Efficacy and Safety of Odevixibat in Children With Biliary Atresia Who Have Undergone a Kasai HPE (BOLD)

Albireo, an Ipsen Company

Status and phase: Active, not recruiting; Phase 3

Conditions: Biliary Atresia

Treatments: Drug: Odevixibat; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT04336722

A4250-011

Details and patient eligibility

About

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.

Full description

Up to 70 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.

Enrollment: 254 patients

Sex: All

Ages: Under 111 days old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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254 participants in 2 patient groups, including a placebo group

Odevixibat (A4250)

Experimental group

Description:

Capsules for oral administration once daily for 104 weeks.

Treatment:

Drug: Odevixibat

Placebo

Placebo Comparator group

Description:

Capsules for oral administration (to match active) once daily for 104 weeks.

Treatment:

Drug: Placebo