily for 12 weeks. Receives: Drug: Placebo Comparator |
| Seladelpar 50 mg Dose | Experimental | Participants received seladelpar 50 mg capsule (1 x 50 mg capsule) and a PTM seladelpar capsule, orally, once daily for 12 weeks. Receives: Drug: Placebo Comparator; Drug: Experimental: Seladelpar 50 mg |
| Seladelpar 200 mg Dose | Experimental | Participants received seladelpar 200 mg capsules (2 x 100 mg capsules), orally, once daily for 12 weeks. Receives: Drug: Experimental: Seladelpar / MBX-8025 200 mg |
What is being measured
The primary outcome is the main question the study is designed to answer.
Baseline Alkaline Phosphatase (AP) Levels
Baseline for AP level was defined as the mean between assessments at Visit 1 (Week -4 to Week 0) and Visit 2 (Week 0).
Measured: Baseline
Percent Change From Baseline in Alkaline Phosphatase (AP) Levels
Percent change in AP serum levels from Baseline to the end of treatment was analyzed. For missing postbaseline data, the last non-missing postbaseline value on treatment was carried forward, with missing assessments imputed by last observation carried forward (LOCF). Baseline for AP level was defined as the mean between assessments at Visit 1 (Week -4 to Week 0) and Visit 2 (Week 0).
Measured: Baseline, Week 12
Secondary outcomes (21)
Percentage of Participants With Response as Determined by Composite Endpoint of Alkaline Phosphatase and Total Bilirubin
Participants were defined as responders for the composite endpoint of AP and total bilirubin if their AP level was <1.67 × upper limit of normal (ULN) and had decreased at least 15% from their Baseline level and their total bilirubin value was within the normal range [0.1-1.1 milligrams/deciliter (mg/dL)].
Measured: Week 12
Percent Change From Baseline in Aspartate Aminotransferase (AST)
Percentage change in AST levels from Baseline to the end of treatment was analyzed. For missing postbaseline data, the last non-missing postbaseline value on treatment was carried forward, with missing assessments imputed by LOCF. Baseline for AST level was defined as the last non-missing assessments prior to or on the date of the first study dose.
Measured: Baseline, Week 12
Percent Change From Baseline in Alanine Aminotransferase (ALT)
Percentage change in ALT levels from Baseline to the end of treatment was analyzed. For missing postbaseline data, the last non-missing postbaseline value on treatment was carried forward, with missing assessments imputed by LOCF. Baseline for ALT level was defined as the last non-missing assessments prior to or on the date of the first study dose.
Measured: Baseline, Week 12
Percent Change From Baseline in Gamma-glutamyl Transferase (GGT)
Percentage change in GGT levels from Baseline to the end of treatment was analyzed. For missing postbaseline data, the last non-missing postbaseline value on treatment was carried forward, with missing assessments imputed by LOCF. Baseline for GGT level was defined as the last non-missing assessments prior to or on the date of the first study dose.
Measured: Baseline, Week 12
Percent Change From Baseline in 5'Nucleotidase (5NT)
Percentage change in 5'Nucleotidase levels from Baseline to the end of treatment was analyzed. For missing postbaseline data, the last non-missing postbaseline value on treatment was carried forward, with missing assessments imputed by LOCF. Baseline for 5NT level was defined as the last non-missing assessments prior to or on the date of the first study dose.
Measured: Baseline, Week 12
Percent Change From Baseline in Total Bilirubin
Percentage change in total bilirubin levels from Baseline to the end of treatment was analyzed. For missing postbaseline data, the last non-missing postbaseline value on treatment was carried forward, with missing assessments imputed by LOCF. Baseline for total bilirubin level was defined as the last non-missing assessments prior to or on the date of the first study dose.
Measured: Baseline, Week 12
Percent Change From Baseline in Conjugated Bilirubin
Percentage change in conjugated bilirubin levels from Baseline to the end of treatment was analyzed. For missing postbaseline data, the last non-missing postbaseline value on treatment was carried forward, with missing assessments imputed by LOCF. Baseline for conjugated bilirubin level was defined as the last non-missing assessments prior to or on the date of the first study dose.
Measured: Baseline, Week 12
Percent Change From Baseline in Unconjugated Bilirubin
Percentage change in unconjugated bilirubin levels from Baseline to the end of treatment was analyzed. For missing postbaseline data, the last non-missing postbaseline value on treatment was carried forward, with missing assessments imputed by LOCF. Baseline for unconjugated bilirubin level was defined as the last non-missing assessments prior to or on the date of the first study dose.
Measured: Baseline, Week 12
Percent Change From Baseline in Bone-specific AP Levels
Percentage change in bone-specific AP levels from Baseline to the end of treatment was analyzed. For missing postbaseline data, the last non-missing postbaseline value on treatment was carried forward, with missing assessments imputed by LOCF. Baseline for bone-specific level was defined as the last non-missing assessments prior to or on the date of the first study dose.
Measured: Baseline, Week 12
Percent Change From Baseline in Triglycerides (TG)
Percentage change in TG levels from Baseline to the end of treatment was analyzed. For missing postbaseline data, the last non-missing postbaseline value on treatment was carried forward, with missing assessments imputed by LOCF. Baseline for TG level was defined as the last non-missing assessments prior to or on the date of the first study dose.
Measured: Baseline, Week 12
Percent Change From Baseline in Total Cholesterol (TC)
Percentage change in TC levels from Baseline to the end of treatment was analyzed. For missing postbaseline data, the last non-missing postbaseline value on treatment was carried forward, with missing assessments imputed by LOCF. Baseline for TC level was defined as the last non-missing assessments prior to or on the date of the first study dose.
Measured: Baseline, Week 12
Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C)
Percentage change in HDL-C levels from Baseline to the end of treatment was analyzed. For missing postbaseline data, the last non-missing postbaseline value on treatment was carried forward, with missing assessments imputed by LOCF. Baseline for HDL-C level was defined as the last non-missing assessments prior to or on the date of the first study dose.
Measured: Baseline, Week 12
Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C)
Percentage change in LDL-C levels from Baseline to the end of treatment was analyzed. For missing postbaseline data, the last non-missing postbaseline value on treatment was carried forward, with missing assessments imputed by LOCF. Baseline for LDL-C level was defined as the last non-missing assessments prior to or on the date of the first study dose.
Measured: Baseline, Week 12
Number of Participants Who Were Non-responders as Determined by Published PBC Response Criteria (Paris I)
Published criteria for response to treatment in PBC included Paris I criteria. For Paris I, the criteria used to measure the PBC participants' response to study treatment was: AP ≤ 3x ULN and AST ≤ 2x ULN and Total Bilirubin ≤ 1 mg/dL. Participants who satisfied the criteria were defined as responders and those who did not were considered nonresponders if all the elements had non-missing assessments at that visit. The number of nonresponders were reported, the missing assessments were imputed by LOCF.
Measured: Week 12
Number of Participants Who Were Non-responders as Determined by Published PBC Response Criteria (Paris II)
Published criteria for response to treatment in PBC included Paris II criteria. For Paris II, the criteria used to measure the PBC participants' response to study treatment was: AP ≤ 1.5x ULN and AST ≤ 1.5x ULN and Total Bilirubin ≤ 1 mg/dL. Participants who satisfied the criteria were defined as responders and those who did not were considered nonresponders if all the elements had non-missing assessments at that visit. The number of nonresponders were reported, the missing assessments were imputed by LOCF.
Measured: Week 12
Number of Participants Who Were Non-responders as Determined by Published PBC Response Criteria (Toronto I)
Published criteria for response to treatment in PBC included Toronto I criteria. For Toronto I, the criteria used to measure the PBC participants' response to study treatment was: AP ≤ 1.67x ULN. Participants who satisfied the criteria were defined as responders and those who did not were considered nonresponders if all the elements had non-missing assessments at that visit. The number of nonresponders were reported, the missing assessments were imputed by LOCF.
Measured: Week 12
Number of Participants Who Were Non-responders as Determined by Published PBC Response Criteria (Toronto II)
Published criteria for response to treatment in PBC included Toronto II criteria. For Toronto II, the criteria used to measure the PBC participants' response to study treatment was: AP ≤ 1.76x ULN. Participants who satisfied the criteria were defined as responders and those who did not were considered nonresponders if all the elements had non-missing assessments at that visit. The number of nonresponders were reported, the missing assessments were imputed by LOCF.
Measured: Week 12
United Kingdom-Primary Biliary Cirrhosis/Cholangitis (UK-PBC) Risk Score
The UK-PBC Risk Score was used to estimate the risk that a PBC participant would develop liver failure that would require liver transplantation within 5, 10 or 15 years from diagnosis. The UK-PBC Risk Score calculation was adapted based on the AP assessment, transaminases (refers to the ALT, where available, otherwise the AST assessment) and total bilirubin assessment respectively at end of treatment divided by its upper limits of the corresponding normal ranges; "albumin" and "platelet" refer to the baseline values of these parameters divided by their corresponding lower limits of normal ranges. Missing assessments at end of treatment was imputed by last observation carried forward (LOCF). The UK-PBC risk scores for 5, 10 and 15 years was calculated for each treatment group
Measured: Week 12
Change From Baseline in 5-Dimension (5-D) Itch Scale Score
The 5-D itch scale was used to for the multidimensional quantification of pruritus over time. It assessed five aspects of itching: degree, duration, direction, disability, and distribution.The 5-D itch Scale is a measure of itching that has been validated in patients with chronic pruritus to detect changes over time. A 5-D itch scale score typically ranges from 5 to 25 with a higher score indicating greater itch severity, where 5 represents no pruritus (itching) and 25 represents the most severe pruritus; Each dimension is scored separately, and the scores are added together to get a total 5-D itch score. Higher scores indicate worsening of itching. The total 5-D itch Scale score change from Baseline was calculated. Baseline for 5-D itch scale was defined as the last non-missing assessments prior to or on the date of the first study dose. Missing assessments at end of treatment was imputed by LOCF.
Measured: Baseline, Week 12
Change From Baseline in Pruritus Visual Analog Scale (VAS) Score
VAS was used to measure pruritis in participants with PBC. Participants placed a mark representing their level of itching since the previous measurement on a 100-mm VAS with 0 indicating no itching and 100 mm indicating the worst possible itching. Higher scores indicated worsening of itching The change from Baseline was presented by treatment group. Baseline for VAS score was defined as the last non-missing assessments prior to or on the date of the first study dose. Missing assessments at end of treatment was imputed by LOCF.
Measured: Baseline, Week 12
Change From Baseline in PBC-40 Quality of Life Questionnaire
The PBC-40 questionnaire was used to evaluate health-related quality of life measures, specifically fatigue. The PBC-40 questionnaire is a disease-specific tool developed to specifically measure the psychometric profile of PBC patients. It consists of 40 items divided into the six domains relevant to PBC including Cognitive, Social, EmotionalFunction, Fatigue, Itch, and Other Symptoms/General Questions. Items are scored from 1 to 5 and individual items scores are summed to give a total domain score. PBC 40 QoL domain score ranges are:Cognitive (6-30), Emotional Function (3-15),Fatigue (11-55), Itch (3-15),Social (10-50),Symptoms (7-35).Higher scores represent high impact and lower scores low impact of PBC on quality of life.The change and percentage change from Baseline for individual domain score at end of treatment were reported. Baseline for PBC-40 questionnaire was the last non-missing assessments prior to or on the date of the first study dose.Missing assessments imputed by LOCF.
Measured: Baseline, Week 12
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Nov 2015 (estimated)
- Study start4 Nov 2015
- Primary completion1 Jul 2016
- Study completion1 Jul 2016
Why it stopped: Study stopped early after review of safety and efficacy demonstrated efficacy proof-of-concept and need for dose reduction.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Primary Biliary Cirrhosis (PBC)Studies of the same condition, open and under way.
- More trials run by Gilead SciencesThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 41 participants (actual)
Sponsor and investigators
- Lead sponsor
- Gilead Sciences
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Pol F Boudes, M.D., Study chair, Gilead Sciences
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02609048
- Sponsor's protocol ID
- CB8025-21528
- Other IDs
- 2015-002698-39, 2015
- Status last verified
- Jan 2025
- First submitted
- 13 Nov 2015
- Last updated
- 25 Feb 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Publications
Jones D, Boudes PF, Swain MG, Bowlus CL, Galambos MR, Bacon BR, Doerffel Y, Gitlin N, Gordon SC, Odin JA, Sheridan D, Worns MA, Clark V, Corless L, Hartmann H, Jonas ME, Kremer AE, Mells GF, Buggisch P, Freilich BL, Levy C, Vierling JM, Bernstein DE, Hartleb M, Janczewska E, Rochling F, Shah H, Shiffman ML, Smith JH, Choi YJ, Steinberg A, Varga M, Chera H, Martin R, McWherter CA, Hirschfield GM. Seladelpar (MBX-8025), a selective PPAR-delta agonist, in patients with primary biliary cholangitis with an inadequate response to ursodeoxycholic acid: a double-blind, randomised, placebo-controlled, phase 2, proof-of-concept study. Lancet Gastroenterol Hepatol. 2017 Oct;2(10):716-726. doi: 10.1016/S2468-1253(17)30246-7. Epub 2017 Aug 14.
PubMed 28818518
Study at a glance
From the registry record, last updated by the sponsor on 25 Feb 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A 12-week, Double-blind, Randomized, Placebo-controlled, Phase 2 Study, to Evaluate the Effects of Two Doses of MBX-8025 in Subjects With Primary Biliary Cirrhosis (PBC) and an Inadequate Response to Ursodeoxycholic Acid (UDCA)
- ClinicalTrials.gov ID
- NCT02609048
- Acronym / sponsor ID
- CB8025-21528
- Conditions
- Primary Biliary Cirrhosis (PBC)
- Keywords
- PBC
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Study stopped early after review of safety and efficacy demonstrated efficacy proof-of-concept and need for dose reduction.
- Enrolment
- 41 participants (actual)
- Lead sponsor
- Gilead SciencesIndustry (pharma, biotech, devices)
- Study start
- 4 Nov 2015 (actual)
- Primary completion
- 1 Jul 2016 (actual)
- Study completion
- 1 Jul 2016 (actual)
- First posted
- 20 Nov 2015 (estimated)
- Results
- Posted 25 Feb 2025
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of this study is to evaluate the effect of seladelpar (MBX-8025) on alkaline phosphatase (AP) levels in participants with primary biliary cirrhosis (PBC).
Detailed description
Primary:
To evaluate the effect of MBX-8025 on Alkaline Phosphatase (AP) levels
Secondary:
To evaluate the safety and tolerability of MBX-8025 in subjects with Primary Biliary Cirrhosis (PBC) To evaluate the effects of MBX-8025 on Primary Biliary Cirrhosis (PBC) response criteria To evaluate the effects of MBX-8025 on other markers of liver function, lipids, pruritus and Quality of Life (QoL)
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Placebo Dose | Placebo comparator | Drug: Placebo ComparatorParticipants received 2 placebo-to-match (PTM) seladelpar capsules, orally, once daily for 12 weeks. |
| Seladelpar 50 mg Dose | Experimental | Drug: Placebo Comparator, Drug: Experimental: Seladelpar 50 mgParticipants received seladelpar 50 mg capsule (1 x 50 mg capsule) and a PTM seladelpar capsule, orally, once daily for 12 weeks. |
| Seladelpar 200 mg Dose | Experimental | Drug: Experimental: Seladelpar / MBX-8025 200 mgParticipants received seladelpar 200 mg capsules (2 x 100 mg capsules), orally, once daily for 12 weeks. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (9 listed)
- Must have given written informed consent (signed and dated) and any authorizations required by local law
- 18 to 75 years old (inclusive)
- Male or female with a diagnosis of PBC, by at least two of the following criteria:
- On a stable and recommended dose of UDCA for the past twelve months
- AP ≥ 1.67 × ULN
Key exclusion criteria (15 listed)
- 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 on active treatment)
- Aspartate Aminotransferase (AST) or Alanine Aminotransferase (ALT) > 3 × ULN
- Total bilirubin > 2 × ULN
- Auto-immune hepatitis
- Primary sclerosing cholangitis
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Placebo ComparatorAlso: PlaceboPlacebo Capsule | Drug | Placebo Dose, Seladelpar 50 mg Dose |
| Experimental: Seladelpar 50 mgAlso: MBX-8025, Livdelzi®Seladelpar 50 mg capsule | Drug | Seladelpar 50 mg Dose |
| Experimental: Seladelpar / MBX-8025 200 mgAlso: MBX-8025, Livdelzi®Seladelpar 100 mg capsules | Drug | Seladelpar 200 mg Dose |
Primary outcome
Baseline Alkaline Phosphatase (AP) Levels
Time frame: Baseline
Baseline for AP level was defined as the mean between assessments at Visit 1 (Week -4 to Week 0) and Visit 2 (Week 0).
Percent Change From Baseline in Alkaline Phosphatase (AP) Levels
Time frame: Baseline, Week 12
Percent change in AP serum levels from Baseline to the end of treatment was analyzed. For missing postbaseline data, the last non-missing postbaseline value on treatment was carried forward, with missing assessments imputed by last observation carried forward (LOCF). Baseline for AP level was defined as the mean between assessments at Visit 1 (Week -4 to Week 0) and Visit 2 (Week 0).
21 secondary outcomes
Percentage of Participants With Response as Determined by Composite Endpoint of Alkaline Phosphatase and Total Bilirubin
Time frame: Week 12
Percent Change From Baseline in Aspartate Aminotransferase (AST)
Time frame: Baseline, Week 12
Percent Change From Baseline in Alanine Aminotransferase (ALT)
Time frame: Baseline, Week 12
Percent Change From Baseline in Gamma-glutamyl Transferase (GGT)
Time frame: Baseline, Week 12
Percent Change From Baseline in 5'Nucleotidase (5NT)
Time frame: Baseline, Week 12
Percent Change From Baseline in Total Bilirubin
Time frame: Baseline, Week 12
Percent Change From Baseline in Conjugated Bilirubin
Time frame: Baseline, Week 12
Percent Change From Baseline in Unconjugated Bilirubin
Time frame: Baseline, Week 12
Percent Change From Baseline in Bone-specific AP Levels
Time frame: Baseline, Week 12
Percent Change From Baseline in Triglycerides (TG)
Time frame: Baseline, Week 12
Percent Change From Baseline in Total Cholesterol (TC)
Time frame: Baseline, Week 12
Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C)
Time frame: Baseline, Week 12
Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C)
Time frame: Baseline, Week 12
Number of Participants Who Were Non-responders as Determined by Published PBC Response Criteria (Paris I)
Time frame: Week 12
Number of Participants Who Were Non-responders as Determined by Published PBC Response Criteria (Paris II)
Time frame: Week 12
Number of Participants Who Were Non-responders as Determined by Published PBC Response Criteria (Toronto I)
Time frame: Week 12
Number of Participants Who Were Non-responders as Determined by Published PBC Response Criteria (Toronto II)
Time frame: Week 12
United Kingdom-Primary Biliary Cirrhosis/Cholangitis (UK-PBC) Risk Score
Time frame: Week 12
Change From Baseline in 5-Dimension (5-D) Itch Scale Score
Time frame: Baseline, Week 12
Change From Baseline in Pruritus Visual Analog Scale (VAS) Score
Time frame: Baseline, Week 12
Change From Baseline in PBC-40 Quality of Life Questionnaire
Time frame: Baseline, Week 12
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Feb 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 28818518Jones D, Boudes PF, Swain MG, Bowlus CL, Galambos MR, Bacon BR, Doerffel Y, Gitlin N, Gordon SC, Odin JA, Sheridan D, Worns MA, Clark V, Corless L, Hartmann H, Jonas ME, Kremer AE, Mells GF, Buggisch P, Freilich BL, Levy C, Vierling JM, Bernstein DE, Hartleb M, Janczewska E, Rochling F, Shah H, Shiffman ML, Smith JH, Choi YJ, Steinberg A, Varga M, Chera H, Martin R, McWherter CA, Hirschfield GM. Seladelpar (MBX-8025), a selective PPAR-delta agonist, in patients with primary biliary cholangitis with an inadequate response to ursodeoxycholic acid: a double-blind, randomised, placebo-controlled, phase 2, proof-of-concept study. Lancet Gastroenterol Hepatol. 2017 Oct;2(10):716-726. doi: 10.1016/S2468-1253(17)30246-7. Epub 2017 Aug 14.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Pol F Boudes, M.D. study chair
Gilead Sciences
49 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Experimental: Seladelpar 50 mg and Experimental: Seladelpar / MBX-8025 200 mg in people with Primary Biliary Cirrhosis (PBC), run by Gilead Sciences at 49 sites in 5 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 9 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 25 Feb 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 49 sites in United States, Germany, United Kingdom, Poland, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 25 Feb 2025). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Baseline Alkaline Phosphatase (AP) Levels (time frame: Baseline). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02609048, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 Feb 2025.