A Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients With Alagille Syndrome (ASSERT)
Overview
- Phase
- Phase 3
- Status
- Completed
- Sponsor
- Albireo
- Enrollment
- 52
- Locations
- 32
- Primary Endpoint
- Change From Baseline in Scratching Score
Overview
Brief 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.
Detailed Description
Approximately 35 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.
Study Design
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel
- Primary Purpose
- Treatment
- Masking
- Triple (Participant, Care Provider, Investigator)
Eligibility Criteria
- Sex
- All
- Accepts Healthy Volunteers
- No
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
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
Arms & Interventions
Odevixibat (A4250)
Capsules for oral administration once daily for 24 weeks.
Intervention: Odevixibat (Drug)
Placebo
Capsules for oral administration (to match active) once daily for 24 weeks.
Intervention: Placebo (Drug)
Outcomes
Primary Outcomes
Change From Baseline in Scratching Score
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.
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 Outcomes
- 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.)