Prospective Registry-Based Study Evaluating the Effectiveness and Safety of Odevixibat in Participants With Alagille Syndrome (ALGS)

Study type

• Study topic:
  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Effectiveness study (incl. comparative)

  Safety study (incl. comparative)
• Data collection methods:
  Combined primary data collection and secondary use of data

Non-interventional study

• Non-interventional study design:
  Case-only

Clinical trials

• Clinical trial regulatory scope:
  Clinical trial not part of marketing authorisation application or subject to marketing authorisation approval

  Post-authorisation interventional clinical trial

  Pre-authorisation clinical trial

Study drug and medical condition

• Name of medicine:
  BYLVAY
• Study drug International non-proprietary name (INN) or common name:
  ODEVIXIBAT
• Anatomical Therapeutic Chemical (ATC) code:
  (A05AX05) odevixibat

  odevixibat
• Medical condition to be studied:
  Alagille syndrome

Population studied

• Short description of the study population:
  Patients diagnosed with ALGS who start treatment with odevixibat (Bylvay) will be enrolled into the registry. Patients who started odevixibat treatment before the implementation of the registry study may also be enrolled.
• Age groups:
  All
• Estimated number of subjects:
  30

Study design details

• Study design:
  This study will collect information from patients with Alagille syndrome (ALGS) as they use odevixibat (Bylvay) in their daily lives.
• Main study objective:
  To evaluate the incidence of biliary diversion surgery, liver transplantation, all-cause mortality in participants with ALGS chronically treated with odevixibat.
• Outcomes:
  Primary Outcome Measure:; 1. Percentage of participants with Alagille syndrome (ALGS) who are chronically 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)]; 2. 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)]; 3. 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)]; 4. 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)]; Secondary Outcome Measure:; 5. Change from baseline in Body Mass Index (BMI); [Time Frame: From first dose to end of study (approximately 5 years data collection)]; 6. Percentage of participants with Adverse events (AEs) associated with fat-soluble vitamin (FSV) deficiencies and their possible sequelae.; [Time Frame: From signing of the ICF to the last dose of odevixibat + 180 days]; 7. Percentage of participants with suspected hepatotoxic Adverse events (AEs) requiring interruption of odevixibat treatment; [Time Frame: From signing of the ICF to the last dose of odevixibat + 180 days]; 8. Percentage of participants with bleeding AEs; [Time Frame: From signing of the ICF to the last dose of odevixibat + 180 days]; 9. Percentage of participants with AEs; [Time Frame: From signing of the ICF to the last dose of odevixibat + 180 days]
• Data analysis plan:
  No formal sample size calculations have been performed for this registry-based study. Enrolment will be based on the number of participants prescribed odevixibat and their willingness to participate in the study, but the goal will be to enroll approximately 30 to 45 participants with ALGS.