Working…
ClinicalTrials.gov
ClinicalTrials.gov Menu
Trial record 1 of 1 for:    NCT04336722
Previous Study | Return to List | Next Study

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 : June 24, 2021
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.

Layout table for study information
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. 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

  3. 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

  4. 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.


Layout table for eligibility information
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
  • 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

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
Layout table for location contacts
Contact: Albireo +1 (857) 378-2035 medinfo@albireopharma.com

Locations
Show Show 53 study locations
Sponsors and Collaborators
Albireo
Layout table for additonal information
Responsible Party: Albireo
ClinicalTrials.gov Identifier: NCT04336722    
Other Study ID Numbers: A4250-011
First Posted: April 7, 2020    Key Record Dates
Last Update Posted: June 24, 2021
Last Verified: April 2021

Layout table for additional information
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:
Layout table for MeSH terms
Biliary Atresia
Bile Duct Diseases
Biliary Tract Diseases
Digestive System Diseases
Digestive System Abnormalities
Congenital Abnormalities