Ignite Creation Date:
2024-10-26 @ 3:39 PM
Last Modification Date:
2024-10-26 @ 3:39 PM
Study NCT ID:
NCT06581146
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-08-19
Brief Title:
A Study to Check Liver Health in Boys With XLMTM a Serious Genetic Muscle Condition
Sponsor:
None
Organization:
None
Study Overview
Official Title:
A Non-interventional Epidemiologic Study of XLMTM and Clinical Expression in the Liver
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-10
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
EXCEL
Brief Summary:
XLMTM X-linked myotubular myopathy is a serious genetic muscle condition It is caused by changes in the MTM1 gene which stops or slows down normal muscle development causing severe muscle weakness There is currently no cure for XLMTM Ongoing care is needed to manage symptoms and prevent further medical problems from this condition
Recent research shows that individuals with XLMTM often have reduced bile flow which can affect liver and gallbladder health Bile is a liquid made in the liver that helps digest fat Ongoing liver health checks may help with the routine care of people with XLMTM
There is a need to understand liver problems that develop in individuals with XLMTM over time The main aim of the study is to learn how many boys with XLMTM have new cases of liver problems during the study
This study is about collecting information only This is known as an observational study The individuals doctor decides on treatment not the study sponsor Astellas
In this study boys under 18 diagnosed with XLMTM will be followed for about 1 year The health of their liver and gallbladder will be checked about every 6 weeks This can be done at home if preferred A scan called a Fibroscan also known as transient elastography will check for signs of scarring in the liver fibrosis and the build-up of lipids It is suggested that each boy will have a Fibroscan when they start the study and another scan when they complete the study
This study will help understand liver gallbladder and bile duct issues in individuals with XLMTM over time The goal is to improve their care and provide information to use in future clinical studies
Recent research shows that individuals with XLMTM often have reduced bile flow which can affect liver and gallbladder health Bile is a liquid made in the liver that helps digest fat Ongoing liver health checks may help with the routine care of people with XLMTM
There is a need to understand liver problems that develop in individuals with XLMTM over time The main aim of the study is to learn how many boys with XLMTM have new cases of liver problems during the study
This study is about collecting information only This is known as an observational study The individuals doctor decides on treatment not the study sponsor Astellas
In this study boys under 18 diagnosed with XLMTM will be followed for about 1 year The health of their liver and gallbladder will be checked about every 6 weeks This can be done at home if preferred A scan called a Fibroscan also known as transient elastography will check for signs of scarring in the liver fibrosis and the build-up of lipids It is suggested that each boy will have a Fibroscan when they start the study and another scan when they complete the study
This study will help understand liver gallbladder and bile duct issues in individuals with XLMTM over time The goal is to improve their care and provide information to use in future clinical studies
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None