Clinical trial NCT04336722: Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)
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
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Biliary Atresia
Keywords: Kasai
Lead sponsor: Ipsen
Sponsor class: INDUSTRY
First posted: 2020-04-07
Enrollment: 254
Sex: ALL
Maximum age (years): 111
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility 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

Primary outcomes:
measure: Time from randomization to first occurrence of liver transplant, or death
timeFrame: From baseline to Week 104
Secondary outcomes:
measure: Proportion of patients with liver transplant
timeFrame: From baseline to Week 104
measure: Time to onset of any sentinel events
timeFrame: From baseline to Week 104
measure: Total bilirubin levels
timeFrame: From baseline to Weeks 13, 26, 52 and 104
measure: Serum bile acid levels
timeFrame: From baseline to Weeks 13, 26, 52 and 104
measure: Time to pediatric end-stage liver disease (PELD) score >15
timeFrame: From baseline to Week 104
measure: Percentage of participants experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)
timeFrame: From baseline to Week 104
measure: Percentage of participants with clinically significant changes in Physical Examination
timeFrame: From baseline to Week 104
measure: Percentage of participants with clinically significant changes in Laboratory Parameters (blood chemistry, hematology and coagulation)
timeFrame: From baseline to Week 104
measure: Percentage of participants with clinically significant changes in Abdominal Ultrasound findings
timeFrame: From baseline to Week 26 and Week 104
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Children's Hospital Los Angeles
state: California
city: Palo Alto
country: United States
facility: Stanford Children's Health
state: California
city: San Diego
country: United States
facility: Rady Children's Hospital
state: California
city: San Francisco
country: United States
facility: UCSF Benioff Children's Hospital San Francisco
state: California
city: Aurora
country: United States
facility: Children's Hospital Colorado
state: Colorado
city: Wilmington
country: United States
facility: Nemours/Alfred I. duPont Hospital for Children
state: Delaware
city: Washington
country: United States
facility: Children's National Medical Center
state: District of Columbia
city: Miami
country: United States
facility: University of Miami
state: Florida
city: Atlanta
country: United States
facility: Children's Healthcare of Atlanta - Emory University School of Medicine
state: Georgia
city: Chicago
country: United States
facility: Ann & Robert H. Lurie Children's Hospital
state: Illinois
city: Indianapolis
country: United States
facility: Indiana University school of Medicine
state: Indiana
city: Baltimore
country: United States
facility: Johns Hopkins Children's Center
state: Maryland
city: Boston
country: United States
facility: Boston Children's Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan Children's Hospital
state: Michigan
city: Kansas City
country: United States
facility: Children's Mercy Hospital
state: Missouri
city: Saint Louis
country: United States
facility: Washington University in St. Louis
state: Missouri
city: Bronx
country: United States
facility: The Children's Hospital at Montefiore
state: New York
city: New York
country: United States
facility: NYU Grossman school of Medicine
state: New York
city: New York
country: United States
facility: Icahn School of Medicine at Mount Sinai
state: New York
city: New York
country: United States
facility: NewYork-Presbyterian Morgan Stanley Children's Hospital
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Children's Hospital
state: Ohio
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Portland
country: United States
facility: Oregon Health & Science University
state: Oregon
city: Philadelphia
country: United States
facility: The Children´s Hospital of Philadelphia
state: Pennsylvania
city: Pittsburgh
country: United States
facility: UPMC Children's Hospital of Pittsburgh
state: Pennsylvania
city: Dallas
country: United States
facility: UT Southwestern Medical Center
state: Texas
city: Salt Lake City
country: United States
facility: University of Utah
state: Utah
city: Seattle
country: United States
facility: Seattle Children's Hospital
state: Washington
city: Milwaukee
country: United States
facility: Medical College of Wisconsin
state: Wisconsin
city: Parkville
country: Australia
facility: Royal Children's Hospital
city: Sydney
country: Australia
facility: The Children´s Hospital at Westmead
city: Gent
country: Belgium
facility: UZ Gent
city: Montréal
country: Canada
facility: CHU Sainte-Justine
city: Toronto
country: Canada
facility: The Hospital for Sick Children
city: Guangzhou
country: China
facility: Guangzhou Women and Children's Medical Center
state: Guangdong
city: Shanghai
country: China
facility: Children's Hospital of Fudan University
city: Bron
country: France
facility: Hôpital Femme Mère Enfant
city: Le Kremlin-Bicêtre
country: France
facility: Bicêtre Hospital
city: Lille
country: France
facility: Jeanne de Flandre Hospital
city: Paris
country: France
facility: Necker University Hospital - Enfants malades
city: Berlin
country: Germany
facility: Charité - Universitätsmedizin Berlin
city: Hamburg
country: Germany
facility: University Medical Center Hamburg-Eppendorf UKE
city: Hanover
country: Germany
facility: Hannover Medical School
city: Tuebingen
country: Germany
facility: University Children´s Hospital Tuebingen
city: Budapest
country: Hungary
facility: Semmelweis Egyetem I.sz Gyermekgyógyászati Klinika
city: Petah tikva
country: Israel
facility: Schneider Children´s Medical Center of Israel
city: Bergamo
country: Italy
facility: ASST Papa Giovanni XXIII
city: Florence
country: Italy
facility: Meyer Children´s University Hospital
city: Padova
country: Italy
facility: University Hospital of Padova
city: Palermo
country: Italy
facility: ISMETT - Istituto Mediterraneo per i Trapianti e Terapie ad Alta Specializzazione
city: Roma
country: Italy
facility: Ospedale Pediatrico Bambino Gesù
city: Turin
country: Italy
facility: Regina Margherita Children´s Hospital
city: Seoul
country: Korea, Republic of
facility: Samsung Medical Center
city: Seoul
country: Korea, Republic of
facility: Seoul National University Children's Hospital
city: Kota Bharu
country: Malaysia
facility: Hospital Raja Perempuan Zainab II
city: Kuala Lumpur
country: Malaysia
facility: University of Malaya Medical Centre
city: Groningen
country: Netherlands
facility: University Medical Center Groningen
city: Auckland
country: New Zealand
facility: Starship Child Health
city: Warsaw
country: Poland
facility: Instytut Pomnik-Centrum Zdrowia Dziecka
city: Barcelona
country: Spain
facility: Hospital Universitari Vall d'Hebron
city: Taipei
country: Taiwan
facility: National Taiwan University Hospital
city: Ankara
country: Turkey
facility: Hacettepe University Ihsan Dogramaci Childrens Hospital
city: Antalya
country: Turkey
facility: Akdeniz University Medical Faculty
city: Istanbul
country: Turkey
facility: Istanbul University, Istanbul Medical Faculty
city: Birmingham
country: United Kingdom
facility: Birmingham Women´s and Children´s Hospital
city: Leeds
country: United Kingdom
facility: Leeds General Infirmary
Study officials:
affiliation: Ipsen
name: Ipsen Medical Director
role: STUDY_DIRECTOR
References: