Ignite Creation Date:
2024-05-06 @ 1:22 PM
Last Modification Date:
2024-10-26 @ 1:13 PM
Study NCT ID:
NCT04001595
Status:
RECRUITING
Last Update Posted:
2024-01-30
First Post:
2019-06-25
Brief Title:
Global FKRP Registry
Sponsor:
Newcastle University
Organization:
Newcastle University
Study Overview
Official Title:
Global Fukutin-Related Protein Registry
Status:
RECRUITING
Status Verified Date:
2024-01
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
Mutations in the Fukutin Related Protein FKRP gene cause the condition Limb Girdle Muscular Dystrophy type R9 LGMDR9 also known as LGMD2I and the rarer conditions Congenital Muscular Dystrophy MDC1C Muscle Eye Brain Disease MEB and Walker-Warburg Syndrome WWS LGMDR9 is the most common FKRP-related condition and is especially prevalent in Northern Europe
The aim is to facilitate a questionnaire based research study in order to better characterise and understand the disease globally By maintaining a global registry this will help identify potential participants eligible for clinical trials in the future
The aim is to facilitate a questionnaire based research study in order to better characterise and understand the disease globally By maintaining a global registry this will help identify potential participants eligible for clinical trials in the future
Detailed Description:
The Global FKRP Registry httpswwwfkrp-registryorg is an international registry for patients with an FKRP-related condition no experimental intervention is involved Patients will receive information on the most up to date standards of care relating to their disease and may be invited to participate in relevant clinical trials Their data will be updated annually and stored indefinitely or until they request their data to be removed
The data will be collected via an online form and will be stored on a secure server based in the United Kingdom and looked after by the registry staff at Newcastle University Data collected from patients will include demographic information diagnosis current condition age of onset medication contractures family history and results of genetic testing if available Other optional questionnaires will focus on patients pain and quality of life Further information collected from patients doctors will include heart and lung function muscle strength muscle and brain MRI findings and genetics
The FKRP registry is funded by LGMD2i Research Fund and CureLGMD2i
The primary objectives of the Global FKRP Registry are to
Accelerate and facilitate clinical trials by locating potential research subjects quickly and efficiently
Facilitate in the planning of clinical trials
Assist the neuromuscular community with the development of recommendations and standards of care
Characterise and describe the FKRP population as a whole enhancing the understanding of the prevalence throughout the world
The data will be collected via an online form and will be stored on a secure server based in the United Kingdom and looked after by the registry staff at Newcastle University Data collected from patients will include demographic information diagnosis current condition age of onset medication contractures family history and results of genetic testing if available Other optional questionnaires will focus on patients pain and quality of life Further information collected from patients doctors will include heart and lung function muscle strength muscle and brain MRI findings and genetics
The FKRP registry is funded by LGMD2i Research Fund and CureLGMD2i
The primary objectives of the Global FKRP Registry are to
Accelerate and facilitate clinical trials by locating potential research subjects quickly and efficiently
Facilitate in the planning of clinical trials
Assist the neuromuscular community with the development of recommendations and standards of care
Characterise and describe the FKRP population as a whole enhancing the understanding of the prevalence throughout the world
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None