Ignite Creation Date:
2024-10-26 @ 3:41 PM
Last Modification Date:
2024-10-26 @ 3:41 PM
Study NCT ID:
NCT06623279
Status:
NOT_YET_RECRUITING
Last Update Posted:
None
First Post:
2024-09-30
Brief Title:
Open-laBel Dose-escalation Study for CRISPRcas13- Rna TargetInG THerapy for the Treatment of Neovascular Age-related Macular Degeneration in Phase I Trial
Sponsor:
None
Organization:
None
Study Overview
Official Title:
A Phase 1 Open-label Multiple-cohort Dose-escalation Study to Evaluate the Safety and Tolerability of HG202 High-fidelity CRISPR-Cas13 hfCas13Y RNA-targeting Therapy for Neovascular Age-related Macular Degeneration nAMD
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-09
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:
BRIGHT
Brief Summary:
Age-related macular degeneration AMD leads to severe and irreversible vision loss while neovascular AMD nAMD accounts for 80-90 of AMD blindness Current anti-VEGF therapies are the standard of care but these therapies require life-long repeated intraocular injections These frequent intravitreal injections increase the risk of complications including submacular hemorrhage intraocular hypertension inflammation and retinal detachment Therefore repeated treatments for nAMD place a substantial burden on healthcare systems patients and their caregivers Additionally approximately 25-35 of individuals with aggressive nAMD show suboptimal responses to the anti-VEGF therapies experience treatment-extended failure or require intensive frequent intraocular injections and do not prevent irreversible vision loss
HG202 is a CRISPRCas13 RNA-editing therapy delivered through one single AAV vector to partially knock down the expression of VEGFA and thus inhibit CNV formation in AMD The long-term stable delivery of HG202 following a one-time gene-editing therapy treatment for nAMD may potentially reduce the frequent injections and the potential risks of currently available anti-VEGF therapies since it does not rely on the long-term expression of anti-VEGF antibodies
HG202 is a CRISPRCas13 RNA-editing therapy delivered through one single AAV vector to partially knock down the expression of VEGFA and thus inhibit CNV formation in AMD The long-term stable delivery of HG202 following a one-time gene-editing therapy treatment for nAMD may potentially reduce the frequent injections and the potential risks of currently available anti-VEGF therapies since it does not rely on the long-term expression of anti-VEGF antibodies
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