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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
Timeline
Screening 3 weeks
Treatment Varies
Follow Up weeks 0-4, 0-12, 0-22, 0-24, 0-36, 0-46, 0-48, 0-60, 0-70, or the proportion of positive pruritus assessments at each 4-week interval between v1/screening and v5/week 24, then by each visit between v5/week 24 to v12/week 76
Awards & highlights
Study Summary
This trial is testing a new medication for children with a rare liver disease. The goal is to see if it is safe and effective over the long term.
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.Check my eligibility
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.See study design
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.
Timeline
Screening ~ 3 weeks3 visits
Treatment ~ Varies
Follow Up ~ weeks 0-4, 0-12, 0-22, 0-24, 0-36, 0-46, 0-48, 0-60, 0-70, or the proportion of positive pruritus assessments at each 4-week interval between v1/screening and v5/week 24, then by each visit between v5/week 24 to v12/week 76
Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~weeks 0-4, 0-12, 0-22, 0-24, 0-36, 0-46, 0-48, 0-60, 0-70, or the proportion of positive pruritus assessments at each 4-week interval between v1/screening and v5/week 24, then by each visit between v5/week 24 to v12/week 76
Treatment Details
Study Objectives
Outcome measures can provide a clearer picture of what you can expect from a treatment.Primary outcome measures
EU and rest of world: Change in serum bile acids
US: Change in Pruritus
Secondary outcome measures
All regions. All-cause mortality
All regions: 3. Change from baseline in serum bile acids at Weeks 4, 12, 22, 24, 36, 46, 48, 60, 70, 72, and 76
All regions: Change in AST to platelet ratio idex (APRI) score
+11 moreSide 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%
Otitis media
11%
Vitamin D deficiency
11%
Nasopharyngitis
11%
Cough
11%
Splenomegaly
11%
Blood bilirubin increased
11%
Blood alkaline phosphatase increased
5%
Cholelithiasis
5%
Respiratory tract infection
5%
Supraventricular tachycardia
5%
Cardiac ablation
5%
Urinary tract infection
5%
Vitamin D decreased
5%
Liver function test increased
5%
Otorrhoea
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
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
Find a Location
Who is running the clinical trial?
AlbireoLead Sponsor
16 Previous Clinical Trials
1,055 Total Patients Enrolled
Ipsen Medical DirectorStudy DirectorIpsen
258 Previous Clinical Trials
55,178 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.
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