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. |
ClinicalTrials.gov Identifier: NCT04674761 |
Recruitment Status :
Active, not recruiting
First Posted : December 19, 2020
Last Update Posted : March 31, 2022
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Condition or disease | Intervention/treatment | Phase |
---|---|---|
Alagille Syndrome | Drug: Odevixibat Drug: Placebo | Phase 3 |
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 |

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). |
- 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
- Serum Bile Acid Levels [ Time Frame: From baseline to Week 24 ]Change in serum bile acid levels from baseline to Week 24
- 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)

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.
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

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 ClinicalTrials.gov identifier (NCT number): NCT04674761

Responsible Party: | Albireo |
ClinicalTrials.gov 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 |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
ALGS Alagille syndrome |
Alagille Syndrome Syndrome Disease Pathologic Processes Cholestasis, Intrahepatic Cholestasis 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 |