Biliary Atresia
Conditions
Keywords
Kasai
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.
Interventions
DRUGOdevixibat
Odevixibat is a small molecule and selective inhibitor of IBAT.
DRUGPlacebo
Placebo identical in appearance to experimental drug (odevixibat).
Sponsors
Albireo, an Ipsen Company
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
TRIPLE (Subject, Caregiver, Investigator)
Eligibility
Sex/Gender
ALL
Age
No minimum to 111 Days
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Time from randomization to first occurrence of liver transplant, or death | From baseline to Week 104 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Proportion of patients with liver transplant | From baseline to Week 104 | Proportion of patients who are alive and have not undergone a liver transplant |
| Time to onset of any sentinel events | From baseline to Week 104 | Time to onset of any sentinel events |
| 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 |
| 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. |
Countries
Australia, Belgium, Canada, China, France, Germany, Hungary, Israel, Italy, Malaysia, Netherlands, New Zealand, Poland, South Korea, Spain, Taiwan, Turkey (Türkiye), United Kingdom, United States
Contacts
STUDY_DIRECTORIpsen Medical Director
Ipsen
Outcome results
None listed