Actively Recruiting
An Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients With Alagille Syndrome
Led by Albireo, an Ipsen Company · Updated on 2026-05-28
70
Participants Needed
39
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the long-term safety and effectiveness of odevixibat in people with Alagille syndrome (ALGS), a rare genetic disorder affecting multiple organs including the liver, heart, skeleton, eyes, and kidneys. Symptoms often start in the first three months of life and include bile flow blockage, jaundice, poor growth, and severe itching. Odevixibat was authorized by the FDA in June 2023 for treating cholestatic itching in infants with ALGS aged over 12 months. This Phase 3, open-label study includes two groups: Cohort 1, participants from a prior study who meet entry criteria, and Cohort 2, infants under 12 months old with ALGS. The study has up to three periods: a treatment period lasting 72 weeks for Cohort 1 or 12 weeks for Cohort 2, with daily oral doses of 120 mcg/kg odevixibat; an optional extension for continued treatment visits every 16 weeks until commercial availability; and a safety follow-up period lasting 2 to 4 weeks for those not continuing treatment. Participants will attend clinic visits every 4 to 12 weeks during treatment and may continue some other medications with records kept. Assessments include blood and urine sampling, physical exams, and clinical evaluations. Cohort 1 participants will complete electronic diaries and questionnaires. Researchers will measure changes in itching, bile acid levels, lab tests, vital signs, medication use, quality of life, and adverse events. The study continues until late 2026 with ongoing safety and efficacy monitoring.
CONDITIONS
Brief Title
Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Completion of the 24-week Treatment Period of Study A4250-012 (Cohort 1)
- Signed informed consent and assent as appropriate
- Willingness and ability to use an electronic diary device (Cohort 1)
- Sexually active participants must agree to use reliable contraception from consent through 90 days after last dose (Cohort 1)
- Infant with clinically confirmed Alagille syndrome, 11 months of age or younger at Study Day 1 (Cohort 2)
- Body weight of at least 2 kg at Study Day 1 (Cohort 2)
- Gestational age of 36 weeks or more, or postmenstrual age of 36 weeks if born between 32 and 36 weeks (Cohort 2)
- Signed parent or legal guardian informed consent (Cohort 2)
You will not qualify if you...
- Decompensated liver disease or history of significant ascites, variceal hemorrhage, or encephalopathy (Cohorts 1 and 2)
- Non-compliance with study drug or procedures in prior study A4250-012 (Cohort 1)
- Known hypersensitivity to any components of odevixibat (Cohort 1)
- Other liver diseases such as biliary atresia, progressive familial intrahepatic cholestasis, or benign recurrent intrahepatic cholestasis (Cohort 2)
- Conditions interfering with drug metabolism or absorption, including inflammatory bowel disease (Cohort 2)
- Chronic diarrhea requiring intravenous fluids or nutritional support (Cohort 2)
- Active or past infection with HIV or other significant chronic infections (Cohort 2)
- Recent serious infections requiring hospitalization or anti-infective treatment (Cohort 2)
- Cancer diagnosis except basal cell carcinoma (Cohort 2)
- Chronic kidney disease with impaired renal function (Cohort 2)
- Recent biliary diversion surgery or planned liver transplant (Cohort 2)
- High international normalized ratio (INR) or elevated liver enzymes beyond set limits (Cohort 2)
- Uncontrolled pruritic conditions other than ALGS (Cohort 2)
- Exposure to alcohol or substance abuse in utero (Cohort 2)
- Use of bile acid or lipid binding resins and drugs that slow gut motility (Cohort 2)
- Recent investigational drug or device use within 30 days or 5 half-lives (Cohort 2)
- Any other condition that may compromise safety or study participation (Both cohorts)
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 72 weeks for Cohort 1 or 12 weeks for Cohort 2
Participants receive daily doses of odevixibat and attend clinic visits for assessments and medication management.
Visits every 4 to 12 weeks during treatment period
Duration - Variable duration until drug availability
Participants who choose to continue treatment with odevixibat after the treatment period may remain on the drug with less frequent visits until it becomes commercially available.
Visits every 16 weeks during extension period
Duration - 4 weeks for Cohort 1 or 2 weeks for Cohort 2
Participants who do not continue into the optional extension period have a short safety follow-up to monitor for any adverse effects after stopping treatment.
1 follow-up visit (in-person)
Trial Site Locations
Total: 39 locations
1
Rady Children's Hospital
San Diego, California, United States, 92123
Actively Recruiting
2
UCSF
San Francisco, California, United States, 94158
Actively Recruiting
3
Children's Healthcare of Atlanta
Atlanta, Georgia, United States, 30329
Actively Recruiting
4
Riley Hospital for Children at IU Health
Indianapolis, Indiana, United States, 46202
Actively Recruiting
5
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287
Actively Recruiting
6
Boston Children's Hospital
Boston, Massachusetts, United States, 02115
Completed
7
Children's Mercy Hospital and Clinics
Kansas City, Missouri, United States, 64018
Actively Recruiting
8
Northwell Health System
New Hyde Park, New York, United States, 11042
Actively Recruiting
9
Hassenfeld Children's Hospital at NYU Langone
New York, New York, United States, 10016
Actively Recruiting
10
New York-Presbyterian / Columbia University Irving Medical Center
New York, New York, United States, 10032
Terminated
11
The Childrens Hospital at Montefiore Albert Einstein School of Medicine
The Bronx, New York, United States, 10467
Completed
12
Atrium Health Carolinas Medical
Durham, North Carolina, United States, 27710
Actively Recruiting
13
Cincinnati Children's Hospital
Cincinnati, Ohio, United States, 45229
Actively Recruiting
14
Oregon Health Science University School of Medicine
Portland, Oregon, United States, 97239
Actively Recruiting
15
Monroe Carell Jr. Childrens Hospital at Vanderbilt
Nashville, Tennessee, United States, 37232
Actively Recruiting
16
Childrens Medical Center of Dallas University of Texas Southwestern
Dallas, Texas, United States, 75207
Actively Recruiting
17
Texas Children's Hospital
Houston, Texas, United States, 77030
Actively Recruiting
18
Texas Liver Institute
San Antonio, Texas, United States, 78215
Actively Recruiting
19
Children's Hospital Queensland
South Brisbane, Queensland, Australia, 4101
Terminated
20
The Royal Children's Hospital Melbourne
Parkville, Victoria, Australia, 3052
Terminated
21
Cliniques Universitaires Saint-Luc Bruxelles
Brussels, Belgium, 1200
Completed
22
Hôpital Femme Mère Enfant de Lyon
Bron, France, 69677
Terminated
23
Antenne pediatrique du CIC-Hopital Jeanne De Flandre
Lille, France
Completed
24
Hopital Necker Enfants Malades
Paris, France, 75015
Completed
25
Charité - Universitätsmedizin Berlin
Berlin, Germany, 13353
Active, Not Recruiting
26
Medizinische Hochschul
Hanover, Germany, 30625
Active, Not Recruiting
27
Universitatsklinik fur Kinder-und Jugendmedizin Tubingen
Tübingen, Germany, 72076
Completed
28
Shaare Zedek Schneider Children Medical
Petah Tikva, Israel
Terminated
29
AOU Meyer
Florence, Italy
Completed
30
Azienda Ospedale University
Padova, Italy, 35128
Active, Not Recruiting
31
Ospedale Pediatrico Bambino Gesu
Rome, Italy, 00165
Active, Not Recruiting
32
University of Malaya Medical Center
Kuala Lumpur, Malaysia, 59100
Actively Recruiting
33
Universitair Medisch Centrum Groningen
Groningen, Netherlands, 9713 GZ
Completed
34
Wilhelmina Children's Hospital UMCU Utrecht
Utrecht, Netherlands
Completed
35
Instytut Pomnik-Centrum Zdrowia Dzieck
Warsaw, Poland, 04-730
Active, Not Recruiting
36
National Taiwan University Hospital
Taipei, Taiwan
Terminated
37
Istanbul University Istanbul Medical Faculty Hospital
Istanbul, Turkey (Türkiye)
Active, Not Recruiting
38
Birmingham Women's and Children's NHS Foundation Trust
Birmingham, United Kingdom
Actively Recruiting
39
King's College Hospital NHS Foundation Trust King's College Hospital Paediatric Research
London, United Kingdom, SE5 9RS
Actively Recruiting
Research Team
I
Ipsen Recruitment enquiries
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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