Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome

Alagille Syndrome Clinical Trial

— ASSERT  

Official title:

A Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients With Alagille Syndrome (ASSERT)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04674761
• Other study ID #: A4250-012
• Status: Completed
• Phase: Phase 3
• Start date: March 19, 2021
• Est. completion date: September 9, 2022

Study information

• Verified date: October 2023
• Source: Ipsen
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

Double-blind, randomized, placebo-controlled, Phase 3 study to investigate the efficacy and safety of odevixibat compared to placebo in Patients with Alagille Syndrome.

Description:

Approximately 35 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 52
• Est. completion date: September 9, 2022
• Est. primary completion date: September 9, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Key Inclusion Criteria:

• Genetically confirmed diagnosis of Alagille syndrome
• History of significant pruritus as measured by the Albireo Observer or Patient Reported Outcome instrument
• Elevated serum bile acid level

Key Exclusion Criteria:

• History or ongoing presence of other types of liver disease (eg. biliary atresia, progressive familial intrahepatic cholestasis, hepatocellular carcinoma)
• History of liver transplant, or a liver transplant is planned within 6 months of randomization
• ALT >10× upper limit of normal (ULN) at screening
• Total bilirubin >15 × ULN at screening
• Patient suffers from uncontrolled, recalcitrant pruritic condition other than Alagille syndrome

Related Conditions & MeSH terms

• Alagille Syndrome
• Syndrome

Intervention

Drug:
• Odevixibat
Odevixibat is a small molecule and selective inhibitor of IBAT.
• Placebo
Placebo identical in appearance to experimental drug (odevixibat).

Locations

Country	Name	City	State
Belgium	Cliniques Universitaires Saint-Luc Bruxelles	Brussels
Canada	CHU Sainte-Justine	Montréal	Quebec
France	Hôpital Femme Mère Enfant de Lyon	Bron
France	Antenne pédiatrique du CIC - Hopital Jeanne De Flandre	Lille
France	APHM	Marseille
France	Hopital Necker Enfants Malades	Paris
Germany	Charité-Universitätsmedizin Berlin	Berlin
Germany	Medizinische Hochschule Hannover	Hannover
Germany	Universitätsklinik für Kinder-und Jugendmedizin Tübingen	Tübingen
Israel	Sharie Zedek	Jerusalem
Israel	Schneider Children's Medical Center of Israel	Petah tikva
Italy	ASST Papa Giovanni XXIII	Bergamo
Italy	AOU Meyer	Firenze
Italy	Azienda Ospedale Università Padova	Padova
Italy	Bambino Gesù Children's Hospital	Rome
Malaysia	University of Malaya Medical Centre	Kuala Lumpur
Netherlands	Universitair Medisch Centrum Groningen (UMCG)	Groningen
Netherlands	University Medical Centre Utrecht, WKZ	Utrecht
New Zealand	Starship Child Health	Auckland
Poland	Instytut Pomnik-Centrum Zdrowia Dziecka	Warszawa
Turkey	Hacettepe Üniversitesi Ihsan Dogramaci Çocuk Hastanesi	Ankara
Turkey	Istanbul University Medical Faculty Hospital	Fatih
United Kingdom	King's College Hospital	London
United States	Johns Hopkins Hospital	Baltimore	Maryland
United States	Boston Children's Hospital	Boston	Massachusetts
United States	The Childrens Hospital at Montefiore Albert Einstein School of Medicine	Bronx	New York
United States	Children's Mercy Hospital and Clinics	Kansas City	Missouri
United States	Columbia University Medical Center	New York	New York
United States	Icahn School of Medicine at Mount Sinai	New York	New York
United States	UPMC Children's Hospital of Pittsburgh	Pittsburgh	Pennsylvania
United States	Oregon Health Science University School of Medicine	Portland	Oregon
United States	University of California - San Francisco	San Francisco	California

Sponsors (1)

Lead Sponsor	Collaborator
Albireo

Countries where clinical trial is conducted

United States,  Belgium,  Canada,  France,  Germany,  Israel,  Italy,  Malaysia,  Netherlands,  New Zealand,  Poland,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change From Baseline in Scratching Score	Change from baseline in average AM (measured after waking up) and PM (measured before bedtime) scratching score to Month 6 as measured by the Albireo Observer-Reported Outcome (ObsRO) Instrument. The ObsRO instrument was used to assess severity of observed scratching twice a day (AM and PM) with scores from 0 to 4 where 0 is no scratching and 4 is worst possible scratching.	Change from baseline for each four-week average pruritis score to Month 6 (Weeks 21 to 24), in which baseline was calculated based on the 14 days before the start of treatment.
Secondary	Serum Bile Acid Levels	Change in serum bile acid levels (µmol/L) from baseline to average of week 20 and 24	Change from baseline to average of week 20 and 24, where baseline was calculated by averaging the last two values preceding start of treatment, and average of Week 20 and Week 24 was defined as the average of Week 20 and Week 24 values.