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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04336722
Recruitment Status : Recruiting
First Posted : April 7, 2020
Last Update Posted : September 14, 2020
Sponsor:
Information provided by (Responsible Party):
Albireo

Brief 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.

Condition or disease Intervention/treatment Phase
Biliary Atresia Drug: Odevixibat Drug: Placebo Phase 3

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 200 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
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
Actual Study Start Date : July 10, 2020
Estimated Primary Completion Date : May 2024
Estimated Study Completion Date : June 2024

Resource links provided by the National Library of Medicine


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).




Primary Outcome Measures :
  1. 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 after 104 weeks of study treatment.


Secondary Outcome Measures :
  1. Time to onset of any sentinel events [ Time Frame: From baseline to Week 104 ]
    Time to onset of any sentinel events

  2. 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

  3. 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



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


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Ages Eligible for Study:   up to 111 Days   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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
  • Patient on total parenteral nutrition at randomization
  • Acute ascending cholangitis (patients may be randomized after resolution of acute ascending cholangitis)
  • Choledochal cystic disease

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04336722


Contacts
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Contact: Albireo +1 (857) 378-2035 medinfo@albireopharma.com

Locations
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United States, California
UCSF Benioff Children's Hospital San Francisco Recruiting
San Francisco, California, United States, 94158
Contact: Phillip Rosenthal, MD         
United States, Florida
University of Miami Recruiting
Miami, Florida, United States, 33146
Contact: Tamir Miloh, MD         
United States, Georgia
Children's Healthcare of Atlanta - Emory University School of Medicine Recruiting
Atlanta, Georgia, United States, 30322
Contact: Saul Karpen, MD, PhD         
United States, Indiana
Indiana University school of Medicine Recruiting
Indianapolis, Indiana, United States, 46202
Contact: Jean Molleston, MD         
United States, Maryland
Johns Hopkins Children's Center Recruiting
Baltimore, Maryland, United States, 21287
Contact: Wikrom Karnsakul, MD         
United States, New York
The Children's Hospital at Montefiore Recruiting
Bronx, New York, United States, 10467
Contact: Nadia Ovchinsky, MD         
Icahn School of Medicine at Mount Sinai Recruiting
New York, New York, United States, 10029
Contact: Jaime Chu, MD         
United States, Pennsylvania
UPMC Children's Hospital of Pittsburgh Recruiting
Pittsburgh, Pennsylvania, United States, 15224
Contact: Patrick McKiernan, MD         
Sponsors and Collaborators
Albireo
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Responsible Party: Albireo
ClinicalTrials.gov Identifier: NCT04336722    
Other Study ID Numbers: A4250-011
First Posted: April 7, 2020    Key Record Dates
Last Update Posted: September 14, 2020
Last Verified: September 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Albireo:
Kasai
Additional relevant MeSH terms:
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Biliary Atresia
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Digestive System Abnormalities
Congenital Abnormalities