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Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome (ASSERT)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT04674761
Recruitment Status : Active, not recruiting
First Posted : December 19, 2020
Last Update Posted : March 31, 2022
Information provided by (Responsible Party):

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.

Condition or disease Intervention/treatment Phase
Alagille Syndrome Drug: Odevixibat Drug: Placebo Phase 3

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 63 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Official Title: A Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients With Alagille Syndrome (ASSERT)
Actual Study Start Date : March 19, 2021
Estimated Primary Completion Date : October 2022
Estimated Study Completion Date : October 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Safety

Arm Intervention/treatment
Experimental: Odevixibat (A4250)
Capsules for oral administration once daily for 24 weeks.
Drug: Odevixibat
Odevixibat is a small molecule and selective inhibitor of IBAT.
Other Name: A4250

Placebo Comparator: Placebo
Capsules for oral administration (to match active) once daily for 24 weeks.
Drug: Placebo
Placebo identical in appearance to experimental drug (odevixibat).

Primary Outcome Measures :
  1. Change from Baseline in Scratching Score [ Time Frame: From baseline to Week 24 ]
    Change from baseline in scratching score to Month 6 as measured by the Albireo Observer-Reported Outcome scratching score

Secondary Outcome Measures :
  1. Serum Bile Acid Levels [ Time Frame: From baseline to Week 24 ]
    Change in serum bile acid levels from baseline to Week 24

  2. Safety and Tolerability [ Time Frame: From first dose of study drug up to 28 days after last dose of study drug or to the completion of study participation date, whichever occurs first ]
    Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT04674761

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Sponsors and Collaborators
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Responsible Party: Albireo Identifier: NCT04674761    
Other Study ID Numbers: A4250-012
First Posted: December 19, 2020    Key Record Dates
Last Update Posted: March 31, 2022
Last Verified: March 2022

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Albireo:
Alagille syndrome
Additional relevant MeSH terms:
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Alagille Syndrome
Pathologic Processes
Cholestasis, Intrahepatic
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Liver Diseases
Heart Defects, Congenital
Cardiovascular Abnormalities
Cardiovascular Diseases
Abnormalities, Multiple
Congenital Abnormalities
Genetic Diseases, Inborn