Actively Recruiting
Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome
Led by Albireo, an Ipsen Company · Updated on 2026-04-30
70
Participants Needed
39
Research Sites
277 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
The purpose of this study is to assess the long-term safety and effectiveness of odevixibat in participants with Alagille syndrome (ALGS). The participants of this study will have ALGS a rare genetic disorder that can affect multiple organ systems of the body including the liver, heart, skeleton, eyes and kidneys. Common symptoms, which often develop during the first three months of life, include blockage of the flow of bile from the liver (cholestasis), yellowing of the skin and mucous membranes (jaundice), poor weight gain and growth and severe itching (pruritis). The drug used for the study is odevixibat and was authorized for the treatment of cholestatic pruritus in infants with ALGS over 12 months of age by the United States Food and Drug Administration on 13 June 2023.
CONDITIONS
Official Title
Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Cohort 1: Completed 24-week treatment period of previous study A4250-012
- Signed informed consent and assent as appropriate; re-consent required if patient turns 18 or legal age during study
- Caregivers and age-appropriate patients willing and able to use an electronic diary device
- Sexually active males and females agree to use reliable contraception from consent through 90 days after last dose
- Cohort 2: Infant with clinically confirmed Alagille syndrome, 11 months old or younger at study start
- Body weight 2 kg or more at study start
- Gestational age 36 weeks or more; if born between 32 and 36 weeks, postmenstrual age must be at least 36 weeks
- Signed parent or legal guardian informed consent
You will not qualify if you...
- Cohort 1: Decompensated liver disease or history of significant ascites, variceal hemorrhage, or encephalopathy
- Non-compliance with study treatment or procedures in prior study A4250-012
- Any condition that may compromise safety or study participation per investigator
- Known allergy to odevixibat components
- Cohort 2: Other liver diseases such as biliary atresia, progressive familial intrahepatic cholestasis, or benign recurrent intrahepatic cholestasis
- Diseases affecting drug absorption, distribution, metabolism, or excretion including inflammatory bowel disease
- Chronic diarrhea requiring IV fluids or nutrition
- Past or active significant infections including HIV
- Recent serious infection needing hospitalization or IV antibiotics within 4 weeks, or oral antibiotics within 2 weeks prior to screening
- Cancer diagnosis except basal cell carcinoma
- Chronic kidney disease with impaired kidney function (GFR less than 70 mL/min/1.73 m2)
- Recent biliary diversion surgery within 6 months
- Liver transplant history or planned within 6 months
- Decompensated liver disease or significant liver complications
- High INR above 1.4 unless corrected
- Very high liver enzymes or bilirubin levels beyond specified limits
- Uncontrolled severe itching conditions unrelated to Alagille syndrome
- Exposure to alcohol or substance abuse in utero
- Use of bile acid or lipid binding resins or drugs slowing gastrointestinal motility
- Recent investigational drug or device use within 30 days or 5 half-lives
- Any other condition that may affect safety or study completion as judged by investigator
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
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
Not Yet Recruiting
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
Not Yet Recruiting
20
The Royal Children's Hospital Melbourne
Parkville, Victoria, Australia, 3052
Not Yet Recruiting
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
Not Yet Recruiting
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
Not Yet Recruiting
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
CONTACT
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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