Alagille Syndrome (ALGS), Progressive Familial Intrahepatic Cholestasis (PFIC)
Conditions
Keywords
Alagille Syndrome (ALGS), Progressive Familial Intrahepatic Cholestasis (PFIC)
Brief summary
This study is a database study in Japan for maralixibat (TAK-625) used to treat participants with Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC). The main aim of the study is to evaluate the risk of liver disorder during the use of maralixibat in Japanese patients with ALGS or PFIC. This database study will conduct in use of medical database called Comprehensive and Informative Registry system for Childhood Liver Disease (CIRCLe).
Interventions
DRUGMaralixibat
Maralixibat Oral Solution 10 milligrams (mg)/milliliters (mL)
Sponsors
Takeda
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
ALL
Healthy volunteers
No
Inclusion criteria
* Has a diagnosis of ALGS or PFIC within the enrollment period. * There is a prescription for Livmarli during the enrollment period (Index date: the date of the first prescription within the enrollment period).
Exclusion criteria
\- None
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants with ALGS who Experience Liver Disorder Evaluated with Outcome Definition 1 (Side Effects) | Up to 6 years as data period | Percentage of participants with ALGS who will experience liver disorder will be reported. Outcome Definition 1 (side effects) will be used to estimate this outcome measure. Outcome Definition 1 (side effects) will define as follows: For the adverse event entry form, if all of the following conditions are met, it is defined as "liver disorder". (1) "Maralixibat (Livmarli)" has been selected as the drug to be administered. (2) "Yes" is selected for the presence or absence of adverse events due to the administered drug. (3) "Liver impairment and liver failure" are selected as symptoms/findings due to the administered drugs. (4) Date of occurrence or discovery of adverse event within the follow-up period. (5) "Unlikely" or "Not related" was not selected for the association between this adverse event and the treatment. |
| Percentage of Participants with PFIC who Experience Liver Disorder Evaluated with Outcome Definition 1 (Side Effects) | Up to 6 years as data period | Percentage of participants with PFIC who will experience liver disorder will be reported. Outcome Definition 1 (side effects) will be used to estimate this outcome measure. Outcome Definition 1 (side effects) will define as follows: For the adverse event entry form, if all of the following conditions are met, it is defined as "liver disorder". (1) "Maralixibat (Livmarli)" has been selected as the drug to be administered. (2) "Yes" is selected for the presence or absence of adverse events due to the administered drug. (3) "Liver impairment and liver failure" are selected as symptoms/findings due to the administered drugs. (4) Date of occurrence or discovery of adverse event within the follow-up period. (5) "Unlikely" or "Not related" was not selected for the association between this adverse event and the treatment. |
| Time to Onset of Liver Disorder in Participants with ALGS Evaluated with Outcome Definition 1 (Side Effects) | Up to 6 years as data period | Time to onset of liver disorder in participants with ALGS will be reported. Outcome Definition 1 (side effects) will be used to estimate this outcome measure. Outcome Definition 1 (side effects) will define as follows: For the adverse event entry form, if all of the following conditions are met, it is defined as "liver disorder". (1) "Maralixibat (Livmarli)" has been selected as the drug to be administered. (2) "Yes" is selected for the presence or absence of adverse events due to the administered drug. (3) "Liver impairment and liver failure" are selected as symptoms/findings due to the administered drugs. (4) Date of occurrence or discovery of adverse event within the follow-up period. (5) "Unlikely" or "Not related" was not selected for the association between this adverse event and the treatment. |
| Time to Onset of Liver Disorder in Participants with PFIC Evaluated with Outcome Definition 1 (Side Effects) | Up to 6 years as data period | Time to onset of liver disorder in participants with PFIC will be reported. Outcome Definition 1 (side effects) will be used to estimate this outcome measure. Outcome Definition 1 (side effects) will define as follows: For the adverse event entry form, if all of the following conditions are met, it is defined as "liver disorder". (1) "Maralixibat (Livmarli)" has been selected as the drug to be administered. (2) "Yes" is selected for the presence or absence of adverse events due to the administered drug. (3) "Liver impairment and liver failure" are selected as symptoms/findings due to the administered drugs. (4) Date of occurrence or discovery of adverse event within the follow-up period. (5) "Unlikely" or "Not related" was not selected for the association between this adverse event and the treatment. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Percentage of Participants with ALGS who Experience Liver Disorder Evaluated with Outcome Definition 2 (Adverse Events) | Up to 6 years as data period | Percentage of participants with ALGS who will experience liver disorder will be reported. Outcome Definition 2 (Adverse Events) will be used to estimate this outcome measure. Outcome Definition 2 (Adverse Events) will define as follows: For the adverse event entry form, if all of the following conditions are met, it is defined as "liver disorder". (1) "Maralixibat (Livmarli)" has been selected as the drug to be administered. (2) "Yes" is selected for the presence or absence of adverse events due to the administered drug. (3) "Liver impairment and liver failure" are selected as symptoms/findings due to the administered drugs. (4) Date of occurrence or discovery of adverse event within the follow-up period. |
| Percentage of Participants with PFIC who Experience Liver Disorder Evaluated with Outcome Definition 2 (Adverse Events) | Up to 6 years as data period | Percentage of participants with PFIC who will experience liver disorder will be reported. Outcome Definition 2 (Adverse Events) will be used to estimate this outcome measure. Outcome Definition 2 (Adverse Events) will define as follows: For the adverse event entry form, if all of the following conditions are met, it is defined as "liver disorder". (1) "Maralixibat (Livmarli)" has been selected as the drug to be administered. (2) "Yes" is selected for the presence or absence of adverse events due to the administered drug. (3) "Liver impairment and liver failure" are selected as symptoms/findings due to the administered drugs. (4) Date of occurrence or discovery of adverse event within the follow-up period. |
| Time to Onset of Liver Disorder in Participants with ALGS Evaluated with Outcome Definition 2 (Adverse Events) | Up to 6 years as data period | Time to onset of liver disorder in participants with ALGS will be reported. Outcome Definition 2 (adverse events) will be used to estimate this outcome measure. Outcome Definition 2 (adverse events) will define as follows: For the adverse event entry form, if all of the following conditions are met, it is defined as "liver disorder". (1) "Maralixibat (Livmarli)" has been selected as the drug to be administered. (2) "Yes" is selected for the presence or absence of adverse events due to the administered drug. (3) "Liver impairment and liver failure" are selected as symptoms/findings due to the administered drugs. (4) Date of occurrence or discovery of adverse event within the follow-up period. |
| Time to Onset of Liver Disorder in Participants with PFIC Evaluated with Outcome Definition 2 (Adverse Events) | Up to 6 years as data period | Time to onset of liver disorder in participants with PFIC will be reported. Outcome Definition 2 (adverse events) will be used to estimate this outcome measure. Outcome Definition 2 (adverse events) will define as follows: For the adverse event entry form, if all of the following conditions are met, it is defined as "liver disorder". (1) "Maralixibat (Livmarli)" has been selected as the drug to be administered. (2) "Yes" is selected for the presence or absence of adverse events due to the administered drug. (3) "Liver impairment and liver failure" are selected as symptoms/findings due to the administered drugs. (4) Date of occurrence or discovery of adverse event within the follow-up period. |
Countries
Japan
Contacts
CONTACTTakeda Contact
STUDY_DIRECTORStudy Director
Takeda
Outcome results
None listed