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Trial record 1 of 1 for:    NCT04336722
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Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)

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ClinicalTrials.gov Identifier: NCT04336722
Recruitment Status : Recruiting
First Posted : April 7, 2020
Last Update Posted : September 17, 2021
Sponsor:
Information provided by (Responsible Party):
Albireo

Tracking Information
First Submitted Date  ICMJE March 27, 2020
First Posted Date  ICMJE April 7, 2020
Last Update Posted Date September 17, 2021
Actual Study Start Date  ICMJE July 10, 2020
Estimated Primary Completion Date May 2024   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: April 3, 2020)
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.
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: December 15, 2020)
  • Time to onset of any sentinel events [ Time Frame: From baseline to Week 104 ]
    Time to onset of any sentinel events
  • 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
  • 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
  • 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
Original Secondary Outcome Measures  ICMJE
 (submitted: April 3, 2020)
  • Time to onset of any sentinel events [ Time Frame: From baseline to Week 104 ]
    Time to onset of any sentinel events
  • 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
  • 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
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)
Official Title  ICMJE 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
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.
Detailed Description Up to 70 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple (Participant, Care Provider, Investigator)
Primary Purpose: Treatment
Condition  ICMJE Biliary Atresia
Intervention  ICMJE
  • Drug: Odevixibat
    Odevixibat is a small molecule and selective inhibitor of IBAT.
  • Drug: Placebo
    Placebo identical in appearance to experimental drug (odevixibat).
Study Arms  ICMJE
  • Experimental: Odevixibat (A4250)
    Capsules for oral administration once daily for 104 weeks.
    Intervention: Drug: Odevixibat
  • Placebo Comparator: Placebo
    Capsules for oral administration (to match active) once daily for 104 weeks.
    Intervention: Drug: Placebo
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: April 3, 2020)
200
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 2024
Estimated Primary Completion Date May 2024   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 111 Days   (Child)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Albireo +1 (857) 378-2035 medinfo@albireopharma.com
Listed Location Countries  ICMJE Australia,   Belgium,   Canada,   France,   Germany,   Hungary,   Israel,   Italy,   Korea, Republic of,   Malaysia,   Netherlands,   New Zealand,   Poland,   Spain,   Taiwan,   Turkey,   United Kingdom,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04336722
Other Study ID Numbers  ICMJE A4250-011
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Albireo
Study Sponsor  ICMJE Albireo
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Albireo
Verification Date April 2021

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP