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

Biliary Atresia Clinical Trial

Official 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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT04336722
• Other study ID #: A4250-011
• Status: Recruiting
• Phase: Phase 3
• Start date: July 8, 2020
• Est. completion date: July 31, 2026

Study information

• Verified date: May 2024
• Source: Ipsen
• Contact: Ipsen Clinical Study Enquiries
• Phone: See email
• Email: clinical.trials[@]ipsen.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Description:

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

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 245
• Est. completion date: July 31, 2026
• Est. primary completion date: June 30, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 111 Days

Eligibility

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

Related Conditions & MeSH terms

• Biliary Atresia

Intervention

Drug:
• Odevixibat
Odevixibat is a small molecule and selective inhibitor of IBAT.
• Placebo
Placebo identical in appearance to experimental drug (odevixibat).

Locations

Country	Name	City	State
Australia	Royal Children's Hospital	Parkville
Australia	The Children´s Hospital at Westmead	Sydney
Belgium	UZ Gent	Gent
Canada	CHU Sainte-Justine	Montréal
Canada	The Hospital for Sick Children	Toronto
China	Guangzhou Women and Children's Medical Center	Guangzhou	Guangdong
China	Children's Hospital of Fudan University	Shanghai
France	Hôpital Femme Mère Enfant	Bron
France	Bicêtre Hospital	Le Kremlin-Bicêtre
France	Jeanne de Flandre Hospital	Lille
France	Necker University Hospital - Enfants malades	Paris
Germany	Charité - Universitätsmedizin Berlin	Berlin
Germany	University Medical Center Hamburg-Eppendorf UKE	Hamburg
Germany	Hannover Medical School	Hanover
Germany	University Children´s Hospital Tuebingen	Tuebingen
Hungary	Semmelweis Egyetem I.sz Gyermekgyógyászati Klinika	Budapest
Israel	Schneider Children´s Medical Center of Israel	Petah tikva
Italy	ASST Papa Giovanni XXIII	Bergamo
Italy	Meyer Children´s University Hospital	Florence
Italy	University Hospital of Padova	Padova
Italy	ISMETT - Istituto Mediterraneo per i Trapianti e Terapie ad Alta Specializzazione	Palermo
Italy	Ospedale Pediatrico Bambino Gesù	Roma
Italy	Regina Margherita Children´s Hospital	Turin
Korea, Republic of	Samsung Medical Center	Seoul
Korea, Republic of	Seoul National University Children's Hospital	Seoul
Malaysia	Hospital Raja Perempuan Zainab II	Kota Bharu
Malaysia	University of Malaya Medical Centre	Kuala Lumpur
Netherlands	University Medical Center Groningen	Groningen
New Zealand	Starship Child Health	Auckland
Poland	Instytut Pomnik-Centrum Zdrowia Dziecka	Warsaw
Spain	Hospital Universitari Vall d'Hebron	Barcelona
Taiwan	National Taiwan University Hospital	Taipei
Turkey	Hacettepe University Ihsan Dogramaci Childrens Hospital	Ankara
Turkey	Akdeniz University Medical Faculty	Antalya
Turkey	Istanbul University, Istanbul Medical Faculty	Istanbul
United Kingdom	Birmingham Women´s and Children´s Hospital	Birmingham
United Kingdom	Leeds General Infirmary	Leeds
United States	University of Michigan Children's Hospital	Ann Arbor	Michigan
United States	Children's Healthcare of Atlanta - Emory University School of Medicine	Atlanta	Georgia
United States	Children's Hospital Colorado	Aurora	Colorado
United States	Johns Hopkins Children's Center	Baltimore	Maryland
United States	Boston Children's Hospital	Boston	Massachusetts
United States	The Children's Hospital at Montefiore	Bronx	New York
United States	Ann & Robert H. Lurie Children's Hospital	Chicago	Illinois
United States	Cincinnati Children's Hospital	Cincinnati	Ohio
United States	Cleveland Clinic	Cleveland	Ohio
United States	UT Southwestern Medical Center	Dallas	Texas
United States	Indiana University school of Medicine	Indianapolis	Indiana
United States	Children's Mercy Hospital	Kansas City	Missouri
United States	Children's Hospital Los Angeles	Los Angeles	California
United States	University of Miami	Miami	Florida
United States	Medical College of Wisconsin	Milwaukee	Wisconsin
United States	Icahn School of Medicine at Mount Sinai	New York	New York
United States	NewYork-Presbyterian Morgan Stanley Children's Hospital	New York	New York
United States	NYU Grossman school of Medicine	New York	New York
United States	Stanford Children's Health	Palo Alto	California
United States	The Children´s Hospital of Philadelphia	Philadelphia	Pennsylvania
United States	UPMC Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
United States	Oregon Health & Science University	Portland	Oregon
United States	Washington University in St. Louis	Saint Louis	Missouri
United States	University of Utah	Salt Lake City	Utah
United States	Rady Children's Hospital	San Diego	California
United States	UCSF Benioff Children's Hospital San Francisco	San Francisco	California
United States	Seattle Children's Hospital	Seattle	Washington
United States	Children's National Medical Center	Washington	District of Columbia
United States	Nemours/Alfred I. duPont Hospital for Children	Wilmington	Delaware

Sponsors (1)

Lead Sponsor	Collaborator
Albireo

Countries where clinical trial is conducted

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Time from randomization to first occurrence of liver transplant, or death		From baseline to Week 104
Secondary	Proportion of patients with liver transplant	Proportion of patients who are alive and have not undergone a liver transplant	From baseline to Week 104
Secondary	Time to onset of any sentinel events	Time to onset of any sentinel events	From baseline to Week 104
Secondary	Total bilirubin levels	Total bilirubin level after 13, 26, 52, and 104 weeks of study treatment	From baseline to Weeks 13, 26, 52 and 104
Secondary	Serum bile acid levels	Serum bile acid level after 13, 26, 52, and 104 weeks of study treatment	From baseline to Weeks 13, 26, 52 and 104
Secondary	Time to pediatric end-stage liver disease (PELD) score >15	Time to pediatric end-stage liver disease (PELD) score >15	From baseline to Week 104
Secondary	Percentage of participants experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)	An AE is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.	From baseline to Week 104
Secondary	Percentage of participants with clinically significant changes in Physical Examination	Percentage of participants with clinically significant changes in physical examination findings will be reported. The clinical significance will be graded by the investigator.	From baseline to Week 104
Secondary	Percentage of participants with clinically significant changes in Laboratory Parameters (blood chemistry, hematology and coagulation)	Percentage of participants with clinically significant change in laboratory parameters (blood chemistry, hematology and coagulation) will be reported. The clinical significance will be decided by the investigator.	From baseline to Week 104
Secondary	Percentage of participants with clinically significant changes in Abdominal Ultrasound findings	Percentage of participants with clinically significant change in Abdominal Ultrasound findings will be reported. The clinical significance will be decided by the investigator.	From baseline to Week 26 and Week 104