Ignite Creation Date:
2024-05-06 @ 8:23 PM
Last Modification Date:
2024-10-26 @ 3:27 PM
Study NCT ID:
NCT06369974
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-04-18
First Post:
2024-03-11
Brief Title:
Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy
Sponsor:
Massachusetts General Hospital
Organization:
Massachusetts General Hospital
Study Overview
Official Title:
An Open-label Single Center Single Participant Study of an Experimental Antisense Oligonucleotide Treatment for TUBB4A-related Leukodystrophy
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-04
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 research project entails delivery of a personalized antisense oligonucleotide ASO drug designed for a single pediatric participant with TUBB4A associated leukodystrophy
Detailed Description:
This is an interventional study to evaluate the safety and efficacy of treatment with an individualized antisense oligonucleotide ASO treatment in a single pediatric participant with a de novo pathogenic gain of function TUBB4A mutation associated with severe leukodystrophy with hypomyelination with atrophy of the basal ganglia and cerebellum H-ABC
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