Ignite Creation Date:
2024-06-16 @ 11:50 AM
Last Modification Date:
2024-10-26 @ 3:31 PM
Study NCT ID:
NCT06444217
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-06-06
First Post:
2024-05-30
Brief Title:
Gene Therapy Development and Validation for Huntingtons Disease Fibro TG-HD
Sponsor:
University Hospital Angers
Organization:
University Hospital Angers
Study Overview
Official Title:
Gene Therapy Development and Validation for Huntingtons Disease Fibro TG-HD
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-05
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:
FibroTG-HD
Brief Summary:
Huntingtons disease is a rare and fatal monogenic neurodegenerative disorder whose molecular origin is an expansion of CAG triplets within the first exon of the Huntingtin gene Although a growing number of emerging therapies are in clinical trials there are no proven neuroprotective or curative treatments approved by the health authorities as they have not yet demonstrated any real therapeutic benefit or absence of toxicity Trans-splicing gene therapy is defined as the correction of a mutated endogenous pre-messenger RNA by a therapeutic exogenous pre-messenger RNA Trans-splicing is a suitable alternative approach since it is capable of allelic selectivity and replacement of mutated sequences by the wild-type one criteria that no therapy tested to date meets This project involves the therapeutic validation of trans-splicing of Huntingtin gene transcripts and will evaluate its therapeutic effects in vitro into primary fibroblast cell lines derived from skin biopsies of Huntingtons disease patients
Detailed Description:
None
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
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