Alagille Syndrome
Conditions
Brief summary
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.
Interventions
None listed
Sponsors
Ipsen
Study design
Observational model
OTHER
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Diagnosed with ALGS. * On (or starting) active odevixibat treatment. * Signed informed consent and assent, as appropriate. Consent/assent from the participant or legal representative should be obtained, as appropriate, before any study data collection is conducted. Participants who turn 18 years of age (or legal age per country) while participating in the study will be required to provide consent for themselves.
Exclusion criteria
* Currently participating in a clinical trial with odevixibat. * Currently participating in any interventional clinical trial for ALGS. * Have any contraindication to odevixibat as per the locally approved label. * Had liver transplant before enrolment
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of participants with Alagille syndrome (ALGS) who are treated with odevixibat and undergo biliary diversion surgery or liver transplantation. | From first dose to end of study (approximately 5 years data collection) | — |
| Surgical biliary diversion-free survival | From first dose to end of study (approximately 5 years data collection) | Defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion or death. |
| Liver transplant-free survival | From first dose to end of study (approximately 5 years data collection) | Defined as time from the start of odevixibat treatment to the first occurrence of liver transplant or death. |
| Overall survival | From first dose to end of study (approximately 5 years data collection) | Defined as time from the start of odevixibat treatment to death. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change from Baseline in Weight | From first dose to end of study (approximately 5 years data collection) | 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. |
| Change from Baseline in Height | From first dose to end of study (approximately 5 years data collection) | 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. |
| Percentage of participants with Adverse Events (AEs) associated with fat-soluble vitamin (FSV) deficiencies and their possible sequelae. | From signing of the Informed Consent Form (ICF) to 180 days after the last dose intake 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. |
| Percentage of participants with suspected hepatotoxic Adverse events (AEs) requiring interruption of odevixibat | From signing of the ICF to 180 days after the last dose intake 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. |
| Percentage of participants with bleeding AEs | From signing of the ICF to 180 days after the last dose intake 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. |
| Percentage of participants with AEs | From signing of the ICF to 180 days after the last dose intake 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. |
Countries
United States
Contacts
CONTACTIpsen Recruitment Enquiries
STUDY_DIRECTORIpsen Medical, Director
Ipsen
Outcome results
None listed