AzTrials
AzTrials

Neovascular Age-Related Macular Degeneration (nAMD)Phase 1BRIGHTNCT06623279

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 study testing HG202 in people with Neovascular Age-Related Macular Degeneration (nAMD), run by HuidaGene Therapeutics Co., Ltd..

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
Search all trials

Trial 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. Read more about this study

Who is it for?
Ages 50 to 85 years
What is being tested?
HG202
Type of treatment
Injection or infusion
Could I receive a placebo?
No, everyone receives an active treatment
How many people
15 participants
Where
No sites listed yet
Study dates
1 Apr 2025to 1 Feb 2031
Phase
Phase 1
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 50 to 85 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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.

Who cannot join

  • 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.

Trial locations and contacts

No sites are listed yet. Contact the study team or a site near you to ask about taking part.

Study Director contact

732-318-9873

HG20202@huidagene.com

About this study

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)

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.

Being tested
HG202
Keywords
Macular Degeneration, nAMD, wAMD, Gene-editing, CRISPR, HG202
Medical subject headings
Macular Degeneration

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • HG202Genetic

    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.

Study groups in NCT06623279
Study groupRoleWhat they receive
HG202ExperimentalThe study will enroll up to 3 dose cohorts：Low dose/Middle dose/High dose
Receives: Genetic: HG202

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Incidence and severity of ocular and systemic adverse events

    Number of adverse events (AEs), serious adverse events (SAEs), and dose-limiting toxicities (DLTs)

    Measured: 52 weeks

Secondary outcomes (2)

  1. Mean change from baseline in best-corrected visual acuity (BCVA)

    Change from baseline in BCVA as measured by Early Treatment Diabetic Retinopathy Study (ETDRS) chart in the study eye at different doses

    Measured: 52 weeks

  2. Mean change in annualized rate of supplemental injections

    Change from baseline in the number of anti-VEGF injections which is annualized to a per year rate in the study eye at different doses

    Measured: 52 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted2 Oct 2024
  2. Study start1 Apr 2025 (estimated)
  3. Primary completion1 Feb 2027 (estimated)
  4. Study completion1 Feb 2031 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Sequential
Model description
Three dosing cohort: Low dose : 3 subjects Middle dose: 6 subjects High dose: 6 subjects
Masking
None (open label)
Enrolment
15 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06623279
Sponsor's protocol ID
HG20202
Acronym
BRIGHT
Status last verified
Sep 2024
First submitted
30 Sep 2024
Last updated
2 Oct 2024
Results posted
No
Data monitoring committee
No
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing HG202 in people with Neovascular Age-Related Macular Degeneration (nAMD), run by HuidaGene Therapeutics Co., Ltd..

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: opening soon, as of the sponsor's last update on 2 Oct 2024. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Study Director (732-318-9873, HG20202@huidagene.com), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Incidence and severity of ocular and systemic adverse events (time frame: 52 weeks). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT06623279.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Oct 2024; AzTrials last refreshed its copy on 31 Oct 2026.

AzTrials uses cookies for analytics and advertising. You can accept or reject them; the site works either way. Privacy Policy.

Free account

Log in to keep searching

You’ve used your 3 free searches. AzTrials is free: log in or create a free account to keep searching clinical trials and save the ones you want to follow.

Create a free accountLog in