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: 29 July 2024
• Main ID: EUCTR2019-003807-37-HU
• Date of registration: 17/04/2020
• 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: 29/07/2020
• Target sample size: 200
• 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, Denmark, 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 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:
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



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)

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.

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.

Timepoint(s) of evaluation of this end point: 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 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

Secondary Outcome(s)

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.

Secondary ID(s)

A4250-011

Source(s) of Monetary Support

Albireo AB

Ethics review

Status: Approved
Approval date: 29/07/2020
Contact: