Ignite Creation Date:
2024-05-06 @ 7:47 PM
Last Modification Date:
2024-10-26 @ 3:14 PM
Study NCT ID:
NCT06136416
Status:
COMPLETED
Last Update Posted:
2024-02-09
First Post:
2023-11-13
Brief Title:
Pilot Study for the Development of an Activity and Quality of Life Questionnaire for the Follow-up of Patients With Non-dystrophic Myotonia
Sponsor:
Centre Hospitalier Universitaire de Nice
Organization:
Centre Hospitalier Universitaire de Nice
Study Overview
Official Title:
Pilot Study for the Development of an Activity and Quality of Life Questionnaire for the Follow-up of Patients With Non-dystrophic Myotonia
Status:
COMPLETED
Status Verified Date:
2024-09
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:
MNDActivLife
Brief Summary:
Non-dystrophic myotonias MND are rare neuromuscular diseases caused by mutations in the voltage-dependent channels of skeletal muscles resulting in delayed muscle relaxation after voluntary contraction They include various conditions such as congenital myotonia congenital paramyotonia and sodium channel myotonia The main characteristic is myotonia muscle stiffness accompanied by pain fatigue and weakness Symptoms vary in intensity and fluctuation complicates clinical assessment
Until now no validated scale to assess the severity of myotonia is the subject of a consensus among neurologists It therefore seems necessary to establish a scale to simply and quickly assess the severity of myotonia to fill this need
The areas of this future scale were identified by the study coordinator based on existing questionnaires and scales These areas have been validated by a scientific committee composed of expert neurologists
The main objective of the study is to validate the adequacy and formulation of the scale questions by involving 10 patients who will complete the questionnaire twice to assess its fidelity At the end of the study the committee will exclude inappropriate questions The goal is to create a reliable scale to assess the severity of myotonia
Until now no validated scale to assess the severity of myotonia is the subject of a consensus among neurologists It therefore seems necessary to establish a scale to simply and quickly assess the severity of myotonia to fill this need
The areas of this future scale were identified by the study coordinator based on existing questionnaires and scales These areas have been validated by a scientific committee composed of expert neurologists
The main objective of the study is to validate the adequacy and formulation of the scale questions by involving 10 patients who will complete the questionnaire twice to assess its fidelity At the end of the study the committee will exclude inappropriate questions The goal is to create a reliable scale to assess the severity of myotonia
Detailed Description:
None
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