NCT06623279Not yet recruitingPhase 1
Open-laBel Dose-escalation Study for CRISPR/cas13- Rna TargetInG THerapy for the Treatment of Neovascular Age-related Macular Degeneration in Phase I Trial
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)
Source: ClinicalTrials.gov · Last updated 2 Oct 2024 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
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.
- Status (registry)
- Not yet recruiting
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 15 (estimated)
- First submitted
- 30 Sept 2024
- Start date
- 1 Apr 2025
- Primary completion date
- 1 Feb 2027
- Lead sponsor
- HuidaGene Therapeutics Co., Ltd.
- Diseases
- Macular degeneration
- NCT number
- NCT06623279
What you commit to
- Study type: Interventional · Phase 1
- Planned participants: 15
- From 1 Apr 2025 to the primary completion date, 1 Feb 2027
- No sites listed in no country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study design.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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 illness
- 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;
- Other conditions judged by the investigator as inappropriate for the study.
Other
- Any other cause of CNV than nAMD in the study eye
- Uncontrolled glaucoma in the study eye;
Who the study is looking for
- 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.
Age in the registry: 50 years to 85 years · Sex: All · Does not accept healthy volunteers
This page is information, not medical advice. Discuss any trial with your doctor.