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Neovascular Age-related Macular Degeneration(nAMD)Early Phase 1NCT06031727

CRISPR/cas13-medIated RNA TarGeting THerapy for the Treatment of Neovascular Age-related Macular Degeneration Investigator-initiated Trial (SIGHT-I)

A Early Phase 1 study testing HG202 in people with Neovascular Age-related Macular Degeneration(nAMD), run by HuidaGene Therapeutics Co., Ltd. at 2 sites in China.

Recruiting

Looking for participants now. Contact the study team or a site below to ask about taking part.

Sites and contacts
Official record
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Trial summary

Age-related macular degeneration (AMD) is a progressive disease leading to severe and irreversible vision loss of which the neovascular AMD (nAMD) accounted for 90% blindness in AMD. nAMD is primarily driven by the perturbation of vascular endothelial growth factor (VEGF). Read more about this study

Who is it for?
Ages 50 to 80 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
12 participants
Where
2 sitesChina
Study dates
4 Sep 2023to 30 Jun 2026
Phase
Early 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 80 years
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • Males or females ≥ 50 and ≤ 80 years at the time of signing the ICF
  • Diagnosed of choroidal neovascularization (CNV) secondary to AMD in the study eye;
  • Best-corrected visual acuity (BCVA) ranged from 73 to 23 early treatment diabetic retinopathy study (ETDRS)letter score (corresponding to 20/32 to 20/320 of Snellen visual acuity) in the study eye;
  • BCVA in the non-study eye had an ETDRS letter score of 19(equivalent to Snellen visual acuity20/400) and above;
  • Able to perform visual acuity and retinal function tests and able and willing to comply with study procedures for this clinical trial;
  • RESPONSIVE SUBJECTS:
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  • History of need for and responsive to anti-VEGF therapy in the study eye
  • NON-RESPONSIVE SUBJECTS:
  • History of receiving anti-VEGF therapy but is resistant to treatment, which is defined as: a. complete or near-complete remission of subretinal fluid after the initial 3 doses of anti-VEGF agents and thenno improvement (less than 50um reduction) or deterioration of CRT by OCT

Who cannot join

  • Subretinal hemorrhage, scarring, or fibrosis of greater than 50% of the total lesion in the study eye;
  • Any condition in the Investigator's opinion that could limit visual improvement in the study eye;
  • Other ocular diseases that may affect central vision in the study eye (e.g., retinal vein occlusion, retinal detachment, macular hole, optic nerve disease, etc.);
  • Presence of CNV not due to nAMD in the study eye,
  • Uncontrolled glaucoma in the study eye;
  • Active intraocular inflammation or a history of uveitis in either eye;
Show all 12Show fewer
  • History or presence of corneal dystrophy in the study eye;
  • Subjects with immunodeficiency diseases prone to opportunistic infections;
  • History of other intraocular surgery in the study eye within 3 months prior to baseline that in the Investigator's opinion could impact healing or study outcome interpretation;
  • Prior gene therapy or oligonucleotide therapy;
  • History of acute coronary syndrome, myocardial infarction, coronary revascularization, cerebrovascular accident, or transient ischemic attack within 6 months prior to the Screening Visit;
  • Other conditions judged by the investigator as inappropriate for the study.

Trial locations and contacts

2 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Study Director contact

+86 021-25076143

HG20201@huidagene.com

China2 sites, 2 recruiting

  • Eye & ENT Hospital of Fudan University

    Shanghai, Shanghai Municipality

    Gezhi Xu, PhD, +86 18017316316, drxugezhi@163.com

    Recruiting

  • Tianjin Medical University Eye Hospital

    Tianjin, Tianjing

    Xiaorong Li, PhD, +86 18622818042, Xiaorli@163.com

    Recruiting

Other trials in China

About this study

Official title: A Trial to Evaluate the Safety, Tolerability, and Efficacy of CRISPR-Cas13 RNA-editing Therapy Targeting Knockdown of Vascular Endothelial Growth Factor a (HG202) in the Treatment of Neovascular Age-related Macular Degeneration (nAMD)

Age-related macular degeneration (AMD) is a progressive disease leading to severe and irreversible vision loss of which the neovascular AMD (nAMD) accounted for 90% blindness in AMD. nAMD is primarily driven by the perturbation of vascular endothelial growth factor (VEGF). VEGF overexpression leads to abnormal growth of choroidal neovascularization (CNV), which is a hallmark of AMD. Although anti-VEGF agents are effective in treating nAMD, long-term efficacy decreases over time due to the need for repeated injections impacting patient compliance with treatment regimen while patients still may lose vision during the 7th or 8th year of treatment. These frequent intravitreal injections can increase the risk of complications, including submacular hemorrhage, intraocular hypertension, inflammation, and retinal detachment. Furthermore, there are up to 46% of nAMD patients using anti-VEGF agents who have shown poor response or have developed tachyphylaxis with anti-VEGF therapies. HG202 is a CRISPR/Cas13 RNA-editing therapy packaging novel high-fidelity Cas13 technology using one single AAV vector to partially knock-down the expression of VEGFA and thus inhibit CNV formation in AMD patients who are either responsive or non-responsive to anti-VEGF agents. The long-term, stable delivery of HG202 following a one (1) time gene-editing therapy treatment for nAMD could potentially reduce the frequent injection treatment burden of currently available therapies AND treat nAMD patients who are non-responsive to anti-VEGF therapies and have no treatment.

Being tested
HG202
Keywords
Macular Degeneration/nAMD/wAMD/Gene-editing/CRISPR/HG202

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 is about 52 weeks for each subject including a 4 weeks screening period, enrollment/baseline visit, treatment visit and 48 weeks follow-up period.

Study groups in NCT06031727
Study groupRoleWhat they receive
HG202ExperimentalNot described
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: 24 weeks

Secondary outcomes (4)

  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: 48 weeks

  2. 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: 24 and 48 weeks

  3. Change from baseline in central retinal thickness (CRT)

    Change from baseline in central retinal thickness (CRT) as measured by optical coherence tomography (OCT) in the study eye at different doses

    Measured: 24 and 48 weeks

  4. Change From baseline 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: 48 weeks

Timeline

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

  1. First posted11 Sep 2023
  2. Study start4 Sep 2023
  3. Primary completion30 Jun 2025 (estimated)
  4. Study completion30 Jun 2026 (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
Early Phase 1
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
12 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT06031727
Sponsor's protocol ID
HG20201
Status last verified
Mar 2025
First submitted
1 Sep 2023
Last updated
17 Mar 2025
Results posted
No
Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Early Phase 1 study testing HG202 in people with Neovascular Age-related Macular Degeneration(nAMD), run by HuidaGene Therapeutics Co., Ltd. at 2 sites in China.

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 9 inclusion and 12 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: looking for participants now, as of the sponsor's last update on 17 Mar 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in China. Whether a site is taking part right now should be confirmed with that site. See every site.

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?

Early Phase 1 (sometimes called Phase 0) studies give a very small amount of a treatment to a few people, mainly to see how the body handles it.

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 (+86 021-25076143, HG20201@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: 24 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 NCT06031727.

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 17 Mar 2025.

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