Open-laBel Dose-escalation Study for CRISPR/cas13- Rna TargetInG THerapy for the Treatment of Neovascular Age-related Macular Degeneration in Phase I Trial

NCT ID: NCT06623279

Last Updated: 2024-10-02

Basic Information

• Recruitment Status: NOT_YET_RECRUITING
• Clinical Phase: PHASE1
• Total Enrollment: 15 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2025-04-01
• Study Completion Date: 2031-02-01

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 CRISPR/Cas13 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.

Conditions

Neovascular Age-Related Macular Degeneration (nAMD)

Study Design

• Allocation Method: NA
• Intervention Model: SEQUENTIAL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

HG202

The study will enroll up to 3 dose cohorts：Low dose/Middle dose/High dose

Group Type: EXPERIMENTAL

HG202

Intervention Type: GENETIC

Method of Administration: Once unilateral subretinal injection; The duration of the study includes a 4-week screening period, enrollment visit, treatment visit and 52 weeks follow-up period, 4 more years long term follow up as an extension study.

Interventions

HG202

Method of Administration: Once unilateral subretinal injection; The duration of the study includes a 4-week screening period, enrollment visit, treatment visit and 52 weeks follow-up period, 4 more years long term follow up as an extension study.

Intervention Type: GENETIC

Eligibility Criteria

Inclusion Criteria

• Males or females ≥ 50 and ≤ 85 years at the time of signing the ICF;
• Active macular choroidal neovascularization (MNV) secondary to nAMD in the study eye;
• Sentinel (1st) subject for each dose cohort must have a BCVA ≤ 20/63 and ≥ 20/400 (≤63 and ≥ 19 ETDRS letters) in the study eye. Following the sentinel subject evaluation, the rest of the subjects in the dose cohort must have a BCVA between ≤ 20/40 and ≥ 20/400 (≤ 73 and ≥ 19 ETDRS letters) in the study eye.
• Able to perform visual acuity and retinal function tests and able and willing to comply with study procedures for this clinical trial.

Exclusion Criteria

• Retinal or subretinal hemorrhage, scarring, or fibrosis of greater than 50% of the total lesion in the study eye;
• Other ocular diseases that may affect central vision in the study eye;
• Any other cause of CNV than nAMD in the study eye
• Uncontrolled glaucoma in the study eye;
• History or presence of corneal transplant or corneal dystrophy in the study eye;
• History of other intraocular surgery in the study eye within 3 months prior to baseline;
• Prior gene therapy or oligonucleotide therapy;
• Other conditions judged by the investigator as inappropriate for the study.

• Minimum Eligible Age: 50 Years
• Maximum Eligible Age: 85 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

HuidaGene Therapeutics Co., Ltd.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Study Director

Role: STUDY_DIRECTOR

HuidaGene Therapeutics Co., Ltd.

Central Contacts

Study Director

Role: CONTACT

HG20202@huidagene.com

732-318-9873

Other Identifiers

HG20202

Identifier Type: -

Identifier Source: org_study_id