Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome

Phase 3

Completed

• Conditions: Alagille Syndrome
• Interventions: Drug: Placebo; Drug: Odevixibat

• Registration Number: NCT04674761
• Lead Sponsor: Albireo

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.

Study Timeline

• Study First Submitted: 2020-12-10
• Study First Submitted QC: 2020-12-17
• Study First Posted: 2020-12-19
• Study Start: 2021-03-19
• Primary Completion: 2022-09-09
• Study Completion: 2022-09-09
• Results First Submitted: 2023-09-08
• Status Verified: 2023-10
• Results First Submitted QC: 2023-10-10
• Last Update Submitted: 2023-10-10
• Results First Posted: 2023-11-02
• Last Update Posted: 2023-11-02

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 52

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	Capsules for oral administration (to match active) once daily for 24 weeks.
Odevixibat (A4250)	Odevixibat	Capsules for oral administration once daily for 24 weeks.

Primary Outcome Measures

Name	Time	Method
Change From Baseline in Scratching Score	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.	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.

Secondary Outcome Measures

Name	Time	Method
Serum Bile Acid Levels	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.	Change in serum bile acid levels (μmol/L) from baseline to average of week 20 and 24

Trial Locations

Locations (32)

Antenne pédiatrique du CIC - Hopital Jeanne De Flandre; 🇫🇷 Lille, France

Hopital Necker Enfants Malades; 🇫🇷 Paris, France

Istanbul University Medical Faculty Hospital; 🇹🇷 Fatih, Turkey

CHU Sainte-Justine; 🇨🇦 Montréal, Quebec, Canada

Cliniques Universitaires Saint-Luc Bruxelles; 🇧🇪 Brussels, Belgium

Instytut Pomnik-Centrum Zdrowia Dziecka; 🇵🇱 Warszawa, Poland

Schneider Children's Medical Center of Israel; 🇮🇱 Petah tikva, Israel

Universitair Medisch Centrum Groningen (UMCG); 🇳🇱 Groningen, Netherlands

University of Malaya Medical Centre; 🇲🇾 Kuala Lumpur, Malaysia

University Medical Centre Utrecht, WKZ; 🇳🇱 Utrecht, Netherlands

Icahn School of Medicine at Mount Sinai; 🇺🇸 New York, New York, United States

APHM; 🇫🇷 Marseille, France

Columbia University Medical Center; 🇺🇸 New York, New York, United States

Hôpital Femme Mère Enfant de Lyon; 🇫🇷 Bron, France

Sharie Zedek; 🇮🇱 Jerusalem, Israel

Universitätsklinik für Kinder-und Jugendmedizin Tübingen; 🇩🇪 Tübingen, Germany

Medizinische Hochschule Hannover; 🇩🇪 Hannover, Germany

Charité-Universitätsmedizin Berlin; 🇩🇪 Berlin, Germany

ASST Papa Giovanni XXIII; 🇮🇹 Bergamo, Italy

AOU Meyer; 🇮🇹 Firenze, Italy

Azienda Ospedale Università Padova; 🇮🇹 Padova, Italy

Bambino Gesù Children's Hospital; 🇮🇹 Rome, Italy

Hacettepe Üniversitesi İhsan Doğramacı Çocuk Hastanesi; 🇹🇷 Ankara, Turkey

Starship Child Health; 🇳🇿 Auckland, New Zealand

King's College Hospital; 🇬🇧 London, United Kingdom

University of California - San Francisco; 🇺🇸 San Francisco, California, United States

Johns Hopkins Hospital; 🇺🇸 Baltimore, Maryland, United States

Boston Children's Hospital; 🇺🇸 Boston, Massachusetts, United States

Children's Mercy Hospital and Clinics; 🇺🇸 Kansas City, Missouri, United States

The Childrens Hospital at Montefiore Albert Einstein School of Medicine; 🇺🇸 Bronx, New York, United States

Oregon Health Science University School of Medicine; 🇺🇸 Portland, Oregon, United States

UPMC Children's Hospital of Pittsburgh; 🇺🇸 Pittsburgh, Pennsylvania, United States