Ignite Creation Date:
2024-10-26 @ 3:39 PM
Last Modification Date:
2024-10-26 @ 3:39 PM
Study NCT ID:
NCT06576388
Status:
COMPLETED
Last Update Posted:
None
First Post:
2024-08-26
Brief Title:
Clinical Trial to Evaluate the Safety and Efficacy of EXG001-307 in Patients With Spinal Muscular Atrophy Type I
Sponsor:
None
Organization:
None
Study Overview
Official Title:
Clinical Trial Evaluating the Safety and Efficacy of EXG001-307 in Patients With Type I Spinal Muscular Atrophy
Status:
COMPLETED
Status Verified Date:
2024-08
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:
The purpose of this study was to evaluate the safety and preliminary efficacy of a single intravenous injection of exg001-307 in patients with type I spinal muscular atrophy
The research process includes the screening period the screening period is from the time the subject signs the informed consent to the time before hormone pretreatment with a maximum of 28 days the treatment period the subject receives hospitalization and observation including hormone pretreatment and single infusion of study drugs and the follow-up period the end of the treatment period until the subject reaches the age of 18 months loss of follow-up active withdrawal from the study or death The qualified subjects in the screening period enter the treatment period receive exg001-307 treatment and enter the follow-up period after hospitalization observation At the end of the study visit subjects 18 months old eligible subjects will be asked to transfer to the long-term follow-up study
The research process includes the screening period the screening period is from the time the subject signs the informed consent to the time before hormone pretreatment with a maximum of 28 days the treatment period the subject receives hospitalization and observation including hormone pretreatment and single infusion of study drugs and the follow-up period the end of the treatment period until the subject reaches the age of 18 months loss of follow-up active withdrawal from the study or death The qualified subjects in the screening period enter the treatment period receive exg001-307 treatment and enter the follow-up period after hospitalization observation At the end of the study visit subjects 18 months old eligible subjects will be asked to transfer to the long-term follow-up study
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