Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Measure | Description | Time Frame |
|---|---|---|
| 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 | Defined as time from the start of odevixibat treatment to the first occurrence of surgical biliary diversion or death. | 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. | 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. | From first dose to end of study (approximately 5 years data collection) |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | From first dose to end of study (approximately 5 years data collection) |
| Change from Baseline in Height |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
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.
| Name | Role | Phone | Extension | |
|---|---|---|---|---|
| Ipsen Recruitment Enquiries | Contact | See email | clinical.trials@ipsen.com |
| Name | Affiliation | Role |
|---|---|---|
| Ipsen Medical, Director | Ipsen | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Children's Hospital Los Angeles | Recruiting | Los Angeles | California | 90027 | United States | |
Qualified researchers may request access to patient level data and related study documents including the clinical study report, study protocol with any amendments, annotated case report form, statistical analysis plan, and dataset specifications.
Patient level data will be anonymized and study documents will be redacted to protect the privacy of study participants.
Not provided
Where applicable, data from eligible studies are available 6 months after the studied medicine and indication have been approved in the US and/or EU.
Further details on Ipsen's sharing criteria and process for sharing are available here (https://www.ipsen.com/science/clinical-trials/clinical-data-transparency/).
Not provided
Not provided
Not provided
Not provided
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. |
| 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. | 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. | 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. | 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. | From signing of the ICF to 180 days after the last dose intake of odevixibat |
| UCSF Benioff Children's Hospital |
| Recruiting |
| San Francisco |
| California |
| 94158 |
| United States |
| The Children's Mercy Hospital | Recruiting | Kansas City | Missouri | 64108 | United States |
| SSM Health Cardinal Glennon Children's Hospital | Not yet recruiting | St Louis | Missouri | 63104 | United States |
| NYU Langone - NYU Grossman School of Medicine | Recruiting | New York | New York | 10016 | United States |
| Columbia University Medical Center - Morgan Stanley Children's Hospital of NewYork - Presbyterian (MS-CHONY) | Recruiting | New York | New York | 10032 | United States |
| Le Bonheur Children's Hospital | Recruiting | Memphis | Tennessee | 38105 | United States |
| Children's Medical Center Of Dallas | Recruiting | Dallas | Texas | 75246 | United States |
| Proactive El Paso,LLC | Recruiting | El Paso | Texas | 79902 | United States |
| Primary Children's Hospital (PCH) - Intermountain Healthcare/University of Utah | Not yet recruiting | Salt Lake City | Utah | 84113 | United States |
| ID | Term |
|---|---|
| D016738 | Alagille Syndrome |
| ID | Term |
|---|---|
| D002780 | Cholestasis, Intrahepatic |
| D002779 | Cholestasis |
| D001649 | Bile Duct Diseases |
| D001660 | Biliary Tract Diseases |
| D004066 | Digestive System Diseases |
| D008107 | Liver Diseases |
| D006330 | Heart Defects, Congenital |
| D018376 | Cardiovascular Abnormalities |
| D002318 | Cardiovascular Diseases |
| D000015 | Abnormalities, Multiple |
| D000013 | Congenital Abnormalities |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D030342 | Genetic Diseases, Inborn |
Not provided
Not provided