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NCT Number: NCT04674761

Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome

Double-blind, randomized, placebo-controlled, Phase 3 study to investigate the efficacy and safety of odevixibat compared to placebo in Patients with Alagille Syndrome.

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Key information

Conditions

Alagille Syndrome Abnormalities, Multiple Bile Duct Diseases Biliary Tract Diseases Cardiovascular Abnormalities Cardiovascular Diseases Cholestasis Cholestasis, Intrahepatic Congenital Abnormalities Congenital, Hereditary, and Neonatal Diseases and Abnormalities Digestive System Diseases Genetic Diseases, Inborn Heart Defects, Congenital Liver Diseases

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 3

Primary location

Cliniques Universitaires Saint-Luc Bruxelles, Brussels, Belgium

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About this study

Approximately 35 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Odevixibat

Drug

Odevixibat is a small molecule and selective inhibitor of IBAT.

Other names: A4250

Placebo

Drug

Placebo identical in appearance to experimental drug (odevixibat).

Primary outcomes

  1. 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

  1. Serum Bile Acid Levels

    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.

    Change in serum bile acid levels (μmol/L) from baseline to average of week 20 and 24

Sponsors and collaborators

Lead sponsor

Albireo

Industry

Registry information

Official study title

A Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients With Alagille Syndrome (ASSERT)

Acronym: ASSERT

Important dates

Study start
2021
Primary completion
2022
Study completion
2022
First posted
Dec 19, 2020
Registry last updated
Nov 2, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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