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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

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

ArmDescriptionAssigned Intervention
Experimental
Odevixibat (A4250) 		Capsules for oral administration once daily for 104 weeks.
  • Drug: Odevixibat
    Odevixibat is a small molecule and selective inhibitor of IBAT.
Placebo Comparator
Placebo 		Capsules for oral administration (to match active) once daily for 104 weeks.
  • Drug: Placebo
    Placebo identical in appearance to experimental drug (odevixibat).

Recruiting Locations

The Children's Hospital at Montefiore
Bronx, New York 10467
Contact:
Pan Debra, MD

More Details

Status
Recruiting
Sponsor
Albireo

Study Contact

Ipsen Clinical Study Enquiries
See email
clinical.trials@ipsen.com

Detailed Description

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

Notice

Study information shown on this site is derived from ClinicalTrials.gov (a public registry operated by the National Institutes of Health). The listing of studies provided is not certain to be all studies for which you might be eligible. Furthermore, study eligibility requirements can be difficult to understand and may change over time, so it is wise to speak with your medical care provider and individual research study teams when making decisions related to participation.