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

March 27, 2024 updated by: Albireo

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

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Detailed Description

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

Study Type

Interventional

Enrollment (Estimated)

245

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Australia
      • Parkville, Australia, 3052
        • Recruiting
        • Royal Children's Hospital
        • Contact:
          • Winita Hardikar, PhD
      • Sydney, Australia
        • Recruiting
        • The Children´s Hospital at Westmead
        • Contact:
          • Michael Stormon, MD
  • Belgium
      • Gent, Belgium
        • Recruiting
        • UZ Gent
        • Contact:
          • Ruth De Bruyne, MD, PhD
  • Canada
      • Montréal, Canada
        • Recruiting
        • CHU Sainte-Justine
        • Contact:
          • Fernando Alvarez, MD
      • Toronto, Canada
        • Recruiting
        • The Hospital for Sick Children
        • Contact:
          • Vicky Ng, MD
  • China
      • Shanghai, China
        • Active, not recruiting
        • Children's Hospital of Fudan University
    • Guangdong
      • Guangzhou, Guangdong, China
        • Recruiting
        • Guangzhou Women and Children's Medical Center
        • Contact:
          • Jiakang Yu
  • France
      • Bron, France
        • Recruiting
        • Hôpital Femme Mère Enfant
        • Contact:
          • Mathias Ruiz, MD
      • Le Kremlin-Bicêtre, France
        • Recruiting
        • Bicetre Hospital
        • Contact:
          • Emmanuel Gonzales, MD, PhD
      • Lille, France
        • Recruiting
        • Jeanne de Flandre Hospital
        • Contact:
          • Madeleine Aumar, MD
      • Paris, France
        • Recruiting
        • Necker University Hospital - Enfants malades
        • Contact:
          • Florence Lacaille, MD
  • Germany
      • Berlin, Germany
        • Recruiting
        • Charite - Universitatsmedizin Berlin
        • Contact:
          • Philip Bufler, MD
      • Hamburg, Germany
        • Recruiting
        • University Medical Center Hamburg-Eppendorf UKE
        • Contact:
          • Konrad Reinshagen, MD
      • Hanover, Germany
        • Recruiting
        • Hannover Medical School
        • Contact:
          • Ulrich Baumann, MD
      • Tuebingen, Germany
        • Recruiting
        • University Children´s Hospital Tuebingen
        • Contact:
          • Ekkehard Sturm, MD, PhD
  • Hungary
      • Budapest, Hungary
        • Recruiting
        • Semmelweis Egyetem I.sz Gyermekgyógyászati Klinika
        • Contact:
          • Antal Dezsöfi, MD, PhD
  • Israel
      • Petah tikva, Israel
        • Recruiting
        • Schneider Children´s Medical Center of Israel
        • Contact:
          • Orith Waisbourd-Zinman, MD
  • Italy
      • Bergamo, Italy
        • Recruiting
        • Asst Papa Giovanni Xxiii
        • Contact:
          • Lorenzo D'Antiga, MD
      • Florence, Italy
        • Recruiting
        • Meyer Children´s University Hospital
        • Contact:
          • Giuseppe Indolfi, MD, PhD
      • Padova, Italy
        • Recruiting
        • University Hospital of Padova
        • Contact:
          • Mara Cananzi, MD, PhD
      • Palermo, Italy
        • Recruiting
        • ISMETT - Istituto Mediterraneo per i Trapianti e Terapie ad Alta Specializzazione
        • Contact:
          • Silvia Riva, MD
      • Roma, Italy
        • Recruiting
        • Ospedale Pediatrico Bambino Gesù
        • Contact:
          • Giuseppe Maggiore, MD
      • Turin, Italy
        • Recruiting
        • Regina Margherita Children´s Hospital
        • Contact:
          • Pier Luigi Calvo, MD
  • Korea, Republic of
      • Seoul, Korea, Republic of
        • Recruiting
        • Samsung Medical Center
        • Contact:
          • Yon Ho Choe, MD
      • Seoul, Korea, Republic of
        • Recruiting
        • Seoul National University Children's Hospital
        • Contact:
          • Joong Kee Youn, MD
        • Principal Investigator:
          • Joong Kee Youn, MD
  • Malaysia
      • Kota Bharu, Malaysia
        • Recruiting
        • Hospital Raja Perempuan Zainab II
        • Contact:
          • Wan Mohd Ruzaimie Wan Mohamad Noor, MD
      • Kuala Lumpur, Malaysia
        • Recruiting
        • University of Malaya Medical Centre
        • Contact:
          • Way Seah Lee, MD
  • Netherlands
      • Groningen, Netherlands
        • Recruiting
        • University Medical Center Groningen
        • Contact:
          • Hendrik Jan Verkade, MD, PhD
  • New Zealand
      • Auckland, New Zealand
        • Recruiting
        • Starship Child Health
        • Contact:
          • Helen Evans, MD
  • Poland
      • Warsaw, Poland
        • Recruiting
        • Instytut Pomnik-Centrum Zdrowia Dziecka
        • Contact:
          • Piotr Czubkowski, MD, PhD
  • Spain
      • Barcelona, Spain
        • Recruiting
        • Hospital Universitari Vall d'Hebron
        • Contact:
          • Jesús Quintero, MD, PhD
  • Taiwan
      • Taipei, Taiwan
        • Recruiting
        • National Taiwan University Hospital
        • Contact:
          • Huey-Ling Chen, PhD, MD
  • Turkey
      • Ankara, Turkey
        • Recruiting
        • Hacettepe University Ihsan Dogramaci Childrens Hospital
        • Contact:
          • Hasan Özen, MD
      • Antalya, Turkey
        • Recruiting
        • Akdeniz University Medical Faculty
        • Contact:
          • Reha Artan, MD
      • Istanbul, Turkey
        • Recruiting
        • Istanbul University, Istanbul Medical Faculty
        • Contact:
          • Ozlem Durmaz, MD
  • United Kingdom
      • Birmingham, United Kingdom
        • Recruiting
        • Birmingham Women´s and Children´s Hospital
        • Contact:
          • Chayarani Kelgeri, MD
      • Leeds, United Kingdom
        • Recruiting
        • Leeds General Infirmary
        • Contact:
          • Naved Alizai
  • United States
    • California
      • Los Angeles, California, United States, 90027
        • Recruiting
        • Children's Hospital Los Angeles
        • Contact:
          • Rohit Kohli, MD
      • Palo Alto, California, United States, 94304
        • Recruiting
        • Stanford Children's Health
        • Contact:
          • Amrita Narang, MD
      • San Diego, California, United States, 92123
        • Recruiting
        • Rady Children's Hospital
        • Contact:
          • Laura Bauman, MD
      • San Francisco, California, United States, 94158
        • Recruiting
        • UCSF Benioff Children's Hospital San Francisco
        • Contact:
          • Phillip Rosenthal, MD
    • Colorado
      • Aurora, Colorado, United States, 80045
        • Recruiting
        • Children's Hospital Colorado
        • Contact:
          • Shikha Sundaram, MD
    • Delaware
      • Wilmington, Delaware, United States, 19803
        • Recruiting
        • Nemours/Alfred I. DuPont Hospital for Children
        • Contact:
          • Adebowale Adeyemi, MD
    • District of Columbia
      • Washington, District of Columbia, United States, 20010
        • Terminated
        • Children's National Medical Center
    • Florida
      • Miami, Florida, United States, 33146
        • Recruiting
        • University of Miami
        • Contact:
          • Jennifer Garcia, MD
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Recruiting
        • Children's Healthcare of Atlanta - Emory University School of Medicine
        • Contact:
          • Saul Karpen, MD, PhD
    • Illinois
      • Chicago, Illinois, United States, 60611
        • Recruiting
        • Ann & Robert H. Lurie Children's Hospital
        • Contact:
          • Alyssa Kriegermeier, MD
        • Principal Investigator:
          • Alyssa Kriegermeier, MD
    • Indiana
      • Indianapolis, Indiana, United States, 46202
        • Recruiting
        • Indiana University School of Medicine
        • Contact:
          • Jean Molleston, MD
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Recruiting
        • Johns Hopkins Children's Center
        • Contact:
          • Wikrom Karnsakul, MD
    • Massachusetts
      • Boston, Massachusetts, United States, 02115
        • Recruiting
        • Boston Children's Hospital
        • Contact:
          • Andrew Wehrman, MD
    • Michigan
      • Ann Arbor, Michigan, United States, 48109
        • Recruiting
        • University of Michigan Children's Hospital
        • Contact:
          • Maclovio Lopez, MD
    • Missouri
      • Kansas City, Missouri, United States, 64108
        • Recruiting
        • Children's Mercy Hospital
        • Contact:
          • Ryan Fischer, MD
      • Saint Louis, Missouri, United States, 63110
        • Recruiting
        • Washington University in St. Louis
        • Contact:
          • Janis M. Stoll, MD
    • New York
      • Bronx, New York, United States, 10467
        • Recruiting
        • The Children's Hospital at Montefiore
        • Contact:
          • Pan Debra, MD
        • Principal Investigator:
          • Pan Debra, MD
      • New York, New York, United States, 10029
        • Recruiting
        • Icahn School of Medicine at Mount Sinai
        • Contact:
          • Jaime Chu, MD
      • New York, New York, United States, 10016
        • Recruiting
        • NYU Grossman School of Medicine
        • Contact:
          • Nadia Ovchinsky, MD
        • Principal Investigator:
          • Nadia Ovchinsky
      • New York, New York, United States, 10032
        • Recruiting
        • NewYork-Presbyterian Morgan Stanley Children's Hospital
        • Contact:
          • Dana Goldner, MD
        • Principal Investigator:
          • Dana Goldner, MD
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Recruiting
        • Cincinnati Children's Hospital
        • Contact:
          • Michael Rogers, MD
        • Principal Investigator:
          • Michael Rogers, MD
      • Cleveland, Ohio, United States, 44195
        • Recruiting
        • Cleveland Clinic
        • Contact:
          • Vera Hupertz, MD
    • Oregon
      • Portland, Oregon, United States, 97239
        • Recruiting
        • Oregon Health & Science University
        • Contact:
          • Henry Lin, MD
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • The Children´s Hospital of Philadelphia
        • Contact:
          • Kathleen Loomes, MD
      • Pittsburgh, Pennsylvania, United States, 15224
        • Recruiting
        • UPMC Children's Hospital of Pittsburgh
        • Contact:
          • James Squires, MD
    • Texas
      • Dallas, Texas, United States, 75390
        • Recruiting
        • UT Southwestern Medical Center
        • Contact:
          • Amal Aqul, MD
    • Utah
      • Salt Lake City, Utah, United States, 84113
        • Recruiting
        • University of Utah
        • Contact:
          • Kyle Jensen, MD
    • Washington
      • Seattle, Washington, United States, 98105
        • Recruiting
        • Seattle Children's Hospital
        • Contact:
          • Evelyn Hsu, MD
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53226
        • Terminated
        • Medical College of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

No older than 1 year (Child)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Triple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Odevixibat (A4250)
Capsules for oral administration once daily for 104 weeks.
Drug: Odevixibat
Odevixibat is a small molecule and selective inhibitor of IBAT.
Placebo Comparator: Placebo
Capsules for oral administration (to match active) once daily for 104 weeks.
Drug: Placebo
Placebo identical in appearance to experimental drug (odevixibat).

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Time to onset of any sentinel events
Time Frame: From baseline to Week 104
Time to onset of any sentinel events
From baseline to Week 104
Time to pediatric end-stage liver disease (PELD) score >15
Time Frame: From baseline to Week 104
Time to pediatric end-stage liver disease (PELD) score >15
From baseline to Week 104
Total bilirubin levels
Time Frame: From baseline to Weeks 13, 26, 52 and 104
Total bilirubin level after 13, 26, 52, and 104 weeks of study treatment
From baseline to Weeks 13, 26, 52 and 104
Serum bile acid levels
Time Frame: From baseline to Weeks 13, 26, 52 and 104
Serum bile acid level after 13, 26, 52, and 104 weeks of study treatment
From baseline to Weeks 13, 26, 52 and 104
Percentage of participants experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame: From baseline to Week 104
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
Percentage of participants with clinically significant changes in Physical Examination
Time Frame: From baseline to Week 104
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
Percentage of participants with clinically significant changes in Laboratory Parameters (blood chemistry, hematology and coagulation)
Time Frame: From baseline to Week 104
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
Percentage of participants with clinically significant changes in Abdominal Ultrasound findings
Time Frame: From baseline to Week 26 and Week 104
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
Proportion of patients with liver transplant
Time Frame: From baseline to Week 104
Proportion of patients who are alive and have not undergone a liver transplant
From baseline to Week 104

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Albireo

Investigators

  • Study Director: Ipsen Medical Director, Ipsen

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

July 8, 2020

Primary Completion (Estimated)

June 30, 2026

Study Completion (Estimated)

July 31, 2026

Study Registration Dates

First Submitted

March 27, 2020

First Submitted That Met QC Criteria

April 3, 2020

First Posted (Actual)

April 7, 2020

Study Record Updates

Last Update Posted (Actual)

March 28, 2024

Last Update Submitted That Met QC Criteria

March 27, 2024

Last Verified

March 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • A4250-011

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD Plan 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.

Any requests should be submitted to www.vivli.org for assessment by an independent scientific review board.

IPD Sharing 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 EU or after the primary manuscript describing the results has been accepted for publication, whichever is later.

IPD Sharing Access Criteria

Further details on Ipsen's sharing criteria, eligible studies and process for sharing are available here (https://vivli.org/members/ourmembers/).

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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