Phase 3
N=52
Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome
Alagille Syndrome
Bottom Line
View on ClinicalTrials.gov: NCT04674761 ↗Enrolled (actual)
52
Serious AEs
13.5%
Results posted
Nov 2023
Primary outcome: Primary: Change From Baseline in Scratching Score — -1.69; -0.8 score on a scale — p=0.0012
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Odevixibat (Drug); Placebo (Drug)
- Age
- Pediatric, Adult, Older Adult
- Sex
- All
- Sponsor
- Albireo
- Primary completion
- Sep 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change From Baseline in Scratching Score |
-1.69; -0.8 | 0.0012 sig |
| SECONDARY Serum Bile Acid Levels |
-90.35; 22.39 | 0.0006 sig |
Summary
Double-blind, randomized, placebo-controlled, Phase 3 study to investigate the efficacy and safety of odevixibat compared to placebo in Patients with Alagille Syndrome.
Eligibility Criteria
Key Inclusion Criteria
- Genetically confirmed diagnosis of Alagille syndrome
- History of significant pruritus as measured by the Albireo Observer or Patient Reported Outcome instrument
- Elevated serum bile acid level
Key Exclusion Criteria
- History or ongoing presence of other types of liver disease (eg. biliary atresia, progressive familial intrahepatic cholestasis, hepatocellular carcinoma)
- History of liver transplant, or a liver transplant is planned within 6 months of randomization
- ALT >10× upper limit of normal (ULN) at screening
- Total bilirubin >15 × ULN at screening
- Patient suffers from uncontrolled, recalcitrant pruritic condition other than Alagille syndrome
Data sourced from ClinicalTrials.gov (NCT04674761). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.