What side effects are associated with this type of intervention?

Common treatments for Biliary Atresia include surgical intervention (Kasai procedure) and medications such as ursodeoxycholic acid (UDCA). Side effects of UDCA can include diarrhea, nausea, and abdominal pain. For IBAT inhibitors like Odevixibat, potential side effects may include gastrointestinal issues such as diarrhea, abdominal pain, and possible liver enzyme elevations. It is important to monitor these side effects closely under medical supervision.

What prior FDA approvals exist?

Biliary Atresia is a serious liver condition in infants that often requires surgical intervention, such as the Kasai hepatoportoenterostomy, to restore bile flow. While the provided context does not mention specific FDA-approved drugs for Biliary Atresia, it does highlight a Phase 3 study investigating the efficacy and safety of Odevixibat, an IBAT inhibitor, in children who have undergone the Kasai procedure. This suggests ongoing research into pharmacological treatments to manage the condition post-surgery. Historically, treatment has focused on surgical approaches and supportive care, with limited pharmacological options specifically approved for Biliary Atresia.

What other types of research exist?

Promising novel alternatives to Odevixibat for Biliary Atresia patients include gene therapy approaches targeting the underlying genetic causes of the disease, new pharmacological agents such as other bile acid modulators, and advancements in surgical techniques like improved methods for liver transplantation or Kasai procedure enhancements. Additionally, research into immunomodulatory therapies and regenerative medicine may offer new avenues for treatment.

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Ion Channel Modulator

Odevixibat for Biliary Atresia

Phase 3

Waitlist Available

Research Sponsored by Albireo, an Ipsen Company

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Male or female patient with a clinical diagnosis of BA

Age at Kasai HPE ≤90 days

Must not have

Patients with intractable ascites

History of ileal resection surgery

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from baseline to weeks 13, 26, 52 and 104

Awards & highlights

Pivotal Trial

Summary

This trial is testing odevixibat, a medication that may help children with biliary atresia who have had surgery. The drug aims to lower bile acid levels to protect the liver.

Who is the study for?

This trial is for children with Biliary Atresia who've had a Kasai procedure before they were 90 days old. They should be able to start treatment within 3 weeks post-surgery and weigh more than 3.5kg. Children with severe liver issues, relying solely on IV nutrition, or having certain other conditions are not eligible.

What is being tested?

The study tests the effectiveness and safety of Odevixibat versus a placebo in kids after Kasai surgery for Biliary Atresia. It's a Phase 3 trial where participants are randomly assigned to either the drug or placebo without knowing which one they receive.

What are the potential side effects?

While specific side effects aren't listed here, Odevixibat could potentially cause digestive issues, reactions at the site of administration, changes in liver enzymes, and possibly affect growth due to its role in bile acid processing.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with biliary atresia.

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I had the Kasai procedure done before I was 3 months old.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have severe, untreatable fluid buildup in my abdomen.

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I have had surgery to remove part of my ileum.

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I have had a condition involving cysts in my bile ducts.

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I rely on IV nutrition or cannot take pills by mouth.

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My weight is less than 3.5kg at the time of joining the study.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from baseline to weeks 13, 26, 52 and 104

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from baseline to weeks 13, 26, 52 and 104

This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline to weeks 13, 26, 52 and 104 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Secondary study objectives

Percentage of participants experiencing Adverse Events (AEs) and Serious Adverse Events (SAEs)

Percentage of participants with clinically significant changes in Abdominal Ultrasound findings

Percentage of participants with clinically significant changes in Laboratory Parameters (blood chemistry, hematology and coagulation)

+6 more

Side effects data

From 2022 Phase 3 trial • 52 Patients • NCT04674761

29%

Diarrhoea

23%

Pyrexia

14%

COVID-19

11%

Abdominal pain

Upper respiratory tract infection

Cough

Respiratory tract infection

Bronchitis

Haematoma

Vomiting

Asthenia

Gastroenteritis

Nasopharyngitis

Conjunctivitis

Weight decreased

Pain in extremity

Pharyngitis

Aphthous ulcer

Viral infection

Otitis media

Jaundice

Faeces discoloured

International normalised ratio increased

Lymphadenopathy

Cataract cortical

Nausea

Hypersensitivity

Tonsillitis

Pneumonia

Anaemia macrocytic

Constipation

Coagulopathy

Frequent bowel movements

Haematemesis

Rhinovirus infection

Abdominal pain upper

Faeces soft

Contusion

Ligament sprain

Headache

Asthma

Alanine aminotransferase increased

Blood triglycerides increased

Gamma-glutamyltransferase increased

Hepatic enzyme increased

Platelet count decreased

Vitamin A decreased

Vitamin E decreased

Hypophagia

Vitamin D deficiency

Oropharyngeal pain

Pharyngeal inflammation

Rhinitis allergic

Rhinorrhoea

Skin lesion

Urticaria

100%

80%

60%

40%

20%

Study treatment Arm

Odevixibat (A4250)

Placebo

Awards & Highlights

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Odevixibat (A4250)Experimental Treatment1 Intervention

Capsules for oral administration once daily for 104 weeks.

Group II: PlaceboPlacebo Group1 Intervention

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

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Odevixibat

2021

Completed Phase 3

~60

0 / 7Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Odevixibat, an ileal bile acid transporter (IBAT) inhibitor, works by reducing the reabsorption of bile acids in the ileum, thereby decreasing the bile acid pool in the liver. This reduction in bile acids can alleviate cholestasis and improve bile flow, which is crucial for patients with Biliary Atresia who suffer from impaired bile drainage. By improving bile flow, Odevixibat can potentially reduce liver damage and improve overall liver function, offering a significant therapeutic benefit for managing this condition.

The Effects of Clofibrate on Neonatal Jaundice: A Systematic Review.