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.
Brief Title
Efficacy and Safety of Odevixibat 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.
Categories
Completion Date
Completion Date Type
Actual
Conditions
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
* 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
Inclusion Criteria
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
* 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
Gender
All
Gender Based
false
Keywords
ALGS
Alagille syndrome
Healthy Volunteers
No
Last Update Post Date
Last Update Post Date Type
Actual
Last Update Submit Date
NCT Id
NCT04674761
Org Class
Industry
Org Full Name
Ipsen
Org Study Id
A4250-012
Overall Status
Completed
Phases
Phase 3
Primary Completion Date
Primary Completion Date Type
Actual
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)
Primary Outcomes
Outcome Description
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.
Outcome Measure
Change From Baseline in Scratching Score
Outcome 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.
Secondary Outcomes
Outcome Description
Change in serum bile acid levels (μmol/L) from baseline to average of week 20 and 24
Outcome 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.
Outcome Measure
Serum Bile Acid Levels
Start Date
Start Date Type
Actual
Status Verified Date
First Post Date
First Post Date Type
Actual
First Submit Date
First Submit QC Date
Std Ages
Child
Adult
Older Adult
Maximum Age Number (converted to Years and rounded down)
999
Minimum Age Number (converted to Years and rounded down)
0
Investigators
Investigator Type
Principal Investigator
Investigator Name
Debra Pan
Investigator Email
dpan@montefiore.org
Investigator Phone
718-741-2332
Categories Mesh Debug
Digestive System --- CHOLESTASIS, INTRAHEPATIC
Liver --- CHOLESTASIS, INTRAHEPATIC
Digestive System --- CHOLESTASIS
Liver --- CHOLESTASIS
Digestive System --- BILE DUCT DISEASES
Liver --- BILE DUCT DISEASES
Digestive System --- BILIARY TRACT DISEASES
Liver --- BILIARY TRACT DISEASES
Gastrointestinal (GI) Cancers --- DIGESTIVE SYSTEM DISEASES
Digestive System --- DIGESTIVE SYSTEM DISEASES
Liver --- DIGESTIVE SYSTEM DISEASES
Digestive System --- LIVER DISEASES
Liver --- LIVER DISEASES
Child Development & Autism --- HEART DEFECTS, CONGENITAL
Child Development & Autism --- CARDIOVASCULAR ABNORMALITIES
Blood Disorders --- CARDIOVASCULAR DISEASES
Blood & Bone Marrow Cancers --- CARDIOVASCULAR DISEASES
Heart/Cardiovascular --- CARDIOVASCULAR DISEASES
Blood & Bone Marrow Cancers --- CONGENITAL ABNORMALITIES
MeSH Terms
ALAGILLE SYNDROME
CHOLESTASIS, INTRAHEPATIC
CHOLESTASIS
BILE DUCT DISEASES
BILIARY TRACT DISEASES
DIGESTIVE SYSTEM DISEASES
LIVER DISEASES
HEART DEFECTS, CONGENITAL
CARDIOVASCULAR ABNORMALITIES
CARDIOVASCULAR DISEASES
ABNORMALITIES, MULTIPLE
CONGENITAL ABNORMALITIES
CONGENITAL, HEREDITARY, AND NEONATAL DISEASES AND ABNORMALITIES
GENETIC DISEASES, INBORN
ODEVIXIBAT
COUNTERFEIT DRUGS
SUBSTANDARD DRUGS
PHARMACEUTICAL PREPARATIONS