Ignite Creation Date:
2024-05-06 @ 7:42 PM
Last Modification Date:
2024-10-26 @ 3:11 PM
Study NCT ID:
NCT06100276
Status:
RECRUITING
Last Update Posted:
2024-07-09
First Post:
2023-10-20
Brief Title:
Safety Tolerability and Exploratory Efficacy Study of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants with SOD1 Amyotrophic Lateral Sclerosis SOD1-ALS
Sponsor:
UniQure Biopharma BV
Organization:
UniQure Biopharma BV
Study Overview
Official Title:
A Phase 12 Multicenter Single Ascending Dose Study to Evaluate the Safety Tolerability and Exploratory Efficacy of Intrathecally Administered Gene Therapy AMT-162 in Adult Participants with SOD1 Amyotrophic Lateral Sclerosis SOD1-ALS
Status:
RECRUITING
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:
None
Brief Summary:
This is the study of AMT-162 in Participants with SOD1-ALS and is designed to evaluate the safety tolerability and exploratory efficacy of intrathecally administered gene therapy AMT-162 AMT-162-001 is a Phase 12 multi-center single ascending dose study
Detailed Description:
AMT-162 is an investigational gene therapy that encodes an artificial microribonucleic acid microRNA or miRNA targeting the SOD1 gene This clinical study will test the safety of AMT-162 and explore the hypothesis that it will silence expression of mutant cytosolic SOD1 and thereby ameliorate the course of ALS caused by this mutant gene
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
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