Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)
Purpose
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
- Biliary Atresia
Eligibility
- Eligible Ages
- Under 111 Days
- Eligible Genders
- All
- Accepts 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
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
Study Design
- Phase
- Phase 3
- Study Type
- Interventional
- Allocation
- Randomized
- Intervention Model
- Parallel Assignment
- Primary Purpose
- Treatment
- Masking
- Triple (Participant, Care Provider, Investigator)
Arm Groups
Arm | Description | Assigned Intervention |
---|---|---|
Experimental Odevixibat (A4250) |
Capsules for oral administration once daily for 104 weeks. |
|
Placebo Comparator Placebo |
Capsules for oral administration (to match active) once daily for 104 weeks. |
|
Recruiting Locations
The Children's Hospital at Montefiore
Bronx, New York 10467
Bronx, New York 10467
Contact:
Pan Debra, MD
Pan Debra, MD
More Details
- Status
- Recruiting
- Sponsor
- Albireo
Detailed Description
Up to 70 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.