Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
An 8-week, dose ranging, open label, randomized, Phase 2 study with a 44-week extension, to evaluate the safety and efficacy of MBX-8025 in subjects with Primary Biliary Cholangitis (PBC) and an inadequate response to or intolerance to ursodeoxycholic acid (UDCA)
Primary:
To evaluate the safety and efficacy of MBX-8025 2 mg, 5 mg, and 10 mg over 8 weeks of treatment
Secondary:
To evaluate the safety and efficacy of MBX-8025 2 mg, 5 mg, and 10 mg over 12 and 26 weeks of treatment
To evaluate the safety and efficacy of MBX-8025 2 mg, 5 mg, and 10 mg over 52 weeks of treatment
To evaluate the pharmacokinetics (PK) of MBX-8025
Exploratory:
To evaluate the effect of MBX-8025 on bile acids, additional markers of inflammation and renal function
MBX-8025 doses of 1 mg and 15 mg may be evaluated if dose adjustment occurs
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| MBX-8025 (2 mg) | Experimental | MBX-8025 2 mg capsule once daily |
|
| MBX-8025 (5 mg) | Experimental | MBX-8025 5 mg capsule once daily |
|
| MBX-8025 (10 mg) | Experimental | MBX-8025 10 mg capsule once daily |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| MBX-8025 2 mg Capsule | Drug | Initial 8-week treatment: • MBX-8025 2 mg Extension: The 2 mg group will be started after safety and efficacy review of the 5 mg and the 10 mg groups has been completed. Subjects will initially enter the extension on their assigned dose. The dose might be up- or down-titrated after safety and efficacy data review of the first 8 weeks of treatment. During the extension, a subject's dose might be re-adjusted for safety or efficacy reasons. |
| Measure | Description | Time Frame |
|---|---|---|
| Relative Change From Baseline in Serum Alkaline Phosphatase (ALP) at Week 8 | Relative change from baseline in serum ALP levels at Week 8 (endpoint). The modified Intent-to-Treat (mITT) analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. n, denotes number of subjects evaluable for the respective timepoints | 8 weeks |
| Measure | Description | Time Frame |
|---|---|---|
| Absolute Change From Baseline in Serum Alkaline Phosphatase (ALP) at Week 12 and Week 52 | Absolute change in ALP from baseline to Weeks 12 and 52 The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | 12 weeks and 52 weeks |
Not provided
Inclusion Criteria:
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 or intolerant to UDCA
AP ≥ 1.67 × ULN
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:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Institute for Liver Health | Chandler | Arizona | 85224 | United States | ||
| Southern California Research Center |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 35367282 | Derived | Bowlus CL, Galambos MR, Aspinall RJ, Hirschfield GM, Jones DEJ, Dorffel Y, Gordon SC, Harrison SA, Kremer AE, Mayo MJ, Thuluvath PJ, Levy C, Swain MG, Neff GW, Sheridan DA, Stanca CM, Berg CP, Goel A, Shiffman ML, Vierling JM, Boudes P, Steinberg A, Choi YJ, McWherter CA. A phase II, randomized, open-label, 52-week study of seladelpar in patients with primary biliary cholangitis. J Hepatol. 2022 Aug;77(2):353-364. doi: 10.1016/j.jhep.2022.02.033. Epub 2022 Mar 30. |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
A total of 192 subjects were screened of which 121 subjects randomized (2 subjects were not treated) into the study and 71 were screen failures. Subjects were randomized to the 5 and 10 mg treatment groups for entry to the 8-week initial treatment period study, while those in the 2 mg treatment group entered after being sequentially assigned their dose
Not provided
Not provided
| ID | Title | Description |
|---|---|---|
| FG000 | MBX-8025 (2 mg) | MBX-8025 2 mg capsule once daily MBX-8025 2 mg Capsule: Initial 8-week treatment: • MBX-8025 2 mg Subjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg. |
| FG001 |
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
Not provided
| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Jul 20, 2017 | Mar 21, 2022 |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
|
|
| MBX-8025 5 mg Capsule | Drug | Initial 8-week treatment: • MBX-8025 5 mg Extension: Subjects will initially enter the extension on their assigned dose. The dose might be up- or down-titrated after safety and efficacy data review of the first 8 weeks of treatment. During the extension, a subject's dose might be re-adjusted for safety or efficacy reasons. |
|
|
| MBX-8025 10 mg Capsule | Drug | Initial 8-week treatment: • MBX-8025 10 mg Extension: Subjects will initially enter the extension on their assigned dose. The dose might be up- or down-titrated after safety and efficacy data review of the first 8 weeks of treatment. During the extension, a subject's dose might be re-adjusted for safety or efficacy reasons. |
|
|
| Change in Aspartate Aminotransferase (AST) From Baseline to 12 Weeks and 52 Weeks | Change from baseline in AST levels at endpoint was reported. The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | 12 weeks and 52 weeks |
| Change in Alanine Aminotransferase (ALT) From Baseline to 12 Weeks and 52 Weeks | Change from baseline in ALT levels at endpoint was reported. The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | 12 weeks and 52 weeks |
| Change in Gamma-glutamyl Transferase (GGT) From Baseline to 12 Weeks and 52 Weeks | Change from baseline in GGT levels at endpoint was reported. The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | 12 weeks and 52 weeks |
| Change in Bilirubin - Total Bilirubin (TB) From Baseline to 12 Weeks and 52 Weeks | Change from baseline in TB levels at endpoint is being reported. The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | 12 weeks and 52 weeks |
| Percentage of Participants Meet Composite Endpoint Criteria of ALP and Total Bilirubin | Participant meets composite endpoint is defined by participant meets all of the following criteria:
Endpoint of Alkaline Phosphatase and Total Bilirubin by Visit (mITT Population) | 12 Weeks and 52 Weeks |
| Percentage of Participants Meet Published PBC Response Criteria - Paris I | Percentage of participants with response based on Paris I risk score was defined as ALP less than or equal to (≤) 3x ULN and aspartate aminotransferase (AST) less than or equal to (≤) 2 x ULN and Total Bilirubin ≤ 1 mg/dL. The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | 12 weeks and 52 weeks |
| Percentage of Participants Meet Published PBC Response Criteria - Paris II | Percentage of participants with response based on Paris II risk score was defined as ALP≤1.5xULN and AST≤1.5xULN and Total Bilirubin ≤ 1 mg/dL. The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | 12 weeks and 52 weeks |
| Percentage of Participants Meet Published PBC Response Criteria - Toronto I | Percentage of participants with response based on Toronto I risk score defined as ALP ≤ 1.67 x ULN. The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | 12 weeks and 52 weeks |
| UK-PBC Risk Score Value | The UK-PBC Risk Score at endpoint is defined by the mean percentage risk that a PBC patient treated with ursodeoxycholic acid (UDCA) would develop liver failure requiring liver transplantation in 5, 10 and 15 years from diagnosis. The higher the score might indicate higher risk to death or live transplantation. Formula used for UK-PBC risk score = 1- 0.982 ^EXP(0.0287854*(ALP12 x ULN-1.722136304) - 0.0422873*(((TA12 xULN/10)^-1) - 8.675729006) + 1.4199 * (LN(BIL12 x ULN/10)+2.709607778)-1.960303*(Albumin x LLN-1.17673001)-0.4161954*(Platelet x LLN-1.873564875)). Where, Baseline survivor function = 0.982, 0.941, and 0.893 for 5 years, 10 years and 15 years respectively. ALP12, TA12 and BIL12 refers to the ALP, transaminases (ALT, AST), and total bilirubin assessments, respectively. The modified Intent-to-Treat (mITT) analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | 12 weeks and 52 weeks |
| Change From Baseline in Pruritus Visual Analog Score (VAS) at Week 12 and Week 52 | VAS is the commonly used graphic tool for self-reporting of pruritus intensity in patients. VAS is a simple to use, validated, reliable and widely applicable tool that does not determine the impact of pruritus to quality of life. It comprises of a 100-mm horizontal line labelled as "no symptom" on left end and "worst imaginable symptom" on right end. Based on the intensity of the itch patient is instructed to draw a vertical line on the horizontal scale having a range [VAS values (unit: mm) ranging from 0 to 100, where 0 represents "no itching" and 100 "worst possible itching"]. The modified Intent-to-Treat (mITT) analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | 12 weeks and 52 weeks |
| Change From Baseline in PBC-40 Quality of Life (QoL) at Week 12 and Week 52 | The PBC-40 QoL questionnaire is a disease-specific health-related tool developed for measuring the psychometric profile in PBC patients. It has 10 domains and 43 questions relevant to PBC, including Cognitive, Social, Emotional Function, Fatigue, Itch, and Other Symptoms. Questions in domains: 1) digestion and diet (questions 1-3); 2) experiences (questions 4-7); 3) itching (questions 8-10); 4) fatigue (questions 11-18); 5) effort and planning (questions 19-21); 6) memory and concentration (questions 22-27); 7) affects to you as person (questions 28-33); 8) affects to your social life (questions 34-37); 9) overall impact on your life (questions 38-40); 10) general health and well-being (questions A-C). Within a domain, items are scored from 1 to 5 and the individual item scores are summed to give a total domain score. High scores represent high impact and low scores low impact of PBC on QoL (mITT Population). | 12 weeks and 52 weeks |
| Percentage of Participants Meet Published PBC Response Criteria - Barcelona | Percentage of participants with response based on Barcelona risk scores was defined as Normalization of ALP or a Decrease of ALP ≥ 40%. The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | 12 weeks and 52 weeks |
| Absolute Change in MELD Score From Baseline to 12 Weeks and 52 Weeks | Change from baseline to 12 weeks and 52 weeks in Model for End-stage Liver Disease (MELD) Score (mITT Population) The MELD score ranges from 6 to 40 and is a measure of how severe a patient's liver disease is. The higher the score, the more likely the patients will need a liver transplant. A calculated prognostic risk factor used to assess the potential need for a liver transplant. MELD(i) score = 10*[0.957*ln(creatinine mg/dL) + 0. 378*ln(total bilirubin mg/dL) + 1.120*ln (INR) + 0.643]. If MELD(i) is less than or equal to 11 then MELD = MELD(i). If MELD(i) is greater than 11 then MELD = MELD(i) + (1.32 *(137 - (Na)) - (0.033*MELD(i)*(137 - Na)) | 12 weeks and 52 weeks |
| Change in GLOBE PBC Score From Baseline to 12 Weeks and 52 Weeks | Change from Baseline to 12 weeks and 52 weeks in Global PBC Study Group (GLOBE) score (mITT Population) The GLOBE score is a validated risk assessment tool providing an estimate of transplant-free survival for patients with PBC. It was developed by the Global PBC Study Group using Cox regression model on over 4,000 patients with PBC. Lower GLOBE score predicts lower risk. It is calculated from the following equation: GLOBE score = (0.044378 * age + 0.93982 * LN(total bilirubin/ULN) +(0.335648 * LN(alkaline phosphatase/ULN)) - 2.266708 * albumin /LLN -0.002581 * platelet count per 109/L) + 1.216865 | 12 weeks and 52 weeks |
| Participants Meet Rotterdam Criteria | participants with Response Based on Rotterdam Criteria at Weeks 12 and 52 Rotterdam Published PBC Response Criteria by Visit (mITT Population) Rotterdam criteria: Early (normal total bilirubin and normal albumin), Moderately advanced (either abnormal albumin or abnormal total bilirubin), and Advanced (both abnormal albumin and abnormal total bilirubin). From Early stage to Moderate Stage and to Advanced Stage, it becomes worse and worse in abnormality. | 12 weeks and 52 weeks |
| Percentage of Participants Meet Composite Endpoint of AP and Total Bilirubin Criteria at Week 12 and Week 52 | Percentage of participants with Response Defined by Composite Endpoint (ALP< 1.67 * Upper Limit of Normal [ULN] at Endpoint, Total Bilirubin [BIL] within Normal Limits at Endpoint, and Greater Than Equal To [≥] 15% ALP Reduction) from Baseline to Week 12 and Week 52 The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | 12 weeks and 52 weeks |
| Percent Change in Serum Alkaline Phosphatase (ALP) | Percent change in ALP from baseline to Weeks 12 and 52 The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | 12 weeks and 52 weeks |
| Coronado |
| California |
| 92118 |
| United States |
| Standford University Medicine | Palo Alto | California | 94305 | United States |
| University of California, Davis Medical Center | Sacramento | California | 95817 | United States |
| Ventura Clinical Trials | Ventura | California | 93003 | United States |
| Florida Research Institute | Lakewood Rch | Florida | 34211 | United States |
| University of Miami - Center for Liver Diseases | Miami | Florida | 33136 | United States |
| Atlanta Gastroenterology Associates, LLC | Atlanta | Georgia | 30308 | United States |
| Digestive Healthcare of Georgia | Atlanta | Georgia | 30309 | United States |
| Northwestern University | Chicago | Illinois | 60611 | United States |
| Mercy Medical Center | Baltimore | Maryland | 21202 | United States |
| Henry Ford Health System | Novi | Michigan | 48377 | United States |
| University of Mississippi Medical Center | Jackson | Mississippi | 39216 | United States |
| Saint Louis University, Gastroenterology & Hepatology | St Louis | Missouri | 63104 | United States |
| Northwell Health - Center for Liver Disease and Transplantation | Manhasset | New York | 11030 | United States |
| NYU Langone Medical Center | New York | New York | 10016 | United States |
| The Mount Sinai Medical Center | New York | New York | 10029 | United States |
| Northest Clinical Research Center, LLC. | Bethlehem | Pennsylvania | 18017 | United States |
| UT Southwestern Medical Center Investigation Drug Service | Dallas | Texas | 75390 | United States |
| Baylor College of Medicine | Houston | Texas | 77030 | United States |
| Gastroenterology Consultants of SA | Live Oak | Texas | 78233 | United States |
| Bon Secours St. Mary's Immaculate Hospital | Newport News | Virginia | 23602 | United States |
| University of Washington | Seattle | Washington | 98104 | United States |
| University of Calgary Liver Unit | Calgary | Alberta | T2N 4Z6 | Canada |
| Toronto Centre for Liver Disease | Toronto | Ontario | M5G 2C4 | Canada |
| Outpatient Clinic of Internal Medicine | Berlin | 10117 | Germany |
| University Hospital Erlangen | Erlangen | 91054 | Germany |
| Ifi-Studien und Projekte GmbH, An der Asklepios Klinik St. Georg | Hamburg | 20099 | Germany |
| Center of Internal Medicine - Medical School of Hannover | Hanover | 30625 | Germany |
| University Medical Centre of the Johannes Guttenberg-University | Mainz | 55131 | Germany |
| Universitatsklinikum Giessen und Marburg GmbH | Marburg | 35043 | Germany |
| Medizinische Universitatsklinik Tubingen | Tübingen | 72076 | Germany |
| University Hospitals Birmingham | Birmingham | B15 2GW | United Kingdom |
| Cambridge University Hospitals NHS Foundation Trust | Cambridge | CB2 0QQ | United Kingdom |
| Hull and East Yorkshire Hospitals NHS Trust | Hull | HU3 2JZ | United Kingdom |
| Royal Free London NHS Foundation Trust | London | NW3 2QR | United Kingdom |
| Plymouth Hospitals NHS Trust | Plymouth | PL6 8DH | United Kingdom |
| Portsmouth Hospitals NHS Trust | Portsmouth | PO6 3LY | United Kingdom |
| MBX-8025 (5 mg) |
MBX-8025 5 mg capsule once daily MBX-8025 5 mg Capsule: Initial 8-week treatment: • MBX-8025 5 mg Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment. |
| FG002 | MBX-8025 (10 mg) | MBX-8025 10 mg capsule once daily MBX-8025 10 mg Capsule: Initial 8-week treatment: • MBX-8025 10 mg Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period. |
| COMPLETED |
|
| NOT COMPLETED |
|
|
18 to 75 years of age (inclusive)
Not provided
| ID | Title | Description |
|---|---|---|
| BG000 | MBX-8025 (2 mg) | MBX-8025 2 mg capsule once daily MBX-8025 2 mg Capsule: Initial 8-week treatment: • MBX-8025 2 mg Subjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg. |
| BG001 | MBX-8025 (5 mg) | MBX-8025 5 mg capsule once daily MBX-8025 5 mg Capsule: Initial 8-week treatment: • MBX-8025 5 mg Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment. |
| BG002 | MBX-8025 (10 mg) | MBX-8025 10 mg capsule once daily MBX-8025 10 mg Capsule: Initial 8-week treatment: • MBX-8025 10 mg Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period. |
| BG003 | Total | Total of all reporting groups |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants |
| ||||||||||||||||
| Age, Continuous | Mean | Standard Deviation | years |
| |||||||||||||||
| Sex: Female, Male | Count of Participants | Participants |
| ||||||||||||||||
| Race/Ethnicity, Customized | Number | Subjects |
| ||||||||||||||||
| Region of Enrollment | Number | participants |
|
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Relative Change From Baseline in Serum Alkaline Phosphatase (ALP) at Week 8 | Relative change from baseline in serum ALP levels at Week 8 (endpoint). The modified Intent-to-Treat (mITT) analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. n, denotes number of subjects evaluable for the respective timepoints | mITT Population The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC. | Posted | Mean | Standard Deviation | percentage change from baseline | 8 weeks |
|
|
|
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change From Baseline in Serum Alkaline Phosphatase (ALP) at Week 12 and Week 52 | Absolute change in ALP from baseline to Weeks 12 and 52 The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | mITT Population: The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC. | Posted | Mean | Standard Deviation | U/L | 12 weeks and 52 weeks |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change in Aspartate Aminotransferase (AST) From Baseline to 12 Weeks and 52 Weeks | Change from baseline in AST levels at endpoint was reported. The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | mITT Population The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC. | Posted | Mean | Standard Deviation | U/L | 12 weeks and 52 weeks |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change in Alanine Aminotransferase (ALT) From Baseline to 12 Weeks and 52 Weeks | Change from baseline in ALT levels at endpoint was reported. The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | mITT Population The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC. | Posted | Mean | Standard Deviation | U/L | 12 weeks and 52 weeks |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change in Gamma-glutamyl Transferase (GGT) From Baseline to 12 Weeks and 52 Weeks | Change from baseline in GGT levels at endpoint was reported. The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | mITT Population The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC. | Posted | Mean | Standard Deviation | U/L | 12 weeks and 52 weeks |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change in Bilirubin - Total Bilirubin (TB) From Baseline to 12 Weeks and 52 Weeks | Change from baseline in TB levels at endpoint is being reported. The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | mITT Population The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC. | Posted | Mean | Standard Deviation | mg/dL | 12 weeks and 52 weeks |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants Meet Composite Endpoint Criteria of ALP and Total Bilirubin | Participant meets composite endpoint is defined by participant meets all of the following criteria:
Endpoint of Alkaline Phosphatase and Total Bilirubin by Visit (mITT Population) | mITT Population The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC. | Posted | Number | percentage of participants | 12 Weeks and 52 Weeks |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants Meet Published PBC Response Criteria - Paris I | Percentage of participants with response based on Paris I risk score was defined as ALP less than or equal to (≤) 3x ULN and aspartate aminotransferase (AST) less than or equal to (≤) 2 x ULN and Total Bilirubin ≤ 1 mg/dL. The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | mITT Population The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC. | Posted | Number | percentage of participants | 12 weeks and 52 weeks |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants Meet Published PBC Response Criteria - Paris II | Percentage of participants with response based on Paris II risk score was defined as ALP≤1.5xULN and AST≤1.5xULN and Total Bilirubin ≤ 1 mg/dL. The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | mITT Population. The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC. | Posted | Number | percentage of participants | 12 weeks and 52 weeks |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants Meet Published PBC Response Criteria - Toronto I | Percentage of participants with response based on Toronto I risk score defined as ALP ≤ 1.67 x ULN. The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | mITT Population. The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC. | Posted | Number | percentage of subjects | 12 weeks and 52 weeks |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | UK-PBC Risk Score Value | The UK-PBC Risk Score at endpoint is defined by the mean percentage risk that a PBC patient treated with ursodeoxycholic acid (UDCA) would develop liver failure requiring liver transplantation in 5, 10 and 15 years from diagnosis. The higher the score might indicate higher risk to death or live transplantation. Formula used for UK-PBC risk score = 1- 0.982 ^EXP(0.0287854*(ALP12 x ULN-1.722136304) - 0.0422873*(((TA12 xULN/10)^-1) - 8.675729006) + 1.4199 * (LN(BIL12 x ULN/10)+2.709607778)-1.960303*(Albumin x LLN-1.17673001)-0.4161954*(Platelet x LLN-1.873564875)). Where, Baseline survivor function = 0.982, 0.941, and 0.893 for 5 years, 10 years and 15 years respectively. ALP12, TA12 and BIL12 refers to the ALP, transaminases (ALT, AST), and total bilirubin assessments, respectively. The modified Intent-to-Treat (mITT) analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | mITT Population. The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC. | Posted | Mean | Standard Deviation | units on a scale | 12 weeks and 52 weeks |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in Pruritus Visual Analog Score (VAS) at Week 12 and Week 52 | VAS is the commonly used graphic tool for self-reporting of pruritus intensity in patients. VAS is a simple to use, validated, reliable and widely applicable tool that does not determine the impact of pruritus to quality of life. It comprises of a 100-mm horizontal line labelled as "no symptom" on left end and "worst imaginable symptom" on right end. Based on the intensity of the itch patient is instructed to draw a vertical line on the horizontal scale having a range [VAS values (unit: mm) ranging from 0 to 100, where 0 represents "no itching" and 100 "worst possible itching"]. The modified Intent-to-Treat (mITT) analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | mITT Population. The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC. | Posted | Mean | Standard Deviation | score on a scale | 12 weeks and 52 weeks |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change From Baseline in PBC-40 Quality of Life (QoL) at Week 12 and Week 52 | The PBC-40 QoL questionnaire is a disease-specific health-related tool developed for measuring the psychometric profile in PBC patients. It has 10 domains and 43 questions relevant to PBC, including Cognitive, Social, Emotional Function, Fatigue, Itch, and Other Symptoms. Questions in domains: 1) digestion and diet (questions 1-3); 2) experiences (questions 4-7); 3) itching (questions 8-10); 4) fatigue (questions 11-18); 5) effort and planning (questions 19-21); 6) memory and concentration (questions 22-27); 7) affects to you as person (questions 28-33); 8) affects to your social life (questions 34-37); 9) overall impact on your life (questions 38-40); 10) general health and well-being (questions A-C). Within a domain, items are scored from 1 to 5 and the individual item scores are summed to give a total domain score. High scores represent high impact and low scores low impact of PBC on QoL (mITT Population). | mITT Population. The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC. | Posted | Mean | Standard Deviation | units on a scale | 12 weeks and 52 weeks |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants Meet Published PBC Response Criteria - Barcelona | Percentage of participants with response based on Barcelona risk scores was defined as Normalization of ALP or a Decrease of ALP ≥ 40%. The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | mITT Population. The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC. | Posted | Number | percentage of participants | 12 weeks and 52 weeks |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Absolute Change in MELD Score From Baseline to 12 Weeks and 52 Weeks | Change from baseline to 12 weeks and 52 weeks in Model for End-stage Liver Disease (MELD) Score (mITT Population) The MELD score ranges from 6 to 40 and is a measure of how severe a patient's liver disease is. The higher the score, the more likely the patients will need a liver transplant. A calculated prognostic risk factor used to assess the potential need for a liver transplant. MELD(i) score = 10*[0.957*ln(creatinine mg/dL) + 0. 378*ln(total bilirubin mg/dL) + 1.120*ln (INR) + 0.643]. If MELD(i) is less than or equal to 11 then MELD = MELD(i). If MELD(i) is greater than 11 then MELD = MELD(i) + (1.32 *(137 - (Na)) - (0.033*MELD(i)*(137 - Na)) | mITT Population. The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC. | Posted | Mean | Standard Deviation | Change from Baseline | 12 weeks and 52 weeks |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Change in GLOBE PBC Score From Baseline to 12 Weeks and 52 Weeks | Change from Baseline to 12 weeks and 52 weeks in Global PBC Study Group (GLOBE) score (mITT Population) The GLOBE score is a validated risk assessment tool providing an estimate of transplant-free survival for patients with PBC. It was developed by the Global PBC Study Group using Cox regression model on over 4,000 patients with PBC. Lower GLOBE score predicts lower risk. It is calculated from the following equation: GLOBE score = (0.044378 * age + 0.93982 * LN(total bilirubin/ULN) +(0.335648 * LN(alkaline phosphatase/ULN)) - 2.266708 * albumin /LLN -0.002581 * platelet count per 109/L) + 1.216865 | mITT Population The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC. | Posted | Mean | Standard Deviation | units on a scale | 12 weeks and 52 weeks |
| |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Participants Meet Rotterdam Criteria | participants with Response Based on Rotterdam Criteria at Weeks 12 and 52 Rotterdam Published PBC Response Criteria by Visit (mITT Population) Rotterdam criteria: Early (normal total bilirubin and normal albumin), Moderately advanced (either abnormal albumin or abnormal total bilirubin), and Advanced (both abnormal albumin and abnormal total bilirubin). From Early stage to Moderate Stage and to Advanced Stage, it becomes worse and worse in abnormality. | mITT Population. The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC. | Posted | Number | participants | 12 weeks and 52 weeks |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percentage of Participants Meet Composite Endpoint of AP and Total Bilirubin Criteria at Week 12 and Week 52 | Percentage of participants with Response Defined by Composite Endpoint (ALP< 1.67 * Upper Limit of Normal [ULN] at Endpoint, Total Bilirubin [BIL] within Normal Limits at Endpoint, and Greater Than Equal To [≥] 15% ALP Reduction) from Baseline to Week 12 and Week 52 The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | mITT Population. The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC. | Posted | Number | percentage of subjects | 12 weeks and 52 weeks |
| ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Secondary | Percent Change in Serum Alkaline Phosphatase (ALP) | Percent change in ALP from baseline to Weeks 12 and 52 The mITT analysis set included all randomized subjects with confirmed PBC who received at least one study drug dose and had at least one post baseline ALP evaluation on treatment. | mITT Population. The mITT population is defined as any enrolled subject who receives at least one dose of medication and has at least one post-baseline AP evaluation and who have confirmed PBC. | Posted | Mean | Standard Deviation | percentage Change | 12 weeks and 52 weeks |
|
Up to Week 56
Not provided
Not provided
| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | MBX-8025 2 mg | MBX-8025 2 mg capsule once dailyMBX-8025 2 mg Capsule: Initial 8-week treatment:• MBX-8025 2 mgSubjects received two seladelpar 1 milligram (mg) capsules orally once daily for 8 weeks with a 44-week extension period. Dose up-titration for efficacy reasons could be made after 12 weeks of treatment up to 10 mg | 0 | 11 | 1 | 11 | 11 | 11 |
| EG001 | MBX-8025 5 mg | MBX-8025 5 mg capsule once dailyMBX-8025 5 mg Capsule: Initial 8-week treatment:• MBX-8025 5 mgSubjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment. | 0 | 53 | 8 | 53 | 45 | 53 |
| EG002 | MBX-8025 10 mg | MBX-8025 10 mg capsule once dailyMBX-8025 10 mg Capsule: Initial 8-week treatment:• MBX-8025 10 mgSubjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period. | 0 | 55 | 5 | 55 | 47 | 55 |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Febrile neutropenia | Blood and lymphatic system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Angina pectoris | Cardiac disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Atrial fibrillation | Cardiac disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Supraventricular tachycardia | Cardiac disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Pneumonia | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Pyonephrosis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Femoral neck fracture | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Post procedural haemorrhage | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Procedural pain | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Rib fracture | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Metabolic acidosis | Metabolism and nutrition disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Intervertebral disc protrusion | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Syncope | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
|
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Iron deficiency anaemia | Blood and lymphatic system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Palpitations | Cardiac disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Dry eye | Eye disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Abdominal discomfort | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Abdominal distension | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Abdominal pain | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Abdominal pain upper | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Abdominal tenderness | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Constipation | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Diarrhoea | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Dry mouth | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Dyschezia | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Dyspepsia | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Flatulence | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Haematochezia | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Irritable bowel syndrome | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Nausea | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Vomiting | Gastrointestinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Asthenia | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Chest discomfort | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Fatigue | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Feeling of body temperature change | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Influenza like illness | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Oedema peripheral | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Peripheral swelling | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Pyrexia | General disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Biliary colic | Hepatobiliary disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Cholelithiasis | Hepatobiliary disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Allergy to arthropod sting | Immune system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Allergy to vaccine | Immune system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Bronchitis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Cellulitis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Gastrointestinal viral infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Influenza | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Nasopharyngitis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Rash pustular | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Sinusitis | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Upper respiratory tract infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Urinary tract infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Viral infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Viral upper respiratory tract infection | Infections and infestations | MedDRA 22.0 | Systematic Assessment |
| |
| Contusion | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Fall | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Muscle strain | Injury, poisoning and procedural complications | MedDRA 22.0 | Systematic Assessment |
| |
| Troponin increased | Investigations | MedDRA 22.0 | Systematic Assessment |
| |
| Decreased appetite | Metabolism and nutrition disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Muscle spasms | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Musculoskeletal chest pain | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Myalgia | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Neck pain | Musculoskeletal and connective tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Melanocytic naevus | Neoplasms benign, malignant and unspecified (incl cysts and polyps) | MedDRA 22.0 | Systematic Assessment |
| |
| Dizziness | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Headache | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Memory impairment | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Paraesthesia | Nervous system disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Insomnia | Psychiatric disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Nightmare | Psychiatric disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Chromaturia | Renal and urinary disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Pollakiuria | Renal and urinary disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Urine odour abnormal | Renal and urinary disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Dysmenorrhoea | Reproductive system and breast disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Pruritus genital | Reproductive system and breast disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Cough | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Haemoptysis | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Nasal congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Alopecia | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Erythema | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Pruritus | Skin and subcutaneous tissue disorders | MedDRA 22.0 | Systematic Assessment |
| |
| Hypertension | Vascular disorders | MedDRA 22.0 | Systematic Assessment |
|
First, there was an imbalance in baseline ALP levels among cohorts. Second, the criteria for dose uptitration were not standardized, except that uptitration could only be done at or after Week 12. Third, this was not a placebo-controlled study. Finally, multiplicity adjustments were not made for efficacy endpoints. Nominal p-values were provided for descriptive purposes.
Not provided
Not provided
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Elaine Watkins, DO, MSPH, Vice President of Clinical Development | CymaBay Therapeutics, Inc. | 510-293-8800 | medinfo@cymabay.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 26, 2019 | Mar 21, 2022 | SAP_001.pdf |
| ID | Term |
|---|---|
| D008105 | Liver Cirrhosis, Biliary |
| ID | Term |
|---|---|
| D002780 | Cholestasis, Intrahepatic |
| D002779 | Cholestasis |
| D001649 | Bile Duct Diseases |
| D001660 | Biliary Tract Diseases |
| D004066 | Digestive System Diseases |
| D008107 | Liver Diseases |
| D008103 | Liver Cirrhosis |
| D005355 | Fibrosis |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
Not provided
Not provided
| ID | Term |
|---|---|
| C572244 | (2-methyl-4-(5-methyl-2-(4-trifluoromethyl-phenyl)-2H-(1,2,3)triazol-4-ylmethylsulfanyl)phenoxy)acetic acid |
| C000713688 | seladelpar |
Not provided
Not provided
Not provided
| Between 18 and 65 years |
|
| >=65 years |
|
| Male |
|
| Black or African-American |
|
| Asian |
|
| American Indian or Alaska native |
|
| Other |
|
| Multiple |
|
| United States |
|
| United Kingdom |
|
| Germany |
|
The analyses will assess the change from baseline in each treatment group and will assess the hypothesis that there are no differences in the percent change in ALP serum level between seladelpar 2 mg and 10 mg treatment groups after 8 weeks of treatment
| ANCOVA |
| = 0.0021 |
| Other |
| The analyses will assess the change from baseline in each treatment group and will assess the hypothesis that there are no differences in the percent change in ALP serum level between seladelpar 5 mg and 10 mg treatment groups after 8 weeks of treatment | ANCOVA | = 0.0024 | Other |
MBX-8025 10 mg capsule once daily MBX-8025 10 mg Capsule: Initial 8-week treatment: • MBX-8025 10 mg Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period. |
|
|
MBX-8025 10 mg capsule once daily MBX-8025 10 mg Capsule: Initial 8-week treatment: • MBX-8025 10 mg Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period. |
|
|
MBX-8025 10 mg capsule once daily MBX-8025 10 mg Capsule: Initial 8-week treatment: • MBX-8025 10 mg Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period. |
|
|
MBX-8025 10 mg capsule once daily MBX-8025 10 mg Capsule: Initial 8-week treatment: • MBX-8025 10 mg Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period. |
|
|
MBX-8025 10 mg capsule once daily MBX-8025 10 mg Capsule: Initial 8-week treatment: • MBX-8025 10 mg Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period. |
|
|
| MBX-8025 (10 mg) |
MBX-8025 10 mg capsule once daily MBX-8025 10 mg Capsule: Initial 8-week treatment: • MBX-8025 10 mg Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period. |
|
|
| OG002 | MBX-8025 (10 mg) | MBX-8025 10 mg capsule once daily MBX-8025 10 mg Capsule: Initial 8-week treatment: • MBX-8025 10 mg Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period. |
|
|
| OG002 |
| MBX-8025 (10 mg) |
MBX-8025 10 mg capsule once daily MBX-8025 10 mg Capsule: Initial 8-week treatment: • MBX-8025 10 mg Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period. |
|
|
| MBX-8025 (10 mg) |
MBX-8025 10 mg capsule once daily MBX-8025 10 mg Capsule: Initial 8-week treatment: • MBX-8025 10 mg Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period. |
|
|
| OG001 | MBX-8025 (5 mg) | MBX-8025 5 mg capsule once daily MBX-8025 5 mg Capsule: Initial 8-week treatment: • MBX-8025 5 mg Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment. |
| OG002 | MBX-8025 (10 mg) | MBX-8025 10 mg capsule once daily MBX-8025 10 mg Capsule: Initial 8-week treatment: • MBX-8025 10 mg Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period. |
|
|
MBX-8025 5 mg capsule once daily MBX-8025 5 mg Capsule: Initial 8-week treatment: • MBX-8025 5 mg Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment. |
| OG002 | MBX-8025 (10 mg) | MBX-8025 10 mg capsule once daily MBX-8025 10 mg Capsule: Initial 8-week treatment: • MBX-8025 10 mg Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period. |
|
|
| OG001 |
| MBX-8025 (5 mg) |
MBX-8025 5 mg capsule once daily MBX-8025 5 mg Capsule: Initial 8-week treatment: • MBX-8025 5 mg Subjects were randomized to receive one seladelpar 5 mg capsule orally once daily for 8 weeks with a 44-week extension period. Dose up-titration to 10 mg for efficacy reasons could be made after 12 weeks of treatment. |
| OG002 | MBX-8025 (10 mg) | MBX-8025 10 mg capsule once daily MBX-8025 10 mg Capsule: Initial 8-week treatment: • MBX-8025 10 mg Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period. |
|
|
| OG002 |
| MBX-8025 (10 mg) |
MBX-8025 10 mg capsule once daily MBX-8025 10 mg Capsule: Initial 8-week treatment: • MBX-8025 10 mg Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period. |
|
|
| OG002 | MBX-8025 (10 mg) | MBX-8025 10 mg capsule once daily MBX-8025 10 mg Capsule: Initial 8-week treatment: • MBX-8025 10 mg Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period. |
|
|
| OG002 | MBX-8025 (10 mg) | MBX-8025 10 mg capsule once daily MBX-8025 10 mg Capsule: Initial 8-week treatment: • MBX-8025 10 mg Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period. |
|
|
| OG002 | MBX-8025 (10 mg) | MBX-8025 10 mg capsule once daily MBX-8025 10 mg Capsule: Initial 8-week treatment: • MBX-8025 10 mg Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period. |
|
|
| OG002 | MBX-8025 (10 mg) | MBX-8025 10 mg capsule once daily MBX-8025 10 mg Capsule: Initial 8-week treatment: • MBX-8025 10 mg Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period. |
|
|
MBX-8025 10 mg capsule once daily MBX-8025 10 mg Capsule: Initial 8-week treatment: • MBX-8025 10 mg Subjects were randomized to receive one seladelpar 10 mg capsule orally once daily for 8 weeks with a 44-week extension period. |
|
|