Ignite Creation Date:
2024-10-25 @ 7:49 PM
Last Modification Date:
2024-10-26 @ 3:38 PM
Study NCT ID:
NCT06562283
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-08-05
Brief Title:
Evaluation of the Reproducibility of a Fatigability Test Fitted to Patients With Spinal Muscular Atrophy
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Evaluation of the Reproducibility of a Fatigability Test Fitted to Patients With Spinal Muscular Atrophy
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:
FANTASI-SMART
Brief Summary:
Spinal muscular atrophy SMA is an autosomal recessive neuromuscular disease caused by the degeneration of motor neurons in the anterior horn of the spinal cord due to the absence of the SMN1 gene and the resulting lack of SMN protein Some patients with particularly severe forms types 0 or 1 die before the age of 2 in the absence of treatment while others retain autonomous walking throughout their lives with no reduction in life expectancy Three treatments aimed at restoring SMN TRS protein expression have recently been approved by the US Food and Drug Administration and the European Medicines Agency ie Nusinersen Onasemnogene Abeparvovec Risdiplam Patients treated with TRS after the onset of symptoms symptomatic patients may show significant motor improvement but retain difficulties such as muscle weakness and fatigue leading to limitations in activities of daily living The aim of this study is to adapt a fatigability test widely validated in its original version in different populations QIF test but adapted in this protocol to the motor level and low abilities of certain SMA patients Our objectives are to determine whether these assessments are feasible in SMA patients reproducible and relevant for monitoring this population either routinely or for future clinical trials
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