A Phase 3 interventional study of Odevixibat and Placebo in Alagille Syndrome, sponsored by Albireo. Completed at 32 sites in 13 countries. Per ClinicalTrials.gov, last updated 2023-11-02.
Sponsored by Albireo (part of Ipsen) · Phase 3, Interventional, and Treatment
Double-blind, randomized, placebo-controlled, Phase 3 study to investigate the efficacy and safety of odevixibat compared to placebo in Patients with Alagille Syndrome.
Approximately 35 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.
31 studies on the registry are indexed under Alagille Syndrome; 11 are open to participants now.
This study's enrollment of 52 is above the median of 31 across 15 interventional studies indexed under Alagille Syndrome.
Browse Alagille Syndrome studies →Albireo is the lead sponsor of 11 studies on the registry; none are open to participants now.
First submitted before Albireo became part of Ipsen.
Counted across the registry records on this site, refreshed daily.
Key Inclusion Criteria:
Key Exclusion Criteria:
Capsules for oral administration once daily for 24 weeks.
Drug: Odevixibat
Capsules for oral administration (to match active) once daily for 24 weeks.
Drug: Placebo
Odevixibat is a small molecule and selective inhibitor of IBAT.
Also known as: A4250
Placebo identical in appearance to experimental drug (odevixibat).
Change From Baseline in Scratching Score
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.
Time frame: 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.
Serum Bile Acid Levels
Change in serum bile acid levels (μmol/L) from baseline to average of week 20 and 24
Time frame: 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.
| Milestone | Odevixibat (A4250) | Placebo |
|---|---|---|
| Started | 35 | 17 |
| Completed | 35 | 17 |
| Not completed | 0 | 0 |
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.
| score on a scale | Odevixibat (A4250) | Placebo |
|---|---|---|
| Change From Baseline in Scratching Score | -1.69 ± 0.174 | -0.8 ± 0.233 |
Change in serum bile acid levels (μmol/L) from baseline to average of week 20 and 24
| μmol/L | Odevixibat (A4250) | Placebo |
|---|---|---|
| Serum Bile Acid Levels | -90.35 ± 21.336 | 22.39 ± 28.463 |
Collected over The safety reporting period for adverse events is from the first dose of study drug through the last planned study visit or 28 calendar days after the last dose of the study drug, whichever occurs later, up to 203 days.. Non-serious events are listed at a 0% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| Odevixibat (A4250) | 0/35 (0%) | 5/35 (14.3%) | 26/35 (74.3%) |
| Placebo | 0/17 (0%) | 2/17 (11.8%) | 12/17 (70.6%) |
| Event | Odevixibat (A4250) | Placebo |
|---|---|---|
| PyrexiaGeneral disorders | 0/35 | 1/17 |
| Cerumen impactionEar and labyrinth disorders | 0/35 | 1/17 |
| Otitis media chronicInfections and infestations | 0/35 | 1/17 |
| Subcutaneous abscessInfections and infestations | 0/35 | 1/17 |
| Adenoidal hypertrophyRespiratory, thoracic and mediastinal disorders | 0/35 | 1/17 |
| International normalised ratio increasedInvestigations | 1/35 | 0/17 |
| Abdominal painGastrointestinal disorders | 1/35 | 0/17 |
| ConstipationGastrointestinal disorders | 1/35 | 0/17 |
| HaematemesisGastrointestinal disorders | 1/35 | 0/17 |
| PneumoniaInfections and infestations | 1/35 | 0/17 |
| Event | Odevixibat (A4250) | Placebo |
|---|---|---|
| DiarrhoeaGastrointestinal disorders | 10/35 | 1/17 |
| PyrexiaGeneral disorders | 8/35 | 4/17 |
| COVID-19Infections and infestations | 5/35 | 4/17 |
| Upper respiratory tract infectionInfections and infestations | 3/35 | 2/17 |
| International normalised ratio increasedInvestigations | 0/35 | 2/17 |
| EpistaxisRespiratory, thoracic and mediastinal disorders | 0/35 | 2/17 |
| Abdominal painGastrointestinal disorders | 4/35 | 1/17 |
| BronchitisInfections and infestations | 3/35 | 0/17 |
| Respiratory tract infectionInfections and infestations | 3/35 | 1/17 |
| CoughRespiratory, thoracic and mediastinal disorders | 3/35 | 1/17 |
| Age, Categorical(Participants) | Odevixibat (A4250) | Placebo | Total |
|---|---|---|---|
| <=18 years | 35 | 17 | 52 |
| Between 18 and 65 years | 0 | 0 | 0 |
| >=65 years | 0 | 0 | 0 |
| Age, Continuous(years) | Odevixibat (A4250) | Placebo | Total |
|---|---|---|---|
| Mean | 6.73 ± 3.780 | 5.40 ± 4.411 | 6.29 ± 4.003 |
| Age, Customized(Participants) | Odevixibat (A4250) | Placebo | Total |
|---|---|---|---|
| < 10 years | 29 | 13 | 42 |
| > = 10 years and < 18 years | 6 | 4 | 10 |
| Sex: Female, Male(Participants) | Odevixibat (A4250) | Placebo | Total |
|---|---|---|---|
| Female | 14 | 11 | 25 |
| Male | 21 | 6 | 27 |
| Race/Ethnicity, Customized(Participants) | Odevixibat (A4250) | Placebo | Total |
|---|---|---|---|
| White | 30 | 13 | 43 |
| Black or African American | 2 | 2 | 4 |
| Asian | 2 | 1 | 3 |
| American Indian or Alaska Native | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Other | 1 | 1 | 2 |
| Region of Enrollment(participants) | Odevixibat (A4250) | Placebo | Total |
|---|---|---|---|
| United States | 7 | 4 | 11 |
| United Kingdom | 1 | 0 | 1 |
| Malaysia | 2 | 1 | 3 |
| Netherlands | 3 | 3 | 6 |
| Turkey | 1 | 0 | 1 |
| Belgium | 1 | 1 | 2 |
| Poland | 7 | 3 | 10 |
| Italy | 4 | 2 | 6 |
| France | 3 | 1 | 4 |
| Germany | 6 | 2 | 8 |
| Genetic Mutation(Participants) | Odevixibat (A4250) | Placebo | Total |
|---|---|---|---|
| JAG1 | 32 | 16 | 48 |
| NOTCH2 | 3 | 1 | 4 |
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This study is completed, as verified in Oct 2023. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Odevixibat