This Study Will Investigate the Efficacy and Safety of Odevixibat in Children with Biliary Atresia Who Have Undergone a Kasai HPE

A Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Odevixibat (A4250) in Children with Biliary Atresia Who Have Undergone a Kasai Hepatoportoenterostomy (BOLD)

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2019-003807-37-HU

Enrollment

200

Registered

2020-04-17

Start date

2020-07-29

Completion date

Unknown

Last updated

2024-07-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Biliary Atresia MedDRA version: 20.0 Level: LLT Classification code 10004653 Term: Biliary atresia System Organ Class: 100000004850

Interventions

Product Name: Odevixibat Product Code: A4250 Pharmaceutical Form: Capsule, hard INN or Proposed INN: Odevixibat CAS Number: 501692-44-0 Current Sponsor code: A4250 Concentration unit: µg microgram(s)

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: 1. A male or female patient with a clinical diagnosis of BA 2. Age at Kasai HPE =90 days 3. Eligible to start study treatment within 3 weeks post-Kasai HPE Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range

Exclusion criteria

Exclusion criteria: 1. Patients with intractable ascites 2. Ileal resection surgery 3. ALT =10× upper limit of normal (ULN) at screening 4. Patient on total parenteral nutrition at randomization 5. Acute ascending cholangitis (patients may be randomized after resolution of acute ascending cholangitis) 6. Choledochal cystic disease

Design outcomes

Primary

Measure	Time frame
Main Objective: The primary objective is to evaluate the efficacy of repeated once-daily doses of odevixibat versus placebo in children with biliary atresia (BA) post Kasai hepatoportoenterostomy(HPE) based on survival with native liver after 104 weeks of study treatment.;Secondary Objective: To evaluate the effect of odevixibat compared to placebo on the time to onset of sentinel events To evaluate the effect of odevixibat compared to placebo on total bilirubin after 13, 26, 52, and 104 weeks of study treatment To evaluate the effect of odevixibat compared to placebo on serum bile acids after 13, 26, 52, and 104 weeks of study treatment To assess the long-term safety and tolerability of repeated daily doses of odevixibat compared to placebo for 104 weeks in children with BA post Kasai HPE;Primary end point(s): The primary efficacy endpoint is the proportion of patients who are alive and have not undergone a liver transplant after 104 weeks of study treatment.;Timepoint(s) of evaluation of this end point: 104 weeks	—

Measure

Time frame

Main Objective: The primary objective is to evaluate the efficacy of repeated once-daily doses of odevixibat versus placebo in children with biliary atresia (BA) post Kasai hepatoportoenterostomy(HPE) based on survival with native liver after 104 weeks of study treatment.;Secondary Objective: To evaluate the effect of odevixibat compared to placebo on the time to onset of sentinel events To evaluate the effect of odevixibat compared to placebo on total bilirubin after 13, 26, 52, and 104 weeks of study treatment To evaluate the effect of odevixibat compared to placebo on serum bile acids after 13, 26, 52, and 104 weeks of study treatment To assess the long-term safety and tolerability of repeated daily doses of odevixibat compared to placebo for 104 weeks in children with BA post Kasai HPE;Primary end point(s): The primary efficacy endpoint is the proportion of patients who are alive and have not undergone a liver transplant after 104 weeks of study treatment.;Timepoint(s) of evaluation of this end point: 104 weeks

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Secondary

Measure	Time frame
Secondary end point(s): 1. Time to onset of any sentinel events. 2. Total bilirubin level after 13, 26, 52, and 104 weeks of study treatment. 3. Serum bile acid level after 13, 26, 52, and 104 weeks of study treatment. 4. Safety parameters including AEs, SAEs, findings on physical examination, laboratory assessments and abdominal ultrasound, and stool frequency.;Timepoint(s) of evaluation of this end point: 13, 26, 52 and 104 weeks.	—

Measure

Time frame

Secondary end point(s): 1. Time to onset of any sentinel events. 2. Total bilirubin level after 13, 26, 52, and 104 weeks of study treatment. 3. Serum bile acid level after 13, 26, 52, and 104 weeks of study treatment. 4. Safety parameters including AEs, SAEs, findings on physical examination, laboratory assessments and abdominal ultrasound, and stool frequency.;Timepoint(s) of evaluation of this end point: 13, 26, 52 and 104 weeks.

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Countries

Australia, Belgium, Canada, China, Denmark, France, Germany, Hungary, Israel, Italy, Korea, Republic of, Malaysia, Netherlands, New Zealand, Poland, Spain, Taiwan, Turkey, United Kingdom, United States

Contacts

Public ContactClinical Operations Department

Albireo AB

medinfo@albireopharma.com+46317411480

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026