This study will collect information from patients with Alagille syndrome (ALGS) as they use odevixibat (Bylvay) in their daily lives. Odevixibat is a medicine that helps patients with ALGS, a rare disease that harms their liver and causes itching. The main aim of this study is to observe the long-term, everyday effectiveness and safety of the drug odevixibat in patients with ALGS who are receiving ongoing treatment.
Study Type
OBSERVATIONAL
Enrollment
30
NYU Langone - NYU Grossman School of Medicine
New York, New York, United States
RECRUITINGLe Bonheur Children's Hospital
Memphis, Tennessee, United States
RECRUITINGProactive El Paso,LLC
El Paso, Texas, United States
RECRUITINGPercentage of participants with Alagille syndrome (ALGS) who are treated with odevixibat and undergo biliary diversion surgery or liver transplantation.
Time frame: From first dose to end of study (approximately 5 years data collection)
Surgical biliary diversion-free survival
Defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion or death.
Time frame: From first dose to end of study (approximately 5 years data collection)
Liver transplant-free survival
Defined as time from the start of odevixibat treatment to the first occurrence of liver transplant or death.
Time frame: From first dose to end of study (approximately 5 years data collection)
Overall survival
Defined as time from the start of odevixibat treatment to death.
Time frame: From first dose to end of study (approximately 5 years data collection)
Change from Baseline in Weight
This endpoint will measure the change in participants' weight from baseline, reported as standard-deviation scores (z-scores) to account for age and sex-specific growth variations.
Time frame: From first dose to end of study (approximately 5 years data collection)
Change from Baseline in Height
This endpoint will measure the change in participants' height from baseline, reported as standard-deviation scores (z-scores) to account for age and sex-specific growth variations.
Time frame: From first dose to end of study (approximately 5 years data collection)
Percentage of participants with Adverse Events (AEs) associated with fat-soluble vitamin (FSV) deficiencies and their possible sequelae.
An Adverse event (AE) is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.
Time frame: From signing of the Informed Consent Form (ICF) to 180 days after the last dose intake of odevixibat
Percentage of participants with suspected hepatotoxic Adverse events (AEs) requiring interruption of odevixibat
An Adverse event (AE) is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.
Time frame: From signing of the ICF to 180 days after the last dose intake of odevixibat
Percentage of participants with bleeding AEs
An Adverse event (AE) is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.
Time frame: From signing of the ICF to 180 days after the last dose intake of odevixibat
Percentage of participants with AEs
An Adverse event (AE) is any untoward medical occurrence in a clinical study participant, temporally associated with the use of study intervention, whether or not considered related to the study intervention.
Time frame: From signing of the ICF to 180 days after the last dose intake of odevixibat
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