Back to resultsEfficacy and Safety of Odevixibat in Patients With Alagille Syndrome (ASSERT)
Primary Purpose
Alagille Syndrome
Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
Odevixibat
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Alagille Syndrome focused on measuring ALGS, Alagille syndrome
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
Sites / Locations
- University of California - San Francisco
- Johns Hopkins Hospital
- Boston Children's Hospital
- Children's Mercy Hospital and Clinics
- The Childrens Hospital at Montefiore Albert Einstein School of Medicine
- Icahn School of Medicine at Mount Sinai
- Columbia University Medical Center
- Oregon Health Science University School of Medicine
- UPMC Children's Hospital of Pittsburgh
- Cliniques Universitaires Saint-Luc Bruxelles
- CHU Sainte-Justine
- Hôpital Femme Mère Enfant de Lyon
- Antenne pédiatrique du CIC - Hopital Jeanne De Flandre
- APHM
- Hopital Necker Enfants Malades
- Charité-Universitätsmedizin Berlin
- Medizinische Hochschule Hannover
- Universitätsklinik für Kinder-und Jugendmedizin Tübingen
- Sharie Zedek
- Schneider Children's Medical Center of Israel
- ASST Papa Giovanni XXIII
- AOU Meyer
- Azienda Ospedale Università Padova
- Bambino Gesù Children's Hospital
- University of Malaya Medical Centre
- Universitair Medisch Centrum Groningen (UMCG)
- University Medical Centre Utrecht, WKZ
- Starship Child Health
- Instytut Pomnik-Centrum Zdrowia Dziecka
- Hacettepe Üniversitesi İhsan Doğramacı Çocuk Hastanesi
- Istanbul University Medical Faculty Hospital
- King's College Hospital
Arms of the Study
Arm 1
Arm 2
Arm Type
Experimental
Placebo Comparator
Arm Label
Odevixibat (A4250)
Placebo
Arm Description
Capsules for oral administration once daily for 24 weeks.
Capsules for oral administration (to match active) once daily for 24 weeks.
Outcomes
Primary Outcome Measures
Change from Baseline in Scratching Score
Change from baseline in scratching score to Month 6 as measured by the Albireo Observer-Reported Outcome scratching score
Secondary Outcome Measures
Serum Bile Acid Levels
Change in serum bile acid levels from baseline to Week 24
Safety and Tolerability
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT04674761
Brief Title
Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome
Acronym
ASSERT
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
2. Study Status
Record Verification Date
April 2023
Overall Recruitment Status
Completed
Study Start Date
March 19, 2021 (Actual)
Primary Completion Date
September 9, 2022 (Actual)
Study Completion Date
September 9, 2022 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Albireo
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Alagille Syndrome
Keywords
ALGS, Alagille syndrome
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
52 (Actual)
8. Arms, Groups, and Interventions
Arm Title
Odevixibat (A4250)
Arm Type
Experimental
Arm Description
Capsules for oral administration once daily for 24 weeks.
Arm Title
Placebo
Arm Type
Placebo Comparator
Arm Description
Capsules for oral administration (to match active) once daily for 24 weeks.
Intervention Type
Drug
Intervention Name(s)
Odevixibat
Other Intervention Name(s)
A4250
Intervention Description
Odevixibat is a small molecule and selective inhibitor of IBAT.
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo identical in appearance to experimental drug (odevixibat).
Primary Outcome Measure Information:
Title
Change from Baseline in Scratching Score
Description
Change from baseline in scratching score to Month 6 as measured by the Albireo Observer-Reported Outcome scratching score
Time Frame
From baseline to Week 24
Secondary Outcome Measure Information:
Title
Serum Bile Acid Levels
Description
Change in serum bile acid levels from baseline to Week 24
Time Frame
From baseline to Week 24
Title
Safety and Tolerability
Description
Safety and Tolerability as Assessed by Number of Participants With Treatment-Emergent Adverse Events (AEs) and Serious Adverse Events (SAEs)
Time Frame
From first dose of study drug up to 28 days after last dose of study drug or to the completion of study participation date, whichever occurs first
10. Eligibility
Sex
All
Accepts Healthy Volunteers
No
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
Facility Information:
Facility Name
University of California - San Francisco
City
San Francisco
State/Province
California
ZIP/Postal Code
94158
Country
United States
Facility Name
Johns Hopkins Hospital
City
Baltimore
State/Province
Maryland
ZIP/Postal Code
21287
Country
United States
Facility Name
Boston Children's Hospital
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02115
Country
United States
Facility Name
Children's Mercy Hospital and Clinics
City
Kansas City
State/Province
Missouri
ZIP/Postal Code
64108
Country
United States
Facility Name
The Childrens Hospital at Montefiore Albert Einstein School of Medicine
City
Bronx
State/Province
New York
ZIP/Postal Code
10467
Country
United States
Facility Name
Icahn School of Medicine at Mount Sinai
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Columbia University Medical Center
City
New York
State/Province
New York
ZIP/Postal Code
10032
Country
United States
Facility Name
Oregon Health Science University School of Medicine
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
UPMC Children's Hospital of Pittsburgh
City
Pittsburgh
State/Province
Pennsylvania
ZIP/Postal Code
15224
Country
United States
Facility Name
Cliniques Universitaires Saint-Luc Bruxelles
City
Brussels
ZIP/Postal Code
1200
Country
Belgium
Facility Name
CHU Sainte-Justine
City
Montréal
State/Province
Quebec
ZIP/Postal Code
H3T 1C5
Country
Canada
Facility Name
Hôpital Femme Mère Enfant de Lyon
City
Bron
ZIP/Postal Code
69677
Country
France
Facility Name
Antenne pédiatrique du CIC - Hopital Jeanne De Flandre
City
Lille
ZIP/Postal Code
59037
Country
France
Facility Name
APHM
City
Marseille
ZIP/Postal Code
13385
Country
France
Facility Name
Hopital Necker Enfants Malades
City
Paris
ZIP/Postal Code
75015
Country
France
Facility Name
Charité-Universitätsmedizin Berlin
City
Berlin
ZIP/Postal Code
13353
Country
Germany
Facility Name
Medizinische Hochschule Hannover
City
Hannover
ZIP/Postal Code
30625
Country
Germany
Facility Name
Universitätsklinik für Kinder-und Jugendmedizin Tübingen
City
Tübingen
ZIP/Postal Code
72076
Country
Germany
Facility Name
Sharie Zedek
City
Jerusalem
ZIP/Postal Code
9103102
Country
Israel
Facility Name
Schneider Children's Medical Center of Israel
City
Petah tikva
ZIP/Postal Code
4920235
Country
Israel
Facility Name
ASST Papa Giovanni XXIII
City
Bergamo
ZIP/Postal Code
24127
Country
Italy
Facility Name
AOU Meyer
City
Firenze
ZIP/Postal Code
50139
Country
Italy
Facility Name
Azienda Ospedale Università Padova
City
Padova
ZIP/Postal Code
35128
Country
Italy
Facility Name
Bambino Gesù Children's Hospital
City
Rome
ZIP/Postal Code
00165
Country
Italy
Facility Name
University of Malaya Medical Centre
City
Kuala Lumpur
ZIP/Postal Code
59100
Country
Malaysia
Facility Name
Universitair Medisch Centrum Groningen (UMCG)
City
Groningen
ZIP/Postal Code
9713 GZ
Country
Netherlands
Facility Name
University Medical Centre Utrecht, WKZ
City
Utrecht
ZIP/Postal Code
3584EA
Country
Netherlands
Facility Name
Starship Child Health
City
Auckland
ZIP/Postal Code
1023
Country
New Zealand
Facility Name
Instytut Pomnik-Centrum Zdrowia Dziecka
City
Warszawa
ZIP/Postal Code
04-730
Country
Poland
Facility Name
Hacettepe Üniversitesi İhsan Doğramacı Çocuk Hastanesi
City
Ankara
ZIP/Postal Code
06230
Country
Turkey
Facility Name
Istanbul University Medical Faculty Hospital
City
Fatih
ZIP/Postal Code
34093
Country
Turkey
Facility Name
King's College Hospital
City
London
ZIP/Postal Code
SE5 9RS
Country
United Kingdom
12. IPD Sharing Statement
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Efficacy and Safety of Odevixibat in Patients With Alagille Syndrome
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