Trial · NCT03659916
Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC
NCT03659916, Phase 3, Completed, Albireo, an Ipsen Company; results first posted 2025-07-10
3 sourcestrials-2026-11-12-0.5.0
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ConductScience Library. Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC. Trials, record NCT03659916. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT03659916
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Trial
As registered on ClinicalTrials.gov, in the graph's capture of the registry.
- Title
- Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC
- NCT number
- NCT03659916
- Status
- Completed
- Phases
- Phase 3
- Enrollment
- 116 participants, actual
- Lead sponsor
- Albireo, an Ipsen Company
- Conditions
- Progressive Familial Intrahepatic Cholestasis
- Interventions
- A4250 (odevixibat)
- First posted
- 2018-09-06
- Last updated
- 2026-10-21
- Other registries' IDs
- eudract:2017-002325-38
- Results first posted
- 2025-07-10
Source ClinicalTrials.gov
Registered outcomes21
Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.
| List | Outcome | Time frame | Description |
|---|---|---|---|
| Primary | Percentage of Positive Pruritus Assessments at the Participant Level Over 72-Week Using the Albireo Observer-Reported Outcome (ObsRo) Instrument (Combined AM and PM Scores) | Week 72 | A positive pruritus assessment was defined as a scratching score of \<=1 or at least a 1-point drop from baseline on the Albireo ObsRO instrument. The percentage of positive pruritus assessment was calculated as the number of positive pruritus assessments divided by the total number of reported assessments recorded by each participant multiplied by 100. The data reported represent the combination of pruritus assessments from both AM and PM scores. |
| Secondary | Change From Baseline in Serum Bile Acids at Week 72 | Baseline and Week 72 | Blood samples for analysis of fasting total serum bile acids were drawn at specified timepoints. Participants were to fast (water intake only) for at least 4 hours prior to the collection of samples for serum bile acids. Exceptions were made for infants \<12 months of age if unable to fast for the full 4 hours. Baseline for Cohort 1 placebo/odevixibat and Cohort 2 groups was defined as the average of last 2 values before the first dose of study treatment in the A4250-008 study. Baseline for Cohort 1 odevixibat/odevixibat group was defined as average of last 2 values before the first dose of study treatment in study A4250-005 (NCT03566238). |
| Secondary | Percentage of Positive Pruritus Assessments at the Participant Level Using the Albireo ObsRo Instrument From Weeks 0-4, 0-12, 0-22, 0-24, 0-36, 0-46, 0-48, 0-60, and 0-70 (Combined AM and PM Scores) | Weeks 0-4, Weeks 0-12, Weeks 0-22, Weeks 0-24, Weeks 0-36, Weeks 0-46, Weeks 0-48, Weeks 0-60, and Weeks 0-70 | A positive pruritus assessment was defined as a scratching score of \<=1 or at least a 1-point drop from baseline on the Albireo ObsRO instrument. The percentage of positive pruritus assessment was calculated as the number of positive pruritus assessments divided by the total number of reported assessments by each participant multiplied by 100. The data reported represent the combination of pruritus assessments from both AM and PM scores. |
| Secondary | Percentage of Positive Pruritus Assessments at the Participant Level Using the Albireo ObsRo Instrument From Weeks 0-4, 0-12, 0-22, 0-24, 0-36, 0-46, 0-48, 0-60, 0-70, and 0-72 (AM Score) | Weeks 0-4, Weeks 0-12, Weeks 0-22, Weeks 0-24, Weeks 0-36, Weeks 0-46, Weeks 0-48, Weeks 0-60, Weeks 0-70, and Weeks 0-72 | A positive pruritus assessment was defined as a scratching score of \<=1 or at least a 1-point drop from baseline on the Albireo ObsRO instrument. The percentage of positive pruritus assessment was calculated as the number of positive pruritus assessments divided by the total number of reported assessments by each participant multiplied by 100. AM score represents night-time itching/scratching and sleep disturbance. |
| Secondary | Percentage of Positive Pruritus Assessments at the Participant Level Using the Albireo ObsRo Instrument From Weeks 0-4, 0-12, 0-22, 0-24, 0-36, 0-46, 0-48, 0-60, 0-70, and 0-72 (PM Score) | Weeks 0-4, Weeks 0-12, Weeks 0-22, Weeks 0-24, Weeks 0-36, Weeks 0-46, Weeks 0-48, Weeks 0-60, Weeks 0-70, and Weeks 0-72 | A positive pruritus assessment was defined as a scratching score of \<=1 or at least a 1-point drop from baseline on the Albireo ObsRO instrument. The percentage of positive pruritus assessment was calculated as the number of positive pruritus assessments divided by the total number of reported assessments by each participant multiplied by 100. PM score represents daytime itching/scratching and tiredness. |
| Secondary | Change From Baseline in Serum Bile Acids at Weeks 4, 12, 22, 24, 36, 46, 48, 60, 70, 72 and Every 16 Weeks During the Optional Extension Period | Baseline and Weeks 4, 12, 22, 24, 36, 46, 48, 60, 70, 72, 88, 104, 120, 136, 152, 168, 184, 200, 216, 232, 248, 264, 280, 296, 312, and 328 | Blood samples for analysis of fasting total serum bile acids were drawn at specified timepoints. Participants were to fast (water intake only) for at least 4 hours prior to the collection of samples for serum bile acids. Exceptions were made for infants \<12 months of age if unable to fast for the full 4 hours. Baseline for Cohort 1 placebo/odevixibat, and Cohort 2 groups was defined as the average of last 2 values before the first dose of study treatment in the A4250-008 study. Baseline for Cohort 1 odevixibat/odevixibat group was defined as average of last 2 values before the first dose of study treatment in study A4250-005 (NCT03566238). |
| Secondary | Percentage of Positive Pruritus Assessments at the Participant Level Using the Albireo ObsRo Instrument From Weeks 1-4, 5-8, 9-12, 13-16, 17-20, 21-24, 34-36, 44-46, 47-48, 58-60, 68-70, 71-72, and 73-76 (Combined AM and PM Scores) | Weeks 1-4, 5-8, 9-12, 13-16, 17-20, 21-24, 34-36, 44-46, 47-48, 58-60, 68-70, 71-72, and 73-76 | A positive pruritus assessment was defined as a scratching score of \<=1 or at least a 1-point drop from baseline on the Albireo ObsRO instrument. The percentage of positive pruritus assessment was calculated as the number of positive pruritus assessments divided by the total number of reported assessments in the time interval recorded by each participant multiplied by 100. The data reported represent the combination of pruritus assessments from both AM and PM scores. |
| Secondary | Percentage of Positive Pruritus Assessments at the Participant Level Using the Albireo ObsRo Instrument (AM Score) | Weeks 1-4, 5-8, 9-12, 13-16, 17-20, 21-24, 34-36, 44-46, 47-48, 58-60, 68-70, 71-72, and 73-76 | A positive pruritus assessment was defined as a scratching score of \<=1 or at least a 1-point drop from baseline on the Albireo ObsRO instrument. The percentage of positive pruritus assessment was calculated as the number of positive pruritus assessments divided by the total number of reported assessments in the time interval recorded by each participant multiplied by 100. AM score represents night-time itching/scratching and sleep disturbance. |
| Secondary | Percentage of Positive Pruritus Assessments at the Participant Level Using the Albireo ObsRo Instrument (PM Score) | Weeks 1-4, 5-8, 9-12, 13-16, 17-20, 21-24, 34-36, 44-46, 47-48, 58-60, 68-70, 71-72, and 73-76 | A positive pruritus assessment was defined as a scratching score of \<=1 or at least a 1-point drop from baseline on the Albireo ObsRO instrument. The percentage of positive pruritus assessment was calculated as the number of positive pruritus assessments divided by the total number of reported assessments in the time interval recorded by each participant multiplied by 100. PM score represents daytime itching/scratching and tiredness. |
| Secondary | Percentage of Responders for Pruritus Assessments Bi-Weekly (Combined AM and PM Scores) | Weeks 1-2, 3-4, 5-6, 7-8, 9-10, 11-12, 13-14, 15-16, 17-18, 19-20, 21-22, 23-24, 35-36, 47-48, 59-60, and 71-72 | A responder is defined as a participant who reports a decrease in pruritus score from unrounded baseline equivalent to or greater than the threshold of meaningful change of 1.0 estimated from the blinded psychometric analysis. The averaged pruritus score was used to calculate the percentage of participants achieving meaningful reduction against the threshold value of 1.0 based on bi-weekly scores. ObsRO instrument was used to assess severity of observed scratching twice a day (AM and PM) with score from 0 to 4 where 0 is no scratching and 4 is worst possible scratching. The data reported represent the combination of pruritus assessments from both AM and PM scores. |
1 to 10 of 21Source ClinicalTrials.gov
See all 21 registered outcomesOutcomes19
Each outcome the trial posted results for, in ClinicalTrials.gov's words.
| Type | Outcome | Time frame | Population | Units | Measured as |
|---|---|---|---|---|---|
| PRIMARY | Change From Baseline in Serum Bile Acids | Baseline and Week 72 | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at specified timepoints are reported. | micromole per liter (µmol/L) | MEAN |
| PRIMARY | Proportion of Positive Pruritus Assessments at the Participant Level Over 72-Week Using the Albireo Observer-Reported Outcome (ObsRo) Instrument | Baseline and Week 72 | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at Baseline and Week 72 are reported. | proportion of pruritus-participant-level | MEAN |
| SECONDARY | Proportion of Positive Pruritus Assessments at the Participant Level Using the Albireo ObsRo Instrument From Weeks 0-4, 0-12, 0-22, 0-24, 0-36, 0-46, 0-48, 0-60, and 0-70 | Weeks 0-4, Weeks 0-12, Weeks 0-22, Weeks 0-24, Weeks 0-36, Weeks 0-46, Weeks 0-48, Weeks 0-60, and Weeks 0-70 | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at specified timepoints are reported. | proportion of pruritus-participant-level | MEAN |
| SECONDARY | Change From Baseline in Serum Bile Acids at Weeks 4, 12, 22, 24, 36, 46, 48, 60, 70, and 72 | Baseline and Weeks 4, 12, 22, 24, 36, 46, 48, 60, 70, and 72 | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at specified timepoints are reported. | µmol/L | MEAN |
| SECONDARY | Proportion of Positive Pruritus Assessments at the Participant Level Using the Albireo ObsRo Instrument From Weeks 1-4, 5-8, 9-12, 13-16, 17-20, 21-24, 34-36, 44-46, 47-48, 58-60, 68-70, 71-72, 73-76 | Weeks 1-4, 5-8, 9-12, 13-16, 17-20, 21-24, 34-36, 44-46, 47-48, 58-60, 68-70, 71-72, 73-76 | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at specified timepoints are reported. | proportion of pruritus-participant-level | MEAN |
| SECONDARY | Proportion of Positive Pruritus Assessments at the Participant Level Using the Albireo ObsRo Instrument (AM Score) | Weeks 1-4, 5-8, 9-12, 13-16, 17-20, 21-24, 34-36, 44-46, 47-48, 58-60, 68-70, 71-72, and 73-76 | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at specified timepoints are reported. | proportion of pruritus-participant-level | MEAN |
| SECONDARY | Proportion of Positive Pruritus Assessments at the Participant Level Using the Albireo ObsRo Instrument (PM Score) | Weeks 1-4, 5-8, 9-12, 13-16, 17-20, 21-24, 34-36, 44-46, 47-48, 58-60, 68-70, 71-72, and 73-76 | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at specified timepoints are reported. | proportion of pruritus-participant-level | MEAN |
| SECONDARY | Percentage of Responders for Pruritus Assessments Bi-Weekly (AM and PM) | Weeks 1-2, 3-4, 5-6, 7-8, 9-10, 11-12, 13-14, 15-16, 17-18, 19-20, 21-22, 23-24, 35-36, 47-48, 59-60, and 71-72 | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at specified timepoints are reported. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Responders for Pruritus Assessments Monthly (AM and PM) | Weeks 1-4, 5-8, 9-12, 13-16, 17-20, 21-24, 34-36, 45-48, 58-60, and 68-72 | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with data collected at specified timepoints are reported. | percentage of participants | NUMBER |
| SECONDARY | Percentage of Participants Achieving a Positive Pruritus Assessment for >50% of the Time Based on the Albireo ObsRO (AM and PM) | Week 72 | The FAS consisted of all participants who had received at least 1 dose of the study treatment. Only participants with non-missing value when \>50% were included. | percentage of participants | NUMBER |
1 to 10 of 19Source ClinicalTrials.gov
See all 19 outcomesResults by group356
Each measurement the trial posted, group by group, in the words and numbers it posted.
| Outcome | Group | Category | Value | Spread | Units |
|---|---|---|---|---|---|
| Change From Baseline in Serum Bile Acids | Cohort 1: Placebo/Odevixibat | -104.00 | Standard Deviation 167.318 | micromole per liter (µmol/L) | |
| Change From Baseline in Serum Bile Acids | Cohort 1: Odevixibat/Odevixibat | -139.84 | Standard Deviation 172.070 | micromole per liter (µmol/L) | |
| Change From Baseline in Serum Bile Acids | Cohort 2: Odevixibat | -57.97 | Standard Deviation 137.990 | micromole per liter (µmol/L) | |
| Proportion of Positive Pruritus Assessments at the Participant Level Over 72-Week Using the Albireo Observer-Reported Outcome (ObsRo) Instrument | Cohort 1: Placebo/Odevixibat | 55.20 | Standard Deviation 38.733 | proportion of pruritus-participant-level | |
| Proportion of Positive Pruritus Assessments at the Participant Level Over 72-Week Using the Albireo Observer-Reported Outcome (ObsRo) Instrument | Cohort 1: Odevixibat/Odevixibat | 38.58 | Standard Deviation 34.877 | proportion of pruritus-participant-level | |
| Proportion of Positive Pruritus Assessments at the Participant Level Over 72-Week Using the Albireo Observer-Reported Outcome (ObsRo) Instrument | Cohort 2: Odevixibat | 77.28 | Standard Deviation 28.084 | proportion of pruritus-participant-level | |
| Proportion of Positive Pruritus Assessments at the Participant Level Using the Albireo ObsRo Instrument From Weeks 0-4, 0-12, 0-22, 0-24, 0-36, 0-46, 0-48, 0-60, and 0-70 | Cohort 1: Placebo/Odevixibat | Weeks 0 - 4 | 45.72 | Standard Deviation 34.300 | proportion of pruritus-participant-level |
| Proportion of Positive Pruritus Assessments at the Participant Level Using the Albireo ObsRo Instrument From Weeks 0-4, 0-12, 0-22, 0-24, 0-36, 0-46, 0-48, 0-60, and 0-70 | Cohort 1: Odevixibat/Odevixibat | Weeks 0 - 4 | 23.64 | Standard Deviation 27.005 | proportion of pruritus-participant-level |
| Proportion of Positive Pruritus Assessments at the Participant Level Using the Albireo ObsRo Instrument From Weeks 0-4, 0-12, 0-22, 0-24, 0-36, 0-46, 0-48, 0-60, and 0-70 | Cohort 2: Odevixibat | Weeks 0 - 4 | 60.14 | Standard Deviation 33.985 | proportion of pruritus-participant-level |
| Proportion of Positive Pruritus Assessments at the Participant Level Using the Albireo ObsRo Instrument From Weeks 0-4, 0-12, 0-22, 0-24, 0-36, 0-46, 0-48, 0-60, and 0-70 | Cohort 1: Placebo/Odevixibat | Weeks 0 - 12 | 51.05 | Standard Deviation 37.670 | proportion of pruritus-participant-level |
1 to 10 of 356Source ClinicalTrials.gov
See all 356 results by groupParticipant flow18
How many started, completed or left each group, period by period.
| Period | Milestone | Group | Participants | Units counted |
|---|---|---|---|---|
| Treatment Period (72 Weeks) | STARTED | Cohort 1: Placebo/Odevixibat | 19 | |
| Treatment Period (72 Weeks) | STARTED | Cohort 1: Odevixibat/Odevixibat | 37 | |
| Treatment Period (72 Weeks) | STARTED | Cohort 2: Odevixibat | 60 | |
| Treatment Period (72 Weeks) | COMPLETED | Cohort 1: Placebo/Odevixibat | 15 | |
| Treatment Period (72 Weeks) | COMPLETED | Cohort 1: Odevixibat/Odevixibat | 28 | |
| Treatment Period (72 Weeks) | COMPLETED | Cohort 2: Odevixibat | 40 | |
| Treatment Period (72 Weeks) | NOT COMPLETED | Cohort 1: Placebo/Odevixibat | 4 | |
| Treatment Period (72 Weeks) | NOT COMPLETED | Cohort 1: Odevixibat/Odevixibat | 9 | |
| Treatment Period (72 Weeks) | NOT COMPLETED | Cohort 2: Odevixibat | 20 | |
| Optional Extension Treatment (176 Weeks) | STARTED | Cohort 1: Placebo/Odevixibat | 14 |
1 to 10 of 18Source ClinicalTrials.gov
See all 18 participant flowAdverse events474
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 | Anaemia | Blood and lymphatic system disorders | Cohort 1: Placebo/Odevixibat | 0 | 19 | 0 |
| other | Anaemia | Blood and lymphatic system disorders | Cohort 1: Odevixibat/Odevixibat | 1 | 37 | 1 |
| other | Anaemia | Blood and lymphatic system disorders | Cohort 2: Odevixibat | 4 | 60 | 4 |
| other | Coagulopathy | Blood and lymphatic system disorders | Cohort 1: Placebo/Odevixibat | 0 | 19 | 0 |
| other | Coagulopathy | Blood and lymphatic system disorders | Cohort 1: Odevixibat/Odevixibat | 0 | 37 | 0 |
| other | Coagulopathy | Blood and lymphatic system disorders | Cohort 2: Odevixibat | 3 | 60 | 3 |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Cohort 1: Placebo/Odevixibat | 1 | 19 | 1 |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Cohort 1: Odevixibat/Odevixibat | 2 | 37 | 2 |
| other | Iron deficiency anaemia | Blood and lymphatic system disorders | Cohort 2: Odevixibat | 0 | 60 | 0 |
| other | Splenomegaly | Blood and lymphatic system disorders | Cohort 1: Placebo/Odevixibat | 2 | 19 | 2 |
1 to 10 of 474Source ClinicalTrials.gov
See all 474 adverse eventsAdverse event totals9
The totals the trial posted for each group.
| Total | Group | Affected | At risk |
|---|---|---|---|
| Total, all-cause mortality | Cohort 1: Placebo/Odevixibat | 0 | 19 |
| Total, all-cause mortality | Cohort 1: Odevixibat/Odevixibat | 0 | 37 |
| Total, all-cause mortality | Cohort 2: Odevixibat | 0 | 60 |
| Total, other adverse events | Cohort 1: Placebo/Odevixibat | 18 | 19 |
| Total, other adverse events | Cohort 1: Odevixibat/Odevixibat | 35 | 37 |
| Total, other adverse events | Cohort 2: Odevixibat | 57 | 60 |
| Total, serious adverse events | Cohort 1: Placebo/Odevixibat | 5 | 19 |
| Total, serious adverse events | Cohort 1: Odevixibat/Odevixibat | 7 | 37 |
| Total, serious adverse events | Cohort 2: Odevixibat | 23 | 60 |
Source ClinicalTrials.gov
Papers1
The papers the trial lists on ClinicalTrials.gov.
| PMID | Listed as |
|---|---|
| 37456676 | DERIVED |
Source PubMed
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 comparisons0
Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.
None in this release.
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.
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