Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome

Part of paid clinical trials in San Diego, California.

Sponsor
Albireo, an Ipsen Company
Study ID
NCT05035030
Phase
PHASE3
Status
Recruiting
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Conditions

  • Alagille Syndrome

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • Odevixibat — DRUG
    Odevixibat is a small molecule and selective inhibitor of IBAT.

Study Details

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.

Key Dates

Start date
Sep 3, 2021
Status verified
May 2026
Primary completion
Dec 31, 2026
Completion
Dec 31, 2026

Study Design

Enrollment
70 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Odevixibat (A4250)
    Capsules for oral administration once daily for 72 weeks.

Primary Outcome Measure

Change from baseline in pruritus [ Time Frame: Baseline to week 72 (cohort 1). ]

Central Contacts

Locations (18)

FacilityCityStateZIPSite coordinators
Rady Children's HospitalSan DiegoCalifornia92123-
UCSFSan FranciscoCalifornia94158
Joanna Curry
[email protected]
Philip Rosenthal, MD (PRINCIPAL_INVESTIGATOR)
Children's Healthcare of AtlantaAtlantaGeorgia30329-
Riley Hospital for Children at IU HealthIndianapolisIndiana46202-
Johns Hopkins HospitalBaltimoreMaryland21287
Trung Van
[email protected]
410-614-8583
Ashley Thomas
[email protected]
410-955-9161
Wikrom Karnsakul (PRINCIPAL_INVESTIGATOR)
Boston Children's HospitalBostonMassachusetts02115-
Children's Mercy Hospital and ClinicsKansas CityMissouri64018
Heather Wasserkbury
[email protected]
816-302-9735
Ryan Fisher (PRINCIPAL_INVESTIGATOR)
Northwell Health SystemNew Hyde ParkNew York11042
Shari Sheflin-Findling (PRINCIPAL_INVESTIGATOR)
Hassenfeld Children's Hospital at NYU LangoneNew YorkNew York10016
Rozanne Groen
[email protected]
929-392-4083
Nadia Ovchinsky, MD (PRINCIPAL_INVESTIGATOR)
New York-Presbyterian / Columbia University Irving Medical CenterNew YorkNew York10032-
The Childrens Hospital at Montefiore Albert Einstein School of MedicineThe BronxNew York10467-
Atrium Health Carolinas MedicalDurhamNorth Carolina27710
Vani Gopalareddy (PRINCIPAL_INVESTIGATOR)
Cincinnati Children's HospitalCincinnatiOhio45229
Akihiro Asai (PRINCIPAL_INVESTIGATOR)
Oregon Health Science University School of MedicinePortlandOregon97239-
Monroe Carell Jr. Childrens Hospital at VanderbiltNashvilleTennessee37232-
Childrens Medical Center of Dallas University of Texas SouthwesternDallasTexas75207-
Texas Children's HospitalHoustonTexas77030-
Texas Liver InstituteSan AntonioTexas78215-

Find similar trials in San Diego, CA

By research site
Rady Children's Hospital· San Diego, CAUCSF· San Francisco, CAChildren's Healthcare of Atlanta· Atlanta, GARiley Hospital for Children at IU Health· Indianapolis, INJohns Hopkins Hospital· Baltimore, MDBoston Children's Hospital· Boston, MA

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