Clinical trial NCT04674761: Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome
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)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Alagille Syndrome
Keywords: ALGS
Alagille syndrome
Lead sponsor: Ipsen
Sponsor class: INDUSTRY
First posted: 2020-12-19
Enrollment: 52
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

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

Eligibility criteria: 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

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