Ignite Creation Date:
2024-10-26 @ 3:40 PM
Last Modification Date:
2024-10-26 @ 3:40 PM
Study NCT ID:
NCT06599606
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
None
First Post:
2024-09-12
Brief Title:
Ultrasonographic Muscle Assessment and Functional Scales in Spinal Muscular Atrophy
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Investigation of the Relationship Between Ultrasonographic Muscle Thickness Measurement and Echogenicity Assessment with Functional Scales in Patients with Spinal Muscular Atrophy an Observational Cross-Sectional Case-Control Study
Status:
ACTIVE_NOT_RECRUITING
Status Verified Date:
2024-09
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:
None
Brief Summary:
This study aims to investigate the relationship between ultrasonographic muscle thickness measurement and echogenicity assessment with functional scales in children with spinal muscular atrophy SMA By utilizing ultrasonographic techniques the study seeks to provide objective biomarkers for assessing muscle health and monitoring treatment response Currently the evaluation of SMA often relies on subjective clinical assessments this study addresses that gap by offering more precise and objective indicators of disease progression and functional status The ultimate goal is to improve treatment strategies and enhance patient outcomes through better assessment tools
Detailed Description:
Spinal muscular atrophy SMA is a neuromuscular disorder that typically leads to progressive lower motor neuron loss from early childhood The most common form is caused by deletions in the survival motor neuron 1 SMN1 gene inherited in an autosomal recessive manner SMA is classified into at least three subtypes based on the age of onset and the level of motor development achieved In Type 1 SMA severe form symptoms usually appear before 6 months of age and children are unable to sit unsupported In Type 2 SMA intermediate form symptoms start between 6 and 18 months and children cannot walk unsupported Type 3 SMA mild form presents after the second year of life and affected children can walk unsupported Although the exact prevalence and carrier rates of SMA in our country are not fully determined it is estimated that there are between 130 and 180 new cases annually given approximately 1200000 live births each year The total number of SMA patients in the country is about 3000
Despite current treatments muscle weakness and significant functional loss affecting quality of life are commonly observed in SMA patients At present the efficacy of SMA treatment is measured through clinical assessment including monitoring of changes in motor developmental milestones and functional scales However the lack of objective biomarkers that are less reliant on patient cooperation as opposed to functional scales complicates monitoring treatment response for patients Establishing reliable methods to assess disease progression and treatment response is crucial for conducting robust clinical studies
Musculoskeletal ultrasonography presents itself as a suitable imaging modality for use as a biomarker in SMA management due to its non-invasiveness repeatability absence of radiation ability to evaluate multiple muscles quickly and ease of application Furthermore the feasibility of performing ultrasonographic examinations at the bedside facilitates access to a larger number of patients especially those with limited mobility or requiring respiratory support Previous literature has reported the advantages of muscle ultrasonography in evaluating neuromuscular disorders including SMA
In conclusion this study which will investigate the relationship between ultrasonographic muscle thickness measurement and echogenicity evaluation and functional scales in patients diagnosed with SMA for the first time in our country will guide physicians and researchers seeking to assess disease progression determine treatment efficacy and develop rehabilitation strategies
Despite current treatments muscle weakness and significant functional loss affecting quality of life are commonly observed in SMA patients At present the efficacy of SMA treatment is measured through clinical assessment including monitoring of changes in motor developmental milestones and functional scales However the lack of objective biomarkers that are less reliant on patient cooperation as opposed to functional scales complicates monitoring treatment response for patients Establishing reliable methods to assess disease progression and treatment response is crucial for conducting robust clinical studies
Musculoskeletal ultrasonography presents itself as a suitable imaging modality for use as a biomarker in SMA management due to its non-invasiveness repeatability absence of radiation ability to evaluate multiple muscles quickly and ease of application Furthermore the feasibility of performing ultrasonographic examinations at the bedside facilitates access to a larger number of patients especially those with limited mobility or requiring respiratory support Previous literature has reported the advantages of muscle ultrasonography in evaluating neuromuscular disorders including SMA
In conclusion this study which will investigate the relationship between ultrasonographic muscle thickness measurement and echogenicity evaluation and functional scales in patients diagnosed with SMA for the first time in our country will guide physicians and researchers seeking to assess disease progression determine treatment efficacy and develop rehabilitation strategies
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