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

• Register: EUCTR
• Last refreshed on: 15 July 2024
• Main ID: EUCTR2019-003807-37-BE
• Date of registration: 18/06/2020
• Prospective Registration: Yes
• Primary sponsor: Albireo AB
• Public title: This Study Will Investigate the Efficacy and Safety of Odevixibat in Children with Biliary Atresia Who Have Undergone a Kasai HPE
• Scientific title: 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)
• Date of first enrolment: 08/09/2020
• Target sample size: 245
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2019-003807-37
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2
• Phase: Human pharmacology (Phase I): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

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

Contacts

• Name: Clinical Operations Department
• Affiliation: Albireo AB

• Name: Clinical Operations Department
• Affiliation: Albireo AB

Key inclusion & exclusion 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 treatment within 3 weeks post-Kasai HPE
Are the trial subjects under 18? yes
Number of subjects for this age range: 245
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:
- Patients with intractable ascites
- Ileal resection surgery
- ALT =10× upper limit of normal (ULN) at screening
- Patient on total parenteral nutrition, or not able to take study drug 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


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

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

Intervention(s)

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)
Concentration type: equal
Concentration number: 200-
Pharmaceutical form of the placebo: Capsule, hard
Route of administration of the placebo: Oral use

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)
Concentration type: equal
Concentration number: 600-
Pharmaceutical form of the placebo: Capsule, hard
Route of administration of the placebo: Oral use

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)
Concentration type: equal
Concentration number: 200-
Pharmaceutical form of the placebo: Capsule, hard
Route of administration of the placebo: Oral use

Primary Outcome(s)

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 native liver survival
(NLS) of up to 104 weeks.

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
To evaluate the effect of odevixibat compared to placebo on serum bile
acids after 13, 26, 52, and 104 weeks
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

Timepoint(s) of evaluation of this end point: 104 weeks

Primary end point(s): The primary efficacy endpoint is the time from randomization to first
occurrence of liver transplant, or death, during the 104 week treatment
period.

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: 13, 26, 52 and 104 weeks.

Secondary end point(s): 1. Proportion of patients who are alive and have not undergone a liver
transplant after 104 weeks.
2. Time to onset of first sentinel event during the 104-week treatment
period. Sentinel events are defined in the protocol
3. Total bilirubin level after 13, 26, 52, and 104 weeks.
4. Serum bile acid level after 13, 26, 52, and 104 weeks.
5. Time to pediatric end-stage liver disease (PELD) score =15.
6. Safety parameters including AEs, SAEs, findings on physical
examination, laboratory assessments (including fat-soluble vitamins and
lipids) and abdominal ultrasound.

Secondary ID(s)

141677

A4250-011

2019-003807-37-HU

NCT04336722

Source(s) of Monetary Support

Albireo AB

Ethics review

Status: Approved
Approval date: 08/09/2020
Contact: