Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome

Albireo52 enrolled

Overview

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.

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

Conditions

Alagille Syndrome

Interventions

OdevixibatDRUG

Odevixibat is a small molecule and selective inhibitor of IBAT.

PlaceboDRUG

Placebo identical in appearance to experimental drug (odevixibat).

Eligibility

Sex: ALL

Medical Language ↔ Plain English

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

Locations (32)

Outcomes

Primary Outcomes

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.

Secondary Outcomes

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.

Data from ClinicalTrials.gov

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University of California - San Francisco

San Francisco, California, United States

Johns Hopkins Hospital

Baltimore, Maryland, United States

Boston Children's Hospital

Boston, Massachusetts, United States

Children's Mercy Hospital and Clinics

Kansas City, Missouri, United States

Icahn School of Medicine at Mount Sinai

New York, New York, United States

Columbia University Medical Center

New York, New York, United States

The Childrens Hospital at Montefiore Albert Einstein School of Medicine

The Bronx, New York, United States

Oregon Health Science University School of Medicine

Portland, Oregon, United States

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States

Cliniques Universitaires Saint-Luc Bruxelles

Brussels, Belgium

...and 22 more locations