- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04674761
Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome (ASSERT)
October 10, 2023 updated by: Albireo
A Phase 3 Double-blind, Randomized, Placebo-controlled Study of the Safety and Efficacy of Odevixibat (A4250) in Patients With Alagille Syndrome (ASSERT)
Double-blind, randomized, placebo-controlled, Phase 3 study to investigate the efficacy and safety of odevixibat compared to placebo in Patients with Alagille Syndrome.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Detailed Description
Approximately 35 sites will be initiated for this study in North America, Europe, Middle East, and Asia Pacific.
Study Type
Interventional
Enrollment (Actual)
52
Phase
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Brussels, Belgium, 1200
- Cliniques Universitaires Saint-Luc Bruxelles
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Quebec
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Montréal, Quebec, Canada, H3T 1C5
- CHU Sainte-Justine
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Bron, France, 69677
- Hôpital Femme Mère Enfant de Lyon
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Lille, France, 59037
- Antenne pédiatrique du CIC - Hopital Jeanne De Flandre
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Marseille, France, 13385
- APHM
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Paris, France, 75015
- Hopital Necker Enfants Malades
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Berlin, Germany, 13353
- Charite-Universitatsmedizin Berlin
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Hannover, Germany, 30625
- Medizinische Hochschule Hannover
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Tübingen, Germany, 72076
- Universitatsklinik fur Kinder-und Jugendmedizin Tubingen
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Jerusalem, Israel, 9103102
- Sharie Zedek
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Petah tikva, Israel, 4920235
- Schneider Children's Medical Center of Israel
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Bergamo, Italy, 24127
- ASST Papa Giovanni XXIII
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Firenze, Italy, 50139
- AOU Meyer
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Padova, Italy, 35128
- Azienda Ospedale Università Padova
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Rome, Italy, 00165
- Bambino Gesù Children's Hospital
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Kuala Lumpur, Malaysia, 59100
- University of Malaya Medical Centre
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Groningen, Netherlands, 9713 GZ
- Universitair Medisch Centrum Groningen (Umcg)
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Utrecht, Netherlands, 3584EA
- University Medical Centre Utrecht, WKZ
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Auckland, New Zealand, 1023
- Starship Child Health
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Warszawa, Poland, 04-730
- Instytut Pomnik-Centrum Zdrowia Dziecka
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Ankara, Turkey, 06230
- Hacettepe Üniversitesi İhsan Doğramacı Çocuk Hastanesi
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Fatih, Turkey, 34093
- Istanbul University Medical Faculty Hospital
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London, United Kingdom, SE5 9RS
- King's College Hospital
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California
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San Francisco, California, United States, 94158
- University of California - San Francisco
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Maryland
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Baltimore, Maryland, United States, 21287
- Johns Hopkins Hospital
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Massachusetts
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Boston, Massachusetts, United States, 02115
- Boston Children's Hospital
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Missouri
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Kansas City, Missouri, United States, 64108
- Children's Mercy Hospital and Clinics
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New York
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Bronx, New York, United States, 10467
- The Childrens Hospital at Montefiore Albert Einstein School of Medicine
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New York, New York, United States, 10032
- Columbia University Medical Center
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New York, New York, United States, 10029
- Icahn School of Medicine at Mount Sinai
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Oregon
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Portland, Oregon, United States, 97239
- Oregon Health Science University School of Medicine
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Pennsylvania
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Pittsburgh, Pennsylvania, United States, 15224
- UPMC Children's Hospital of Pittsburgh
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Child
- Adult
- Older Adult
Accepts Healthy Volunteers
No
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: Triple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Odevixibat (A4250)
Capsules for oral administration once daily for 24 weeks.
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Odevixibat is a small molecule and selective inhibitor of IBAT.
Other Names:
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Placebo Comparator: Placebo
Capsules for oral administration (to match active) once daily for 24 weeks.
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Placebo identical in appearance to experimental drug (odevixibat).
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Change From Baseline in Scratching Score
Time Frame: 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.
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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.
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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.
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Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
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Serum Bile Acid Levels
Time Frame: 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.
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Change in serum bile acid levels (μmol/L) from baseline to average of week 20 and 24
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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.
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
March 19, 2021
Primary Completion (Actual)
September 9, 2022
Study Completion (Actual)
September 9, 2022
Study Registration Dates
First Submitted
December 10, 2020
First Submitted That Met QC Criteria
December 17, 2020
First Posted (Actual)
December 19, 2020
Study Record Updates
Last Update Posted (Actual)
November 2, 2023
Last Update Submitted That Met QC Criteria
October 10, 2023
Last Verified
October 1, 2023
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
- Digestive System Diseases
- Pathologic Processes
- Cardiovascular Diseases
- Disease
- Congenital Abnormalities
- Liver Diseases
- Genetic Diseases, Inborn
- Biliary Tract Diseases
- Heart Defects, Congenital
- Cardiovascular Abnormalities
- Abnormalities, Multiple
- Bile Duct Diseases
- Cholestasis, Intrahepatic
- Cholestasis
- Syndrome
- Alagille Syndrome
Other Study ID Numbers
- A4250-012
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Alagille Syndrome
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TakedaActive, not recruiting
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AlbireoActive, not recruitingAlagille SyndromeUnited Kingdom, United States, France, Germany, Netherlands, Italy, Belgium, Malaysia, Poland, Turkey
-
Mirum Pharmaceuticals, Inc.Completed
-
Mirum Pharmaceuticals, Inc.Lumena Pharmaceuticals, Inc.; Childhood Liver Disease Research and Education...Completed
-
Mirum Pharmaceuticals, Inc.CompletedAlagille SyndromeUnited Kingdom
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Mirum Pharmaceuticals, Inc.RecruitingAlagille SyndromeUnited States
-
National Human Genome Research Institute (NHGRI)Completed
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Mirum Pharmaceuticals, Inc.Childhood Liver Disease Research and Education NetworkCompletedAlagille SyndromeUnited States, Canada
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Mirum Pharmaceuticals, Inc.Clinigen, Inc.Approved for marketingAlagille SyndromeUnited States
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Children's Hospital of Fudan UniversityRecruitingAlagille Syndrome | Atorvastatin | XanthomaChina
Clinical Trials on Odevixibat
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AlbireoApproved for marketingProgressive Familial Intrahepatic CholestasisUnited States
-
AlbireoActive, not recruitingAlagille SyndromeUnited Kingdom, United States, France, Germany, Netherlands, Italy, Belgium, Malaysia, Poland, Turkey
-
AlbireoRecruitingBiliary AtresiaNetherlands, United States, Spain, Italy, Taiwan, Korea, Republic of, France, Germany, United Kingdom, Canada, Belgium, China, Australia, Poland, Turkey, Malaysia, New Zealand, Hungary, Israel
-
AlbireoActive, not recruitingProgressive Familial Intrahepatic CholestasisUnited States, Spain, Netherlands, United Kingdom, Turkey, Canada, Italy, Germany, France, Israel, Australia, Belgium, Poland, Saudi Arabia, Sweden
-
AlbireoCompletedPFIC1 | PFIC2United States, Spain, Netherlands, United Kingdom, Turkey, Canada, Belgium, Italy, Germany, Saudi Arabia, France, Israel, Australia, Sweden, Poland
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AlbireoEnrolling by invitationBiliary AtresiaSpain, United States, Netherlands, Malaysia, Canada, Germany, Turkey, Italy, France, Australia, Poland, New Zealand
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Shanghai Ark Biopharmaceutical Co., Ltd.Not yet recruiting