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Bile Acid Sequestrant

Odevixibat for PFIC

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
Cohort 2: Patients with PFIC, excluding BRIC, must have elevated serum bile acid concentration, specifically measured to be ≥100 μmol/L, taken as the average of 2 samples at least 7 days apart (Visits S-1 and S-2) prior to the Screening/Inclusion Visit (Visit 1)
Cohort 2: Patient must have clinical genetic confirmation of PFIC
Must not have
Cohort 2: Patient has a confirmed past diagnosis of infection with human immunodeficiency virus or other present and active, clinically significant, acute, or chronic infection, or past medical history of any major episode of infection requiring hospitalization or treatment with parenteral anti-infective treatment within 4 weeks of treatment start (Study Day 1) or completion of oral anti-infective treatment within 2 weeks prior to start of Screening Period
Cohort 1: Decompensated liver disease: coagulopathy, history, or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy
Timeline
Screening 3 weeks
Treatment Varies
Follow Up from baseline to weeks 24, 48, and 72
Awards & highlights
Pivotal Trial
No Placebo-Only Group

Summary

This trial is testing A4250, a medication, to see if it is safe and effective for children with a rare liver disease called PFIC. The goal is to improve liver function and reduce symptoms over time.

Who is the study for?
This trial is for children with PFIC, a liver disorder causing severe itching and jaundice. Participants need genetic confirmation of PFIC, have had significant pruritus, weigh at least 5 kg, and can use an eDiary. They must have completed or partially completed a prior A4250 study. Those with episodic PFIC should be experiencing a flare-up.
What is being tested?
The trial tests the long-term safety and effectiveness of A4250 (odevixibat) in kids with PFIC. It's an open-label extension study meaning all participants receive the drug and are monitored over time to see how well it works and what side effects occur.
What are the potential side effects?
While specific side effects for A4250 aren't listed here, common issues may include gastrointestinal symptoms like diarrhea or abdominal pain due to its effect on bile acids.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below
Select...
I have PFIC with bile acid levels ≥100 μmol/L, confirmed by 2 tests.
Select...
I have a genetic confirmation of PFIC.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:
Select...
I have had a significant infection or HIV in the past, but it's currently under control.
Select...
I have severe liver disease with symptoms like bleeding, fluid buildup, or confusion.
Select...
I have a genetic variation in the ABCB11 gene leading to no BSEP protein.
Select...
I have had chronic diarrhea for over 3 months needing IV fluids or nutritional support.
Select...
I have severe liver issues, including bleeding, fluid buildup, or mental confusion.

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from baseline to weeks 24, 48, and 72
This trial's timeline: 3 weeks for screening, Varies for treatment, and from baseline to weeks 24, 48, and 72 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
US: Change in Pruritus
Secondary study objectives
All regions: Change in growth

Side effects data

From 2020 Phase 3 trial • 62 Patients • NCT03566238
26%
Pyrexia
26%
Upper respiratory tract infection
21%
Diarrhoea
16%
Alanine aminotransferase increased
16%
Vomiting
11%
Nasopharyngitis
11%
Otitis media
11%
Vitamin D deficiency
11%
Cough
11%
Splenomegaly
11%
Blood bilirubin increased
11%
Blood alkaline phosphatase increased
5%
Otorrhoea
5%
Liver function test increased
5%
Cardiac ablation
5%
Respiratory tract infection
5%
Vitamin D decreased
5%
Cholelithiasis
5%
Urinary tract infection
5%
Supraventricular tachycardia
5%
Abdominal pain
5%
Mouth ulceration
5%
Viral infection
5%
Vitamin A deficiency
5%
Vitamin E deficiency
5%
Cystitis haemorrhagic
5%
Rhinitis allergic
5%
Dehydration
5%
Ear pain
5%
Eye discharge
5%
Abdominal discomfort
5%
Gastroenteritis norovirus
5%
Influenza
5%
Aspartate aminotransferase increased
5%
Platelet count increased
5%
Blood creatine phosphokinase increased
5%
Irritability
5%
Genital rash
5%
Epistaxis
5%
Dermatitis allergic
5%
Pruritus
5%
Sinusitis bacterial
5%
Abdominal pain upper
5%
Dental caries
5%
Hepatomegaly
5%
Parotitis
5%
Headache
5%
Dizziness
100%
80%
60%
40%
20%
0%
Study treatment Arm
A4250 High Dose
A4250 Low Dose
Placebo

Awards & Highlights

Pivotal Trial
The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.
No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: A4250Experimental Treatment1 Intervention
Capsules for oral administration (40 or 120 µg/kg) once daily for 72 weeks, or 40 µg/kg/day for the first 12 weeks followed by 120 µg/kg/day for the remaining 60 weeks"
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
A4250 (odevixibat)
2018
Completed Phase 3
~70

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
The most common treatments for Progressive Familial Intrahepatic Cholestasis (PFIC) include selective inhibitors of the ileal bile acid transporter (IBAT) such as A4250. These inhibitors work by reducing the reabsorption of bile acids in the ileum, thereby decreasing the bile acid pool in the liver and reducing cholestasis. This mechanism is crucial for PFIC patients as it helps to alleviate symptoms like pruritus and liver damage caused by the accumulation of bile acids. Other treatments may include ursodeoxycholic acid (UDCA), which improves bile flow and reduces liver inflammation, and surgical options like partial external biliary diversion (PEBD) or liver transplantation in severe cases. These treatments aim to manage symptoms, improve quality of life, and prevent progression to liver failure.
Reversal of advanced fibrosis after long-term ursodeoxycholic acid therapy in a patient with residual expression of MDR3.

Find a Location

Who is running the clinical trial?

Albireo, an Ipsen CompanyLead Sponsor
4 Previous Clinical Trials
528 Total Patients Enrolled
AlbireoLead Sponsor
16 Previous Clinical Trials
1,084 Total Patients Enrolled
Ipsen Medical DirectorStudy DirectorIpsen
260 Previous Clinical Trials
56,142 Total Patients Enrolled

Media Library

A4250 (odevixibat) (Bile Acid Sequestrant) Clinical Trial Eligibility Overview. Trial Name: NCT03659916 — Phase 3
Progressive Familial Intrahepatic Cholestasis Research Study Groups: A4250
Progressive Familial Intrahepatic Cholestasis Clinical Trial 2023: A4250 (odevixibat) Highlights & Side Effects. Trial Name: NCT03659916 — Phase 3
A4250 (odevixibat) (Bile Acid Sequestrant) 2023 Treatment Timeline for Medical Study. Trial Name: NCT03659916 — Phase 3
~16 spots leftby Dec 2025