Trial · NCT04526665
Study of Elafibranor in Patients With Primary Biliary Cholangitis (PBC)
NCT04526665, Phase 3, Active not recruiting, Ipsen; results first posted 2024-09-05
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Study of Elafibranor in Patients With Primary Biliary Cholangitis (PBC). Trials, record NCT04526665. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04526665
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Study of Elafibranor in Patients With Primary Biliary Cholangitis (PBC)
- NCT number
- NCT04526665
- Status
- Active not recruiting
- Phases
- Phase 3
- Enrollment
- 161 participants, actual
- Lead sponsor
- Ipsen
- Conditions
- Primary Biliary Cirrhosis
- Interventions
- Elafibranor 80mg, Placebo
- First posted
- 2020-08-26
- Last updated
- 2026-11-05
- Other registries' IDs
- eudract:2019-004941-34
- Results first posted
- 2024-09-05
Source ClinicalTrials.gov
Registered outcomes47
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Participants With Response to Treatment Based on Cholestasis Response at Week 52 | At Week 52 | Cholestasis response was defined as alkaline phosphatase (ALP) \< 1.67 x upper limit of normal (ULN) and total bilirubin (TB) \<= ULN and ALP decrease from baseline \>= 15% at Week 52 and based on the composite strategy imputing non-response for participants who experienced intercurrent events (ICEs) (study treatment discontinuation or use of rescue therapy for PBC) prior to Week 52. |
| Secondary | Key Secondary Endpoint: Percentage of Participants With Response to Treatment Based on ALP Normalization at Week 52 | At Week 52 | Response to treatment based on normalization of ALP at Week 52 was defined as percentage of participants with ALP =\<1.0× ULN and based on the composite strategy imputing non-response for participants who experienced ICEs (study treatment discontinuation or use of rescue therapy for PBC) prior to Week 52. |
| Secondary | Key Secondary Endpoint: Change From Baseline in Pruritus Based on PBC Worst Itch Numeric Rating Scale (NRS) Score in Participants With Baseline PBC Worst Itch NRS Score ≥4 to Week 52 | Baseline (up to 14 days pre-dose) and Week 52 | The PBC Worst Itch NRS is a simple, self-administered patient reported outcome (PRO) questionnaire that measures itch intensity. Participants rate intensity of their worst Itch on an 11-point scale ranging from 0 (no itch) to 10 (worst itch imaginable). Total score ranged from 0 (no itch) to 10 (worst itch imaginable). Higher scores indicated worse outcomes. Baseline was defined as the average of the daily PBC Worst itch scores on available data within 14 days prior the day of randomization. Post-baseline data was defined as the average of the daily PBC Worst itch scores on available data in 4-week period intervals (available data every 28 days after the day of first study drug intake). |
| Secondary | Key Secondary Endpoint: Change From Baseline in Pruritus Based on PBC Worst Itch NRS Score in Participants With Baseline PBC Worst Itch NRS Score ≥4 to Week 24 | Baseline (up to 14 days pre-dose) and Week 24 | The PBC Worst Itch NRS is a simple, self-administered PRO questionnaire that measures itch intensity. Participants rate intensity of their worst Itch on an 11-point scale ranging from 0 (no itch) to 10 (worst itch imaginable). Total score ranged from 0 (no itch) to 10 (worst itch imaginable). Higher scores indicated worse outcomes. Baseline was defined as the average of the daily PBC Worst itch scores on available data within 14 days prior the day of randomization. Post-baseline data was defined as the average of the daily PBC Worst itch scores on available data in 4-week period intervals (available data every 28 days after the day of first study drug intake). |
| Secondary | Change From Baseline in ALP at Weeks 4, 13, 26, 39 and 52 | Baseline (Day 1) and at Weeks 4, 13, 26, 39 and 52 | Blood samples were collected at specified timepoints for assessment of ALP. Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication. |
| Secondary | Percentage of Participants With ALP Response From Baseline at Week 52 | Baseline (Day 1) and at Week 52 | ALP response was defined as ALP decrease from baseline \>= 10%; ALP decrease from baseline \>= 20% and ALP decrease from baseline \>= 40% at Week 52 and based on the composite strategy imputing non-response for participants who experienced ICEs (study treatment discontinuation or use of rescue therapy for PBC) prior to Week 52. Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication. |
| Secondary | Percentage of Participants With Response to Treatment According to ALP < 1.5x ULN, ALP Decrease From Baseline >= 40% and TB =<ULN at Week 52 | At Week 52 | Response to treatment was according to ALP \< 1.5x ULN, ALP decrease from baseline \>= 40% and TB =\<ULN at Week 52. It was based on the composite strategy imputing non-response for participants who experienced ICEs (study treatment discontinuation or use of rescue therapy for PBC) prior to Week 52. Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication. |
| Secondary | Percentage of Participants With Response to Treatment According to ALP < 3x ULN, Aspartate Aminotransferase (AST) < 2x ULN and TB =< 1 Milligrams Per Deciliter (mg/dL) (Paris I) at Week 52 | At Week 52 | Response to treatment was according to ALP \< 3x ULN, AST \< 2x ULN and TB =\< 1 mg/dL (Paris I) at Week 52. It was based on the composite strategy imputing non-response for participants who experienced ICEs (study treatment discontinuation or use of rescue therapy for PBC) prior to Week 52. |
| Secondary | Percentage of Participants With Response to Treatment According to ALP =< 1.5x ULN, AST =< 1.5x ULN and TB =< 1 mg/dL (Paris II) at Week 52 | At Week 52 | Response to treatment was according to ALP =\< 1.5x ULN, AST =\< 1.5x ULN and TB =\< 1 mg/dL (Paris II) at Week 52. It was based on the composite strategy imputing non-response for participants who experienced ICEs (study treatment discontinuation or use of rescue therapy for PBC) prior to Week 52. |
| Secondary | Percentage of Participants With Response to Treatment According to TB Decrease of 15% Change From Baseline at Week 52 | At Week 52 | Response to treatment was according to TB decrease of 15% change from baseline at Week 52. It was based on the composite strategy imputing non-response for participants who experienced ICEs (study treatment discontinuation or use of rescue therapy for PBC) prior to Week 52. Baseline was defined as the last non-missing central value on or before the first dose of the randomized study medication. |
1 to 10 of 47Source ClinicalTrials.gov
See all 47 registered outcomesOutcomes47
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Percentage of Participants With Response to Treatment Based on Cholestasis Response at Week 52 | At Week 52 | The ITT population included all randomized participants. | Percentage of participants | NUMBER |
| SECONDARY | Key Secondary Endpoint: Percentage of Participants With Response to Treatment Based on ALP Normalization at Week 52 | At Week 52 | The ITT population included all randomized participants. | Percentage of participants | NUMBER |
| SECONDARY | Key Secondary Endpoint: Change From Baseline in Pruritus Based on PBC Worst Itch Numeric Rating Scale (NRS) Score in Participants With Baseline PBC Worst Itch NRS Score ≥4 to Week 52 | Baseline (up to 14 days pre-dose) and Week 52 | The Pruritus ITT population included all participants from the ITT analysis set with baseline PBC Worst Itch NRS score ≥4. Only those participants with data collected at Week 52 are reported. | Scores on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Key Secondary Endpoint: Change From Baseline in Pruritus Based on PBC Worst Itch NRS Score in Participants With Baseline PBC Worst Itch NRS Score ≥4 to Week 24 | Baseline (up to 14 days pre-dose) and Week 24 | The Pruritus ITT population included all participants from the ITT analysis set with baseline PBC Worst Itch NRS score ≥4. Only those participants with data collected at Week 24 are reported. | Scores on a scale | LEAST_SQUARES_MEAN |
| SECONDARY | Change From Baseline in ALP at Weeks 4, 13, 26, 39 and 52 | Baseline (Day 1) and at Weeks 4, 13, 26, 39 and 52 | The ITT population included all randomized participants. Only those participants with data collected at specified timepoints are reported. | Units per Liter (U/L) | MEAN |
| SECONDARY | Percentage of Participants With ALP Response From Baseline at Week 52 | Baseline (Day 1) and at Week 52 | The ITT population included all randomized participants. Only those participants with data collected at Week 52 are reported. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Response to Treatment According to ALP < 1.5x ULN, ALP Decrease From Baseline >= 40% and TB =<ULN at Week 52 | At Week 52 | The ITT population included all randomized participants. Only those participants with data collected at Week 52 are reported. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Response to Treatment According to ALP < 3x ULN, Aspartate Aminotransferase (AST) < 2x ULN and TB =< 1 Milligrams Per Deciliter (mg/dL) (Paris I) at Week 52 | At Week 52 | The ITT population included all randomized participants. Only those participants with data collected at Week 52 are reported. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Response to Treatment According to ALP =< 1.5x ULN, AST =< 1.5x ULN and TB =< 1 mg/dL (Paris II) at Week 52 | At Week 52 | The ITT population included all randomized participants. Only those participants with data collected at Week 52 are reported. | Percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants With Response to Treatment According to TB Decrease of 15% Change From Baseline at Week 52 | At Week 52 | The ITT population included all randomized participants. Only those participants with data collected at Week 52 are reported. | Percentage of participants | NUMBER |
1 to 10 of 47Source ClinicalTrials.gov
See all 47 outcomesResults by group220
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Percentage of Participants With Response to Treatment Based on Cholestasis Response at Week 52 | Elafibranor 80 mg | 50.9 | 95% Confidence Interval 41.6 to 60.2 | Percentage of participants | |
| Percentage of Participants With Response to Treatment Based on Cholestasis Response at Week 52 | Placebo | 3.8 | 95% Confidence Interval 1.0 to 12.8 | Percentage of participants | |
| Key Secondary Endpoint: Percentage of Participants With Response to Treatment Based on ALP Normalization at Week 52 | Elafibranor 80 mg | 14.8 | 95% Confidence Interval 9.3 to 22.7 | Percentage of participants | |
| Key Secondary Endpoint: Percentage of Participants With Response to Treatment Based on ALP Normalization at Week 52 | Placebo | 0 | 95% Confidence Interval 0.0 to 6.8 | Percentage of participants | |
| Key Secondary Endpoint: Change From Baseline in Pruritus Based on PBC Worst Itch Numeric Rating Scale (NRS) Score in Participants With Baseline PBC Worst Itch NRS Score ≥4 to Week 52 | Elafibranor 80 mg | -1.930 | 95% Confidence Interval -2.602 to -1.258 | Scores on a scale | |
| Key Secondary Endpoint: Change From Baseline in Pruritus Based on PBC Worst Itch Numeric Rating Scale (NRS) Score in Participants With Baseline PBC Worst Itch NRS Score ≥4 to Week 52 | Placebo | -1.146 | 95% Confidence Interval -2.143 to -0.150 | Scores on a scale | |
| Key Secondary Endpoint: Change From Baseline in Pruritus Based on PBC Worst Itch NRS Score in Participants With Baseline PBC Worst Itch NRS Score ≥4 to Week 24 | Elafibranor 80 mg | -1.598 | 95% Confidence Interval -2.246 to -0.951 | Scores on a scale | |
| Key Secondary Endpoint: Change From Baseline in Pruritus Based on PBC Worst Itch NRS Score in Participants With Baseline PBC Worst Itch NRS Score ≥4 to Week 24 | Placebo | -1.255 | 95% Confidence Interval -2.201 to -0.309 | Scores on a scale | |
| Change From Baseline in ALP at Weeks 4, 13, 26, 39 and 52 | Elafibranor 80 mg | Week 4 | -116.4 | Standard Deviation 63.1 | Units per Liter (U/L) |
| Change From Baseline in ALP at Weeks 4, 13, 26, 39 and 52 | Placebo | Week 4 | -14.3 | Standard Deviation 92.6 | Units per Liter (U/L) |
1 to 10 of 220Source ClinicalTrials.gov
See all 220 results by groupParticipant flow6
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Elafibranor 80 mg | 108 | |
| Overall Study | STARTED | Placebo | 53 | |
| Overall Study | COMPLETED | Elafibranor 80 mg | 94 | |
| Overall Study | COMPLETED | Placebo | 46 | |
| Overall Study | NOT COMPLETED | Elafibranor 80 mg | 14 | |
| Overall Study | NOT COMPLETED | Placebo | 7 |
Source ClinicalTrials.gov
Adverse events110
Each adverse event the trial posted, group by group: the participants affected out of those at risk.
| Type | Event | Organ system | Group | Affected | At risk | Events |
|---|---|---|---|---|---|---|
| other | Abdominal pain upper | Gastrointestinal disorders | Elafibranor 80 mg | 8 | 108 | 10 |
| other | Abdominal pain upper | Gastrointestinal disorders | Placebo | 3 | 53 | 5 |
| other | Constipation | Gastrointestinal disorders | Elafibranor 80 mg | 9 | 108 | 11 |
| other | Constipation | Gastrointestinal disorders | Placebo | 1 | 53 | 1 |
| other | Diarrhoea | Gastrointestinal disorders | Elafibranor 80 mg | 12 | 108 | 13 |
| other | Diarrhoea | Gastrointestinal disorders | Placebo | 5 | 53 | 5 |
| other | Dyspepsia | Gastrointestinal disorders | Elafibranor 80 mg | 4 | 108 | 4 |
| other | Dyspepsia | Gastrointestinal disorders | Placebo | 3 | 53 | 3 |
| other | Gastrooesophageal reflux disease | Gastrointestinal disorders | Elafibranor 80 mg | 7 | 108 | 7 |
| other | Gastrooesophageal reflux disease | Gastrointestinal disorders | Placebo | 1 | 53 | 1 |
1 to 10 of 110Source ClinicalTrials.gov
See all 110 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Elafibranor 80 mg | 2 | 108 |
| Total, all-cause mortality | Placebo | 0 | 53 |
| Total, other adverse events | Elafibranor 80 mg | 103 | 108 |
| Total, other adverse events | Placebo | 47 | 53 |
| Total, serious adverse events | Elafibranor 80 mg | 11 | 108 |
| Total, serious adverse events | Placebo | 7 | 53 |
Source ClinicalTrials.gov
Papers3
The papers the trial lists on ClinicalTrials.gov.
Grants0
The grants NIH RePORTER links to the trial, newest funded first: NIH's, and some of AHRQ's, CDC's, FDA's and VA's. Each shows its title, principal investigators, organization and funder in its newest year.
None in this release.
Trial comparisons2
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
| Comparison | Source |
|---|---|
| Elafibranor 80 mg vs Placebo: deaths from any cause | Trial comparisons |
| Elafibranor 80 mg vs Placebo: serious adverse events | Trial comparisons |
Tests0
The ConductCognition tests this trial's registered outcomes name, as ScienceGraph matched them in trial-tests-2026-11-12-0.2.1.
None in this release.
Data dates
The day each source was copied for this release.
Sources 3
- ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
- ClinicalTrials.govNo license stated
- NIH RePORTER's clinical studies and project filesPublic domain
Each source's terms
- ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
- ClinicalTrials.govnone stated
- NIH RePORTER's clinical studies and project filesPublic domain (NIH RePORTER)
ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.
For software
Read this trial from software.
The record and each of its lists are JSON, twenty rows a page. No key.
GET https://conductscience.org/api/trials/v1/NCT04526665
GET https://conductscience.org/api/trials/v1/NCT04526665/registered?page=2