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

Alagille Syndrome Clinical Trial

— ASSERT-EXT  

Official title:

An Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients With Alagille Syndrome (ASSERT-EXT)

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05035030
• Other study ID #: A4250-015
• Status: Active, not recruiting
• Phase: Phase 3
• Start date: September 3, 2021
• Est. completion date: March 31, 2025

Study information

• Verified date: April 2024
• Source: Ipsen
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

An Open Label Study to Evaluate the Long-term Safety and Efficacy of Odevixibat (A4250) in Patients with Alagille Syndrome (ASSERT-EXT)

Description:

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

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 50
• Est. completion date: March 31, 2025
• Est. primary completion date: February 7, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

Inclusion Criteria:

1. Completion of the 24-week Treatment Period of Study A4250-012

2. Signed informed consent and assent as appropriate. Patients who turn 18 years of age (or legal age per country) during the study will be required to re-consent to remain on the study

3. Caregivers (and age-appropriate patients) must be willing and able to use an electronic diary (eDiary) device as required by the study

4. Sexually active males and females must agree to use a reliable contraceptive method with =1% failure rate (such as hormonal contraception, intra-uterine device, or complete abstinence) from signed informed consent through 90 days after last dose of study drug.

Exclusion Criteria:

1. Decompensated liver disease, history or presence of clinically significant ascites, variceal hemorrhage, and/or encephalopathy

2. Patients who were not compliant with study drug treatment or procedures in Study A4250-012

3. Any other conditions or abnormalities which, in the opinion of the investigator, may compromise the safety of the patient, or interfere with the patient participating in or completing the study

4. Known hypersensitivity to any components of odevixibat

Related Conditions & MeSH terms

• Alagille Syndrome
• Syndrome

Intervention

Drug:
• Odevixibat
Odevixibat is a small molecule and selective inhibitor of IBAT.

Locations

Country	Name	City	State
Belgium	Cliniques Universitaires Saint-Luc Bruxelles	Bruxelles
France	Antenne pediatrique du CIC-Hopital Jeanne De Flandre	Lille
France	Hopital Necker Enfants Malades	Paris
Germany	Charité - Universitätsmedizin Berlin	Berlin
Germany	Medizinische Hochschul	Hannover
Germany	Universitatsklinik fur Kinder-und Jugendmedizin Tubingen	Tübingen
Italy	AOU Meyer	Florence
Italy	Azienda Ospedale University	Padova
Italy	Ospedale Pediatrico Bambino Gesu	Rome
Malaysia	University of Malaya Medical Center	Kuala Lumpur
Netherlands	Universitair Medisch Centrum Groningen	Groningen
Netherlands	University Medical Center	Utrecht
Poland	Instytut Pomnik-Centrum Zdrowia Dzieck	Warszawa
Turkey	Istanbul University Istanbul Medical Faculty Hospital	Istanbul
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	Hassenfeld Children's Hospital at NYU Langone	New York	New York
United States	UCSF	San Francisco	California

Sponsors (1)

Lead Sponsor	Collaborator
Albireo

Countries where clinical trial is conducted

United States,  Belgium,  France,  Germany,  Italy,  Malaysia,  Netherlands,  Poland,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in pruritus	Change from baseline in scratching score as measured by the Albireo Observer-Reported Outcome Caregiver Instrument	Baseline to week 72
Secondary	Change in serum bile acids	Change from baseline in serum bile acids	Baseline to week 72
Secondary	Change in Quality of Life	Change from baseline in quality-of-life assessment as measured by the Pediatric Quality of Life Inventory Questionnaire	Baseline to week 72
Secondary	Change in Sleep Parameters	Change from baseline in sleep parameters as measured by the Albireo Patient and Observer-Reported Outcome instruments	Baseline to week 72
Secondary	Change in Global Symptom Relief	Change from baseline in global symptom relief as measured by both the Global Impression of Symptoms and Global Impression of Change questionnaires	Baseline to Weeks, 4, 12, 24, 48, and 72
Secondary	Safety and Tolerability	Safety and Tolerability as assessed by the Number of Participants with Treatment-emergent Adverse Events as Assessed by CTCAE v5.0	Baseline to week 72