Ignite Creation Date:
2024-10-26 @ 3:36 PM
Last Modification Date:
2024-10-26 @ 3:36 PM
Study NCT ID:
NCT06532474
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-07-29
Brief Title:
Exploring the Physiologic Pharmacodynamic and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Pilot Study Exploring the Physiologic Pharmacodynamic and Clinical Responses of Skeletal Muscle in Patients With Spinal Muscular Atrophy Treated With SMN-Directed Therapies
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:
None
Brief Summary:
In this observational study researchers are looking at the effects of spinal muscular atrophy SMA drugs on the muscles and nerve cells in patients with SMA
Primary Objectives
To evaluate the feasibility and reliability of performing MR functional imaging in exercising muscle in patients with SMA
To evaluate patients with SMA types 2 and 3 at baseline and longitudinally at 6 and 12 months
Secondary Objectives
To describe the MR functional bioenergetics response in the leg muscles in four potential groups of patients with spinal muscular atrophy untreated actively treated with nusinersen Spinraza or onasemnogene abeparvovec Zolgensma actively treated with risdiplam Evrysdi and switching from Spinraza or Zolgensma to Evrysdi
To identify changes in motor function in patients with SMA types 2 and 3 who initiate treatment with risdiplam
To obtain biomarkers in blood urine and muscle tissue to provide evidence for risdiplam effect on skeletal muscle
To obtain quality of life and disability data from participants in this study
Primary Objectives
To evaluate the feasibility and reliability of performing MR functional imaging in exercising muscle in patients with SMA
To evaluate patients with SMA types 2 and 3 at baseline and longitudinally at 6 and 12 months
Secondary Objectives
To describe the MR functional bioenergetics response in the leg muscles in four potential groups of patients with spinal muscular atrophy untreated actively treated with nusinersen Spinraza or onasemnogene abeparvovec Zolgensma actively treated with risdiplam Evrysdi and switching from Spinraza or Zolgensma to Evrysdi
To identify changes in motor function in patients with SMA types 2 and 3 who initiate treatment with risdiplam
To obtain biomarkers in blood urine and muscle tissue to provide evidence for risdiplam effect on skeletal muscle
To obtain quality of life and disability data from participants in this study
Detailed Description:
This is an observational study to demonstrate the feasibility of performing MR functional imaging in exercising muscle in patients with SMA The participants will be prescribed medication by their treating physician they will not receive any drug as part of this study
Participants participating in the ML43225 study will be put into one of four groups depending on their type of SMA and the drugs they may or may not be taking They will be asked to come to clinic 3 times over one year Each visit will include magnetic resonance MR studies a muscle ultrasound a nerve test muscle function testing lung function testing blood work vital signs and participants will be asked about their quality of life and daily life activities After participants have completed the 3 required visits they will be taken off study
Participants participating in the ML43225 study will be put into one of four groups depending on their type of SMA and the drugs they may or may not be taking They will be asked to come to clinic 3 times over one year Each visit will include magnetic resonance MR studies a muscle ultrasound a nerve test muscle function testing lung function testing blood work vital signs and participants will be asked about their quality of life and daily life activities After participants have completed the 3 required visits they will be taken off study
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