Ignite Creation Date:
2025-12-24 @ 11:39 PM
Ignite Modification Date:
2026-01-04 @ 8:51 PM
Study NCT ID:
NCT07063251
Status:
RECRUITING
Last Update Posted:
2025-08-27
First Post:
2025-07-03
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
An Clinical Study Evaluating the Safety, Tolerability, and efficAcy of HG005 in StaRgardT Disease
Sponsor:
HuidaGene Therapeutics Co., Ltd.
Organization:
Study Overview
Official Title:
An Investigator-initiated, Open-label, Dose-escalation Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of HG005 in Pediatric Patients With Stargardt Disease (STGD1) Caused by Biallelic ABCA4 Mutations
Status:
RECRUITING
Status Verified Date:
2025-08
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:
START
Brief Summary:
Stargardt disease type 1 (STGD1) is a rare genetic eye condition that causes progressive vision loss, often beginning in childhood or adolescence. It is the most common form of inherited macular degeneration and can lead to legal blindness. STGD1 is caused by mutations in the ABCA4 gene, which normally helps clear waste from the photoreceptor cells in the retina. When ABCA4 gene doesn't function properly, toxic substances like A2E accumulate and damage the retinal pigment epithelium (RPE), leading to vision loss.
There are currently no approved treatments for STGD1. HG005 is an investigational gene therapy designed to deliver a healthy copy of the ABCA4 gene to the retina. Because the gene is too large to fit into a single AAV (adeno-associated virus) vector, HG005 used two AAV vectors that work together in retinal cells to produce the full-length, functional ABCA4 protein. The goal of HG005 is to restore normal waste removal, protect retinal cells from further damage, and slow or stop vision loss.
There are currently no approved treatments for STGD1. HG005 is an investigational gene therapy designed to deliver a healthy copy of the ABCA4 gene to the retina. Because the gene is too large to fit into a single AAV (adeno-associated virus) vector, HG005 used two AAV vectors that work together in retinal cells to produce the full-length, functional ABCA4 protein. The goal of HG005 is to restore normal waste removal, protect retinal cells from further damage, and slow or stop vision loss.
Detailed Description:
None
Study Oversight
Has Oversight DMC:
False
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?: