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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04674761




Registration number
NCT04674761
Ethics application status
Date submitted
10/12/2020
Date registered
19/12/2020
Date last updated
2/11/2023

Titles & IDs
Public title
Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome
Scientific title
A Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients With Alagille Syndrome (ASSERT)
Secondary ID [1] 0 0
A4250-012
Universal Trial Number (UTN)
Trial acronym
ASSERT
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Alagille Syndrome 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Cardiovascular 0 0 0 0
Other cardiovascular diseases
Oral and Gastrointestinal 0 0 0 0
Other diseases of the mouth, teeth, oesophagus, digestive system including liver and colon
Other 0 0 0 0
Research that is not of generic health relevance and not applicable to specific health categories listed above

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - Odevixibat
Treatment: Drugs - Placebo

Experimental: Odevixibat (A4250) - Capsules for oral administration once daily for 24 weeks.

Placebo comparator: Placebo - Capsules for oral administration (to match active) once daily for 24 weeks.


Treatment: Drugs: Odevixibat
Odevixibat is a small molecule and selective inhibitor of IBAT.

Treatment: Drugs: Placebo
Placebo identical in appearance to experimental drug (odevixibat).
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change From Baseline in Scratching Score
Timepoint [1] 0 0
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 outcome [1] 0 0
Serum Bile Acid Levels
Timepoint [1] 0 0
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.

Eligibility
Key inclusion criteria
Key

* 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
Minimum age
No limit
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
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

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s

The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Completed
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
19/03/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
9/09/2022
Sample size
Target
Accrual to date
Final
52
Recruitment in Australia
Recruitment state(s)
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Maryland
Country [3] 0 0
United States of America
State/province [3] 0 0
Massachusetts
Country [4] 0 0
United States of America
State/province [4] 0 0
Missouri
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
Oregon
Country [7] 0 0
United States of America
State/province [7] 0 0
Pennsylvania
Country [8] 0 0
Belgium
State/province [8] 0 0
Brussels
Country [9] 0 0
Canada
State/province [9] 0 0
Quebec
Country [10] 0 0
France
State/province [10] 0 0
Bron
Country [11] 0 0
France
State/province [11] 0 0
Lille
Country [12] 0 0
France
State/province [12] 0 0
Marseille
Country [13] 0 0
France
State/province [13] 0 0
Paris
Country [14] 0 0
Germany
State/province [14] 0 0
Berlin
Country [15] 0 0
Germany
State/province [15] 0 0
Hannover
Country [16] 0 0
Germany
State/province [16] 0 0
Tübingen
Country [17] 0 0
Israel
State/province [17] 0 0
Jerusalem
Country [18] 0 0
Israel
State/province [18] 0 0
Petah tikva
Country [19] 0 0
Italy
State/province [19] 0 0
Bergamo
Country [20] 0 0
Italy
State/province [20] 0 0
Firenze
Country [21] 0 0
Italy
State/province [21] 0 0
Padova
Country [22] 0 0
Italy
State/province [22] 0 0
Rome
Country [23] 0 0
Malaysia
State/province [23] 0 0
Kuala Lumpur
Country [24] 0 0
Netherlands
State/province [24] 0 0
Groningen
Country [25] 0 0
Netherlands
State/province [25] 0 0
Utrecht
Country [26] 0 0
New Zealand
State/province [26] 0 0
Auckland
Country [27] 0 0
Poland
State/province [27] 0 0
Warszawa
Country [28] 0 0
Turkey
State/province [28] 0 0
Ankara
Country [29] 0 0
Turkey
State/province [29] 0 0
Fatih
Country [30] 0 0
United Kingdom
State/province [30] 0 0
London

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Albireo
Address
Country

Ethics approval
Ethics application status

Summary
Brief 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.
Trial website
https://clinicaltrials.gov/study/NCT04674761
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04674761