Efficacy and Safety of Odevixibat in Children With Biliar... | NCT04336722

Studies
Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)

NCT04336722

Sponsor

Albireo, an Ipsen Company

Status

Completed

Last Update Posted

Jul 2, 2026Actual

Enrollment

254Actual

Phase

Phase 3

Conditions

Biliary Atresia

Interventions

Odevixibat
Placebo

Countries

United States
Australia
Belgium
Canada
China
France
Germany
Hungary
Israel
Italy
Malaysia
Netherlands
New Zealand
Poland
South Korea
Spain
Taiwan
Turkey (Türkiye)
United Kingdom

Protocol Section

Identification Module Status Module Sponsor/Collaborators Module Oversight Module Description Module Conditions Module Design Module Arms/Interventions Module Outcomes Module Eligibility Module Contacts/Locations Module References Module IPD Sharing Statement Module

Results Section

Participant Flow Module Baseline Characteristics Module Outcome Measures Module Adverse Events Module More Info Module

Annotation Section

Annotation Module

Document Section

Large Document Module

Derived Section

Miscellaneous Info Module Condition Browse Module Intervention Browse Module

Protocol Section

Identification Module

NCT ID

ID	Type	Description	Link
2019-003807-37	EudraCT Number
2024-512086-14-00	EU Trial (CTIS) Number

Status Module

Record Verification Date

Jul 2026

Overall Recruitment Status or Expanded Access Status

Completed

Last Known Status

Not provided

Delayed Posting

Not provided

Why Stopped

Not provided

Expanded Access Info

Start Date

Jul 8, 2020Actual

Primary Completion Date

Jun 22, 2026Actual

Completion Date

Jun 22, 2026Actual

First Submitted Date

Mar 27, 2020

First Submission Date that Met QC Criteria

Apr 3, 2020

First Posted Date

Apr 7, 2020Actual

Results Waived

Not provided

Results First Submitted Date

Not provided

Results First Submitted that Met QC Criteria

Not provided

Results First Posted Date

Not provided

Certification/Extension (aka Delayed Results) First Submitted Date

Not provided

Certification/Extension First Submitted that Passed QC Review

Not provided

Certification/Extension First Posted Date

Not provided

Last Update Submitted Date

Jul 1, 2026

Last Update Posted Date

Jul 2, 2026Actual

Oversight Module

Has Data Monitoring Committee (DMC)

Yes

Is FDA Regulated Drug

Yes

Is FDA Regulated Device

Is Unapproved Device

Not provided

Pediatric Postmarket Surveillance of a Device Product

Not provided

Product Exported from US

Not provided

FDAAA801 Violation

Not provided

Description Module

Brief Summary

Double-blind, randomized, placebo-controlled, Phase 3 study to investigate the efficacy and safety of odevixibat compared to placebo in children with biliary atresia who have undergone a Kasai hepatoportoenterostomy.

Detailed Description

Up to 70 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.

Design Module

Study Type

Interventional

Number of References to an Expanded Access Study

Not provided

Expanded Access Types

Not provided

Patient Registry

Not provided

Target Follow-Up Duration

Not provided

Phases

Phase 3

Interventional Study Design

Allocation

Biospecimen

No data available

No data is available for this block.

Enrollment

254Actual

Arms/Interventions Module

Arm Groups

Label	Type	Description	Intervention Names
Odevixibat (A4250)	Experimental	Capsules for oral administration once daily for 104 weeks.	Drug: Odevixibat
Placebo	Placebo Comparator	Capsules for oral administration (to match active) once daily for 104 weeks.	Drug: Placebo

Interventions

Name	Type	Description	Arm Group Labels	Other Names
Odevixibat	Drug	Odevixibat is a small molecule and selective inhibitor of IBAT.	Odevixibat (A4250)

Outcomes Module

Primary Outcomes

Measure	Description	Time Frame
Time from randomization to first occurrence of liver transplant, or death		From baseline to Week 104

Secondary Outcomes

Measure	Description	Time Frame
Proportion of patients with liver transplant	Proportion of patients who are alive and have not undergone a liver transplant	From baseline to Week 104
Time to onset of any sentinel events	Time to onset of any sentinel events

Other Outcomes

Not provided

Eligibility Module

Eligibility Criteria

Inclusion Criteria:

A male or female patient with a clinical diagnosis of BA
Age at Kasai HPE ≤90 days
Eligible to start study treatment within 3 weeks post-Kasai HPE

Key Exclusion Criteria:

Patients with intractable ascites
Ileal resection surgery
ALT ≥10× upper limit of normal (ULN) at screening
Patients reliant only on total parenteral nutrition, or not able to take study medication orally, at randomization
Acute ascending cholangitis (patients may be randomized after resolution of acute ascending cholangitis)
Choledochal cystic disease
INR >1.6 (the patient may be treated with Vitamin K intravenously; sample may be redrawn and if INR is ≤1.6 at resampling the patient may be randomized)
Any other conditions or abnormalities, including congenital abnormalities, major cardiac surgery, hepatic, biliary, or GI disease which, in the opinion of the Investigator or Medical Monitor, may compromise the safety of the patient, the integrity of study results, or patient compliance with study requirements
Weight <3.5kg at randomization

Accepts Healthy Volunteers

Sex

All

Sex/Gender Based

Not provided

Sex/Gender Description

Not provided

Minimum Age

Not provided

Maximum Age

111 Days

Standard Ages

Child

Study Population

Not provided

Sampling Method

Not provided

Contacts/Locations Module

Central Contacts

Not provided

Overall Officials

Name	Affiliation	Role
Ipsen Medical Director	Ipsen	Study Director

Locations

Facility	Status	City	State	ZIP	Country	Contacts
Children's Hospital Los Angeles		Los Angeles	California	90027	United States
Stanford Children's Health

IPD Sharing Statement Module

Plan to Share IPD

Yes

Description

Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications. Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants.

Types

Not provided

Time Frame

Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and/or EU.

Access Criteria

Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).

URL

https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/

ID	Term
D001649	Bile Duct Diseases
D001660	Biliary Tract Diseases
D004066	Digestive System Diseases
D004065	Digestive System Abnormalities
D000013	Congenital Abnormalities
D009358	Congenital, Hereditary, and Neonatal Diseases and Abnormalities