Trial · NCT04674761

Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome

NCT04674761, Phase 3, Completed, Albireo; results first posted 2023-11-02

3 sourcestrials-2026-11-12-0.5.0

Cite this record

ConductScience Library. Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome. Trials, record NCT04674761. Release trials-2026-11-12-0.5.0, 12 Nov 2026. https://conductscience.org/library/trials/NCT04674761

On this page

Trial

As registered on ClinicalTrials.gov, in the graph's capture of the registry.

Title: Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome
NCT number: NCT04674761
Status: Completed
Phases: Phase 3
Enrollment: 52 participants, actual
Lead sponsor: Albireo
Conditions: Alagille Syndrome
Interventions: Odevixibat, Placebo
First posted: 2020-12-19
Last updated: 2023-11-02
Results first posted: 2023-11-02

Source ClinicalTrials.gov

Registered outcomes2

Each outcome the trial registered, primary first, in ClinicalTrials.gov's words.

List	Outcome	Time frame	Description
Primary	Change From Baseline in Scratching Score	Change from baseline for each four-week average pruritis score to Month 6 (Weeks 21 to 24), in which baseline was calculated based on the 14 days before the start of treatment.	Change from baseline in average AM (measured after waking up) and PM (measured before bedtime) scratching score to Month 6 as measured by the Albireo Observer-Reported Outcome (ObsRO) Instrument. The ObsRO instrument was used to assess severity of observed scratching twice a day (AM and PM) with scores from 0 to 4 where 0 is no scratching and 4 is worst possible scratching.
Secondary	Serum Bile Acid Levels	Change from baseline to average of week 20 and 24, where baseline was calculated by averaging the last two values preceding start of treatment, and average of Week 20 and Week 24 was defined as the average of Week 20 and Week 24 values.	Change in serum bile acid levels (μmol/L) from baseline to average of week 20 and 24

Source ClinicalTrials.gov

Outcomes2

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 Scratching Score	Change from baseline for each four-week average pruritis score to Month 6 (Weeks 21 to 24), in which baseline was calculated based on the 14 days before the start of treatment.		score on a scale	LEAST_SQUARES_MEAN
SECONDARY	Serum Bile Acid Levels	Change from baseline to average of week 20 and 24, where baseline was calculated by averaging the last two values preceding start of treatment, and average of Week 20 and Week 24 was defined as the average of Week 20 and Week 24 values.		μmol/L	LEAST_SQUARES_MEAN

Source ClinicalTrials.gov

Results by group4

Each measurement the trial posted, group by group, in the words and numbers it posted.

Outcome	Group	Value	Spread	Units
Change From Baseline in Scratching Score	Odevixibat (A4250)	-1.69	Standard Error 0.174	score on a scale
Change From Baseline in Scratching Score	Placebo	-0.8	Standard Error 0.233	score on a scale
Serum Bile Acid Levels	Odevixibat (A4250)	-90.35	Standard Error 21.336	μmol/L
Serum Bile Acid Levels	Placebo	22.39	Standard Error 28.463	μmol/L

Source ClinicalTrials.gov

Participant flow6

How many started, completed or left each group, period by period.

Period	Milestone	Group	Participants
Overall Study	STARTED	Odevixibat (A4250)	35
Overall Study	STARTED	Placebo	17
Overall Study	COMPLETED	Odevixibat (A4250)	35
Overall Study	COMPLETED	Placebo	17
Overall Study	NOT COMPLETED	Odevixibat (A4250)	0
Overall Study	NOT COMPLETED	Placebo	0

Source ClinicalTrials.gov

Adverse events154

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 macrocytic	Blood and lymphatic system disorders	Odevixibat (A4250)	1	35	1
other	Anaemia macrocytic	Blood and lymphatic system disorders	Placebo	0	17	0
other	Coagulopathy	Blood and lymphatic system disorders	Odevixibat (A4250)	1	35	1
other	Coagulopathy	Blood and lymphatic system disorders	Placebo	0	17	0
other	Lymphadenopathy	Blood and lymphatic system disorders	Odevixibat (A4250)	1	35	1
other	Lymphadenopathy	Blood and lymphatic system disorders	Placebo	0	17	0
other	Cataract cortical	Eye disorders	Odevixibat (A4250)	1	35	1
other	Cataract cortical	Eye disorders	Placebo	0	17	0
other	Abdominal pain	Gastrointestinal disorders	Odevixibat (A4250)	4	35	4
other	Abdominal pain	Gastrointestinal disorders	Placebo	1	17	1

1 to 10 of 154Source ClinicalTrials.gov

See all 154 adverse events

Adverse event totals6

The totals the trial posted for each group.

Total	Group	Affected	At risk
Total, all-cause mortality	Odevixibat (A4250)	0	35
Total, all-cause mortality	Placebo	0	17
Total, other adverse events	Odevixibat (A4250)	26	35
Total, other adverse events	Placebo	12	17
Total, serious adverse events	Odevixibat (A4250)	5	35
Total, serious adverse events	Placebo	2	17

Source ClinicalTrials.gov

Papers1

The papers the trial lists on ClinicalTrials.gov.

PMID	Listed as
38670135	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 comparisons2

Comparisons of the trial's two groups, calculated by ConductScience from the counts it posted.

Comparison	Source
Odevixibat (A4250) vs Placebo: deaths from any cause	Trial comparisons
Odevixibat (A4250) 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.

ClinicalTrials.gov: 2026-11-12; ClinicalTrials.gov
Results, from AACT: 2026-03-23; AACT
NIH grant links: 2026-11-13; NIH RePORTER

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/NCT04674761

GET https://conductscience.org/api/trials/v1/NCT04674761/events?page=2

API reference For AI assistants