Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)
- Registration Number
- NCT04336722
- Lead Sponsor
- Albireo, an Ipsen Company
- 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.
Recruitment & Eligibility
- Status
- ACTIVE_NOT_RECRUITING
- Sex
- All
- Target Recruitment
- 254
- 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
- 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 & Design
- Study Type
- INTERVENTIONAL
- Study Design
- PARALLEL
- Arm && Interventions
Group Intervention Description Odevixibat (A4250) Odevixibat Capsules for oral administration once daily for 104 weeks. Placebo Placebo Capsules for oral administration (to match active) once daily for 104 weeks.
- Primary Outcome Measures
Name Time Method Time from randomization to first occurrence of liver transplant, or death From baseline to Week 104
- Secondary Outcome Measures
Name Time Method Time to onset of any sentinel events From baseline to Week 104 Time to onset of any sentinel events
Serum bile acid levels From baseline to Weeks 13, 26, 52 and 104 Serum bile acid level after 13, 26, 52, and 104 weeks of study treatment
Time to pediatric end-stage liver disease (PELD) score >15 From baseline to Week 104 Time to pediatric end-stage liver disease (PELD) score \>15
Percentage of participants experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs) 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.
Percentage of participants with clinically significant changes in Physical Examination 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.
Percentage of participants with clinically significant changes in Laboratory Parameters (blood chemistry, hematology and coagulation) 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.
Percentage of participants with clinically significant changes in Abdominal Ultrasound findings 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.
Proportion of patients with liver transplant From baseline to Week 104 Proportion of patients who are alive and have not undergone a liver transplant
Total bilirubin levels From baseline to Weeks 13, 26, 52 and 104 Total bilirubin level after 13, 26, 52, and 104 weeks of study treatment
Trial Locations
- Locations (66)
Children's Healthcare of Atlanta - Emory University School of Medicine
🇺🇸Atlanta, Georgia, United States
Johns Hopkins Children's Center
🇺🇸Baltimore, Maryland, United States
Seattle Children's Hospital
🇺🇸Seattle, Washington, United States
UZ Gent
🇧🇪Gent, Belgium
UCSF Benioff Children's Hospital San Francisco
🇺🇸San Francisco, California, United States
Indiana University school of Medicine
🇺🇸Indianapolis, Indiana, United States
Cincinnati Children's Hospital
🇺🇸Cincinnati, Ohio, United States
University of Utah
🇺🇸Salt Lake City, Utah, United States
The Hospital for Sick Children
🇨🇦Toronto, Canada
Meyer Children´s University Hospital
🇮🇹Florence, Italy
Bicêtre Hospital
🇫🇷Le Kremlin-Bicêtre, France
Schneider Children´s Medical Center of Israel
🇮🇱Petah tikva, Israel
The Children's Hospital at Montefiore
🇺🇸Bronx, New York, United States
Children's Mercy Hospital
🇺🇸Kansas City, Missouri, United States
Hôpital Femme Mère Enfant
🇫🇷Bron, France
Cleveland Clinic
🇺🇸Cleveland, Ohio, United States
University of Michigan Children's Hospital
🇺🇸Ann Arbor, Michigan, United States
CHU Sainte-Justine
🇨🇦Montréal, Canada
University Hospital of Padova
🇮🇹Padova, Italy
NYU Grossman school of Medicine
🇺🇸New York, New York, United States
Nemours/Alfred I. duPont Hospital for Children
🇺🇸Wilmington, Delaware, United States
Guangzhou Women and Children's Medical Center
🇨🇳Guangzhou, Guangdong, China
Ospedale Pediatrico Bambino Gesù
🇮🇹Roma, Italy
UT Southwestern Medical Center
🇺🇸Dallas, Texas, United States
University of Malaya Medical Centre
🇲🇾Kuala Lumpur, Malaysia
University Medical Center Groningen
🇳🇱Groningen, Netherlands
Akdeniz University Medical Faculty
🇹🇷Antalya, Turkey
Hannover Medical School
🇩🇪Hanover, Germany
Charité - Universitätsmedizin Berlin
🇩🇪Berlin, Germany
University Children´s Hospital Tuebingen
🇩🇪Tuebingen, Germany
Hospital Universitari Vall d'Hebron
🇪🇸Barcelona, Spain
Rady Children's Hospital
🇺🇸San Diego, California, United States
Children's National Medical Center
🇺🇸Washington, District of Columbia, United States
Ann & Robert H. Lurie Children's Hospital
🇺🇸Chicago, Illinois, United States
Boston Children's Hospital
🇺🇸Boston, Massachusetts, United States
NewYork-Presbyterian Morgan Stanley Children's Hospital
🇺🇸New York, New York, United States
UPMC Children's Hospital of Pittsburgh
🇺🇸Pittsburgh, Pennsylvania, United States
The Children´s Hospital of Philadelphia
🇺🇸Philadelphia, Pennsylvania, United States
Oregon Health & Science University
🇺🇸Portland, Oregon, United States
The Children´s Hospital at Westmead
🇦🇺Sydney, Australia
Royal Children's Hospital
🇦🇺Parkville, Australia
Children's Hospital of Fudan University
🇨🇳Shanghai, China
Jeanne de Flandre Hospital
🇫🇷Lille, France
Necker University Hospital - Enfants malades
🇫🇷Paris, France
University Medical Center Hamburg-Eppendorf UKE
🇩🇪Hamburg, Germany
Leeds General Infirmary
🇬🇧Leeds, United Kingdom
Semmelweis Egyetem I.sz Gyermekgyógyászati Klinika
🇭🇺Budapest, Hungary
ASST Papa Giovanni XXIII
🇮🇹Bergamo, Italy
ISMETT - Istituto Mediterraneo per i Trapianti e Terapie ad Alta Specializzazione
🇮🇹Palermo, Italy
Regina Margherita Children´s Hospital
🇮🇹Turin, Italy
Samsung Medical Center
🇰🇷Seoul, Korea, Republic of
Hospital Raja Perempuan Zainab II
🇲🇾Kota Bharu, Malaysia
Seoul National University Children's Hospital
🇰🇷Seoul, Korea, Republic of
National Taiwan University Hospital
🇨🇳Taipei, Taiwan
Instytut Pomnik-Centrum Zdrowia Dziecka
🇵🇱Warsaw, Poland
Birmingham Women´s and Children´s Hospital
🇬🇧Birmingham, United Kingdom
Istanbul University, Istanbul Medical Faculty
🇹🇷Istanbul, Turkey
Hacettepe University Ihsan Dogramaci Childrens Hospital
🇹🇷Ankara, Turkey
Children's Hospital Colorado
🇺🇸Aurora, Colorado, United States
Washington University in St. Louis
🇺🇸Saint Louis, Missouri, United States
Starship Child Health
🇳🇿Auckland, New Zealand
Medical College of Wisconsin
🇺🇸Milwaukee, Wisconsin, United States
Stanford Children's Health
🇺🇸Palo Alto, California, United States
Children's Hospital Los Angeles
🇺🇸Los Angeles, California, United States
University of Miami
🇺🇸Miami, Florida, United States
Icahn School of Medicine at Mount Sinai
🇺🇸New York, New York, United States