Trial · NCT05543187
A Study of TAK-625 for the Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC)
NCT05543187, Phase 3, Completed, Takeda; results first posted 2024-09-24
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. A Study of TAK-625 for the Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC). Trials, record NCT05543187. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT05543187
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- A Study of TAK-625 for the Treatment of Progressive Familial Intrahepatic Cholestasis (PFIC)
- NCT number
- NCT05543187
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 5 participants, actual
- Lead sponsor
- Takeda
- Conditions
- Progressive Familial Intrahepatic Cholestasis (PFIC)
- Interventions
- TAK-625
- First posted
- 2022-09-16
- Last updated
- 2026-01-28
- Results first posted
- 2024-09-24
Source ClinicalTrials.gov
Registered outcomes5
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Change in the Average Morning Itch Reported Outcome (ItchRO) (Observer Instrument [Obs]) Severity Score Between Baseline and the Average of Week 15 Through Week 26 | Baseline to Week 15 through Week 26 | The ItchRO (Obs) scale measures severity of pruritus. The score on ItchRO (Obs) scale ranged from 0 to 4, where 0=None observed or reported, 1=Mild, 2=Moderate, 3=Severe, 4=Very severe. A higher score indicated more severe pruritus. Average baseline morning ItchRO (Obs) scores were calculated as sum of the morning scores divided by number of morning scores for the 4-week (28 days) time periods (that is \[i.e.\], Day -28 to Day -1). Average morning ItchRO (Obs) scores Week 15 through Week 26 were calculated as the sum of the morning scores divided by the number of morning scores from Week 15 to Week 26. Change was calculated as: Average value of Weeks 15 to 26 - Average value of Baseline (Day -28 to Day -1). |
| Secondary | Change in the Average Morning ItchRO (Obs) Frequency Score Between Baseline and the Average of Week 15 Through Week 26 | Baseline to Week 15 through Week 26 | The ItchRO (Obs) scale measures frequency of pruritus. The score on ItchRO (Obs) scale ranged from 0 to 4, where 0=None observed or reported, 1= A little bit of the time, 2= Some of the time, 3= Most of the time, 4= Almost all of the time/constantly, I don't know) to describe their pruritus condition. 'I don't know' was categorized as missing data. A higher score indicated more severe pruritus. Baseline average morning ItchRO (Obs) frequency scores were calculated as the sum of the morning frequency scores divided by the number of morning severity scores for the 4-week (28 days) time periods. (i.e., Day -28 to Day -1). Average morning ItchRO (Obs) frequency scores of Week 15 through Week 26 were calculated as the sum of the morning frequency scores divided by the number of mornings frequency scores from Week 15 to Week 26. Change was calculated as: Average value of Weeks 15 to 26 - Average value of Baseline (Day -28 to Day -1). |
| Secondary | Change From Baseline in Total Serum Bile Acid (sBA) Levels to Week 26 | Baseline to Week 26 | Change from baseline was calculated as: Post-baseline observed value - Baseline (before first dosing) observed value. Change from baseline in total sBA levels to Week 26 was reported. |
| Secondary | Percentage of Participants (Responders) Who Experienced an sBA Control From Baseline Through Week 26 | Baseline through Week 26 | Responders to sBA control were defined as participants who achieved a decrease to less than (\<) 102 mcmol/L, a decrease of greater than (\>) 75 percentage (%), or normalization at any timepoint from baseline through Week 26. |
| Secondary | Change in the ItchRO (Obs) Weekly Average Severity Between Baseline and the Average of Week 15 Through Week 26 | Baseline to Week 15 through Week 26 | ItchRO (Obs) scale measures severity of pruritus, score ranged from 0 to 4, where 0=None observed or reported, 1=Mild, 2=Moderate, 3=Severe, 4=Very severe. A higher score indicated more severe pruritus. Weekly average severity was calculated based on daily maximum severity scores from both morning and evening. Average baseline morning and evening ItchRO (Obs) scores were calculated as sum of morning or evening scores divided by number of morning or evening scores for 4-week (28 days) time periods (i.e., Day -28 to Day -1). Average morning and evening ItchRO (Obs) scores Week 15 to Week 26 were calculated as sum of morning or evening scores divided by number of morning or evening scores from Week 15 to Week 26. Change was calculated as: Average value of Weeks 15 to 26 - Average value of Baseline (Day -28 to Day -1). |
Source ClinicalTrials.gov
Outcomes5
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change in the Average Morning Itch Reported Outcome (ItchRO) (Observer Instrument [Obs]) Severity Score Between Baseline and the Average of Week 15 Through Week 26 | Baseline to Week 15 through Week 26 | ITT included all participants who received at least one dose of study drug. As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study. | score on a scale | MEAN |
| SECONDARY | Change in the Average Morning ItchRO (Obs) Frequency Score Between Baseline and the Average of Week 15 Through Week 26 | Baseline to Week 15 through Week 26 | ITT included all participants who received at least one dose of study drug. Here, overall number of participants analyzed signified those participants who were evaluable for this outcome measure. As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study. | score on a scale | MEAN |
| SECONDARY | Change From Baseline in Total Serum Bile Acid (sBA) Levels to Week 26 | Baseline to Week 26 | ITT included all participants who received at least one dose of study drug. As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study. | micromoles per liter (mcmol/L) | MEAN |
| SECONDARY | Percentage of Participants (Responders) Who Experienced an sBA Control From Baseline Through Week 26 | Baseline through Week 26 | ITT included all participants who received at least one dose of study drug. As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study. | percentage of participants | NUMBER |
| SECONDARY | Change in the ItchRO (Obs) Weekly Average Severity Between Baseline and the Average of Week 15 Through Week 26 | Baseline to Week 15 through Week 26 | ITT included all participants who received at least one dose of study drug. As per-planned analysis, the cohort-wise (primary and supplemental) data were analyzed, collected, and reported in this study. | score on a scale | MEAN |
Source ClinicalTrials.gov
Results by group10
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change in the Average Morning Itch Reported Outcome (ItchRO) (Observer Instrument [Obs]) Severity Score Between Baseline and the Average of Week 15 Through Week 26 | Primary Cohort: TAK-625 | -1.514 | 95% Confidence Interval -3.6541 to 0.6255 | score on a scale | |
| Change in the Average Morning Itch Reported Outcome (ItchRO) (Observer Instrument [Obs]) Severity Score Between Baseline and the Average of Week 15 Through Week 26 | Supplemental Cohort: TAK-625 | -0.202 | 95% Confidence Interval -3.6957 to 3.2911 | score on a scale | |
| Change in the Average Morning ItchRO (Obs) Frequency Score Between Baseline and the Average of Week 15 Through Week 26 | Primary Cohort: TAK-625 | -0.740 | 95% Confidence Interval -4.8063 to 3.3254 | score on a scale | |
| Change in the Average Morning ItchRO (Obs) Frequency Score Between Baseline and the Average of Week 15 Through Week 26 | Supplemental Cohort: TAK-625 | -0.735 | 95% Confidence Interval -6.0922 to 4.6213 | score on a scale | |
| Change From Baseline in Total Serum Bile Acid (sBA) Levels to Week 26 | Primary Cohort: TAK-625 | -149.900 | 95% Confidence Interval -561.4008 to 261.6008 | micromoles per liter (mcmol/L) | |
| Change From Baseline in Total Serum Bile Acid (sBA) Levels to Week 26 | Supplemental Cohort: TAK-625 | -18.250 | 95% Confidence Interval -173.9010 to 137.4010 | micromoles per liter (mcmol/L) | |
| Percentage of Participants (Responders) Who Experienced an sBA Control From Baseline Through Week 26 | Primary Cohort: TAK-625 | 33.3 | 95% Confidence Interval 0.84 to 90.57 | percentage of participants | |
| Percentage of Participants (Responders) Who Experienced an sBA Control From Baseline Through Week 26 | Supplemental Cohort: TAK-625 | 0.0 | 95% Confidence Interval 0.00 to 84.19 | percentage of participants | |
| Change in the ItchRO (Obs) Weekly Average Severity Between Baseline and the Average of Week 15 Through Week 26 | Primary Cohort: TAK-625 | -1.629 | 95% Confidence Interval -3.5064 to 0.2481 | score on a scale | |
| Change in the ItchRO (Obs) Weekly Average Severity Between Baseline and the Average of Week 15 Through Week 26 | Supplemental Cohort: TAK-625 | -0.254 | 95% Confidence Interval -4.3906 to 3.8824 | score on a scale |
Source ClinicalTrials.gov
Participant flow14
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Overall Study | STARTED | Primary Cohort: TAK-625 | 3 | |
| Overall Study | STARTED | Supplemental Cohort: TAK-625 | 2 | |
| Overall Study | Dosed TAK-625 150 mcg/kg | Primary Cohort: TAK-625 | 3 | |
| Overall Study | Dosed TAK-625 150 mcg/kg | Supplemental Cohort: TAK-625 | 2 | |
| Overall Study | Dosed TAK-625 300 mcg/kg | Primary Cohort: TAK-625 | 3 | |
| Overall Study | Dosed TAK-625 300 mcg/kg | Supplemental Cohort: TAK-625 | 2 | |
| Overall Study | Dosed TAK-625 450 mcg/kg | Primary Cohort: TAK-625 | 3 | |
| Overall Study | Dosed TAK-625 450 mcg/kg | Supplemental Cohort: TAK-625 | 2 | |
| Overall Study | Dosed TAK-625 600 mcg/kg | Primary Cohort: TAK-625 | 3 | |
| Overall Study | Dosed TAK-625 600 mcg/kg | Supplemental Cohort: TAK-625 | 2 |
1 to 10 of 14Source ClinicalTrials.gov
See all 14 participant flowAdverse events64
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 | Primary Cohort: TAK-625 | 0 | 3 | |
| other | Abdominal pain upper | Gastrointestinal disorders | Supplemental Cohort: TAK-625 | 1 | 2 | |
| other | Alanine aminotransferase increased | Investigations | Primary Cohort: TAK-625 | 1 | 3 | |
| other | Alanine aminotransferase increased | Investigations | Supplemental Cohort: TAK-625 | 0 | 2 | |
| other | Anaemia | Blood and lymphatic system disorders | Primary Cohort: TAK-625 | 1 | 3 | |
| other | Anaemia | Blood and lymphatic system disorders | Supplemental Cohort: TAK-625 | 0 | 2 | |
| other | Aspartate aminotransferase increased | Investigations | Primary Cohort: TAK-625 | 1 | 3 | |
| other | Aspartate aminotransferase increased | Investigations | Supplemental Cohort: TAK-625 | 0 | 2 | |
| other | Asthma | Respiratory, thoracic and mediastinal disorders | Primary Cohort: TAK-625 | 1 | 3 | |
| other | Asthma | Respiratory, thoracic and mediastinal disorders | Supplemental Cohort: TAK-625 | 0 | 2 |
1 to 10 of 64Source ClinicalTrials.gov
See all 64 adverse eventsAdverse event totals6
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Primary Cohort: TAK-625 | 0 | 3 |
| Total, all-cause mortality | Supplemental Cohort: TAK-625 | 0 | 2 |
| Total, other adverse events | Primary Cohort: TAK-625 | 3 | 3 |
| Total, other adverse events | Supplemental Cohort: TAK-625 | 2 | 2 |
| Total, serious adverse events | Primary Cohort: TAK-625 | 0 | 3 |
| Total, serious adverse events | Supplemental Cohort: TAK-625 | 0 | 2 |
Source ClinicalTrials.gov
Papers0
The papers the trial lists on ClinicalTrials.gov.
None in this release.
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 |
|---|---|
| Primary Cohort: TAK-625 vs Supplemental Cohort: TAK-625: deaths from any cause | Trial comparisons |
| Primary Cohort: TAK-625 vs Supplemental Cohort: TAK-625: 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/NCT05543187
GET https://conductscience.org/api/trials/v1/NCT05543187/events?page=2