Long-term Safety and Efficacy of Odevixibat in Patients With Alagille Syndrome
Condition(s) studied
Investigational drug(s) / intervention(s)
Odevixibat: Odevixibat is a small molecule and selective inhibitor of IBAT.
Study summary
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.
Eligibility
Primary outcome measure(s)
- Change from baseline in pruritus — Baseline to week 72 (cohort 1).
Assessed as change in scratching score as measured by measured by the Albireo Observer-Reported Outcome Caregiver Instrument. - Percentage of participants with Treatment Emergent Adverse Event (TEAEs) and Serious Adverse Events (SAEs) — Baseline to week 12 (cohort 2).
An Adverse event (AE) is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention. A serious adverse event (SAE) is an AE that results in any of following outcomes: death; life threatening; persistent/significant disability/incapacity; initial or prolonged inpatient hospitalization; congenital anomaly/birth defect or was otherwise considered medically important. Treatment-emergent adverse events. TEAEs included both Serious TEAEs and non-serious TEAEs. - Percentage of participants with clinically significant changes from baseline in Physical Examination — Baseline to week 12 (cohort 2).
The clinical significance will be graded by the investigator. - Percentage of participants with clinically significant changes in Laboratory Parameters — Baseline to week 12 (cohort 2).
The following laboratory parameters will be reported: blood chemistry, hematology and coagulation. The clinical significance will be graded by the investigator. - Percentage of participants with clinically significant changes from baseline in Vital Signs. — Baseline to week 12 (cohort 2).
The clinical significance will be graded by the investigator. - Change from baseline in concomitant medications. — Baseline to week 12 (cohort 2).
- Change from baseline in fat-soluble vitamin levels. — Baseline to week 12 (cohort 2).
Trial sites (35)
| Facility | City | Region | Status |
|---|---|---|---|
| Rady Children's Hospital | San Diego | California | |
| UCSF | San Francisco | California | |
| Children's Healthcare of Atlanta | Atlanta | Georgia | |
| Riley Hospital for Children at IU Health | Indianapolis | Indiana | |
| Johns Hopkins Hospital | Baltimore | Maryland | |
| Boston Children's Hospital | Boston | Massachusetts | |
| Children's Mercy Hospital and Clinics | Kansas City | Missouri | |
| Northwell Health System | New Hyde Park | New York | |
| Hassenfeld Children's Hospital at NYU Langone | New York | New York | |
| New York-Presbyterian / Columbia University Irving Medical Center | New York | New York | |
| The Childrens Hospital at Montefiore Albert Einstein School of Medicine | The Bronx | New York | |
| Atrium Health Carolinas Medical | Durham | North Carolina | |
| Cincinnati Children's Hospital | Cincinnati | Ohio | |
| Oregon Health Science University School of Medicine | Portland | Oregon | |
| Monroe Carell Jr. Childrens Hospital at Vanderbilt | Nashville | Tennessee | |
| Childrens Medical Center of Dallas University of Texas Southwestern | Dallas | Texas | |
| Texas Children's Hospital | Houston | Texas | |
| Texas Liver Institute | San Antonio | Texas | |
| Cliniques Universitaires Saint-Luc Bruxelles | Brussels | Belgium | |
| Hôpital Femme Mère Enfant de Lyon | Bron | France | |
| Antenne pediatrique du CIC-Hopital Jeanne De Flandre | Lille | France | |
| Hopital Necker Enfants Malades | Paris | France | |
| Charité - Universitätsmedizin Berlin | Berlin | Germany | |
| Medizinische Hochschul | Hanover | Germany | |
| Universitatsklinik fur Kinder-und Jugendmedizin Tubingen | Tübingen | Germany | |
| AOU Meyer | Florence | Italy | |
| Azienda Ospedale University | Padova | Italy | |
| Ospedale Pediatrico Bambino Gesu | Rome | Italy | |
| University of Malaya Medical Center | Kuala Lumpur | Malaysia | |
| Universitair Medisch Centrum Groningen | Groningen | Netherlands | |
| Wilhelmina Children's Hospital UMCU Utrecht | Utrecht | Netherlands | |
| Instytut Pomnik-Centrum Zdrowia Dzieck | Warsaw | Poland | |
| Istanbul University Istanbul Medical Faculty Hospital | Istanbul | Turkey (Türkiye) | |
| Birmingham Women's and Children's NHS Foundation Trust | Birmingham | United Kingdom | |
| King's College Hospital NHS Foundation Trust King's College Hospital Paediatric Research | London | United Kingdom |
More Albireo, an Ipsen Company trials in France
Other trials for the same condition
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05035030 on ClinicalTrials.gov ↗ ← All trials in France