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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 belowSelect...
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 weeks3 visits
Treatment ~ Varies
Follow Up ~ from baseline to weeks 24, 48, and 72
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~from baseline to weeks 24, 48, and 72
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 • NCT0356623826%
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.
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
Eligibility Criteria:
This trial includes the following eligibility criteria:- I have a condition that affects how my body processes medication.I have PFIC and have experienced significant itching, noted by myself or my caregiver.I am taking medication that affects bile acid, lipids, or slows my digestion.My INR is above 1.4, but I can be treated to lower it for inclusion.I have not been part of any medical study or taken any experimental drugs or used any experimental devices in the last 30 days or 5 half-lives of the study agent, whichever is longer.I have PFIC with bile acid levels ≥100 μmol/L, confirmed by 2 tests.I (or my guardian) have signed the consent form, and I will sign again if I turn 18 during the study.I have PFIC with current severe itching and high bile acid levels.I have severe liver disease with symptoms like bleeding, fluid buildup, or confusion.I am sexually active and use reliable contraception or practice complete abstinence.I have a liver condition other than PFIC, but may have significant portal hypertension.I have cancer, but it's not basal cell carcinoma or a non-liver cancer treated over 5 years ago without recurrence.I have had a liver transplant or will have one within 6 months.I have a severe itching skin condition that doesn't respond to treatment.I have a genetic variation in the ABCB11 gene leading to no BSEP protein.I am willing to use an eDiary device for the study.I am using or will use effective birth control during and 90 days after the study.I have PFIC or BRIC, weigh at least 5 kg, and can be any age.I have had a significant infection or HIV in the past, but it's currently under control.I have a caregiver who will be with me throughout the study.I have had chronic diarrhea for over 3 months needing IV fluids or nutritional support.I have severe liver issues, including bleeding, fluid buildup, or mental confusion.I have a genetic confirmation of PFIC.
Research Study Groups:
This trial has the following groups:- Group 1: A4250
Awards:
This trial has 2 awards, including:- 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.
Timeline:
This trial has the following timeline:- Screening: It may take up to 3 Weeks to process to see if you qualify in this trial.
- Treatment: The duration you will receive the treatment varies.
- Follow Ups: You may be asked to continue sharing information regarding the trial for 6 Months after you stop receiving the treatment.