Age-Related Macular DegenerationPhase 1NCT05657301
Safety and Tolerability of KH631 Gene Therapy in Participants With Neovascular Age-related Macular Degeneration
A Phase 1 study testing KH631 in people with Age-Related Macular Degeneration, run by Chengdu Origen Biotechnology Co., Ltd. at 7 sites in United States.
Trial summary
VAN-2201 is Phase I clinical trial to assess the safety and tolerability of KH631 in subjects with neovascular AMD. KH631 is gene therapy designed to deliver a protein which targets and blocks VEGF via an adeno-associated viral vector. Read more about this study
- Who is it for?
- Ages 50 to 85 years
- Conditions the study is for
- Age-Related Macular Degeneration
- What is being tested?
- KH631
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 7 sitesUnited States
- Study dates
- 16 Oct 2023to Sep 2027
- 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
- 1. Males and Females ages 50 to 85 (inclusive) with a study eye which meets the following criteria:
- Previously received IVT treatment of anti-VEGF for neovascular AMD, with documented response to anti-VEGF therapy during the first 2 weeks of screening b. active macular CNV lesion secondary to AMD evidenced by SD-OCT c. Have a ETDRS BCVA letter score of 63 to 19 (approximately 20/63 to 20/400 Snellen equivalent) in the study eye at Screening for the first subject in each cohort (sentinel subject), followed by ETDRS BCVA letter score of 73 to 19 (approximately 20/40 to 20/400 Snellen equivalent) for the rest of the subjects each cohort; d. Pseudophakia in the study eye, with ocular media to permit high quality fundus imaging at screening and allow planned vitrectomy and subretinal injection; e. Are willing and able to sign the study written informed consent form (ICF).
Who cannot join
- Have had any prior ocular or systemic treatment (investigational or approved) or surgery for the treatment of neovascular AMD except IVT anti-VEGF
- Retinal pigment epithelial tears or rips at screening
- Any history or presence of vitreous hemorrhage;
- Have any condition preventing visual acuity improvement;
- Have any other cause of CNV; prior pars plana vitrectomy or scleral buckling or retinal detachment surgery; macular hole, Epiretinal membrane or vitreo-macular traction; full thickness macular hole;
- History of intraocular or periocular surgery in the prior 3 months;
- Prior trabeculectomy or other filtration surgery ;
- Any use of long-acting intraocular steroids, including implants, within six months prior;
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
United States7 sites
Kanghong Investigative Site
Boston, Massachusetts, 02114
Kanghong Investigative Site
Cherry Hill, New Jersey, 08034
Kanghong Investigative Site
Dallas, Texas, 75231
Kanghong Investigative Site
Germantown, Tennessee, 38138
Kanghong Investigative Site
Lemont, Illinois, 60439
Kanghong Investigative Site
Phoenix, Arizona, 85016
Kanghong Investigative Site
Reno, Nevada, 89502
About this study
Official title: A Phase I, Open-label, Multicenter, Dose-Escalating Study to Evaluate the Safety and Tolerability of KH631 Gene Therapy in Participants With Neovascular Age-related Macular Degeneration
VAN-2201 is Phase I clinical trial to assess the safety and tolerability of KH631 in subjects with neovascular AMD. KH631 is gene therapy designed to deliver a protein which targets and blocks VEGF via an adeno-associated viral vector. The standard of care for patients with neovascular AMD are anti-VEGF treaments, which have demonstrated improvement in vision and reduction in fluid. A one time placement of a product which inhibits VEGF has the potential to reduce the patient burden of regular intraocular injections.
Read the detailed description
The VAN-2201 clinical trial is a multicenter, open-label, dose-escalating clinical study. The primary objective of the study will be to establish a safe and tolerable dose range in subjects with neovascular AMD. Five dose cohorts are planned to be included in the study, with approximately five subjects per cohort. Subjects who meet the eligibility criteria (i.e. all the inclusion and no exclusion criteria) will be enrolled to receive KH631. KH631 will be delivered at the dose level according to the cohort via intraocular injection. Subjects will be seen monthly until the primary outcome measurement at 24 weeks and through week 52. The subjects will be continued to followed with regular visits until they complete the 104 week visit.
- Conditions
- Age-Related Macular Degeneration
- Being tested
- KH631
- Medical subject headings
- Macular Degeneration
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
KH631Drug
KH631: AAV vector containing a coding sequence for an anti-VEGF protein
| Study group | Role | What they receive |
|---|---|---|
| KH631 Dose 1 | Experimental | KH631 One-Time Intraocular Injection Dose Level 1 Receives: Drug: KH631 |
| KH631 Dose 2 | Experimental | KH631 One-Time Intraocular Injection Dose Level 2 Receives: Drug: KH631 |
| KH631 Dose 3 | Experimental | KH631 One-Time Intraocular Injection Dose Level 3 Receives: Drug: KH631 |
| KH631 Dose 4 | Experimental | KH631 One-Time Intraocular Injection Dose Level 4 Receives: Drug: KH631 |
| KH631 Dose 5 | Experimental | KH631 One-Time Intraocular Injection Dose Level 5 Receives: Drug: KH631 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety (Type, Severity and Incidence of ocular and systemic AEs and SAEs)
type, severity and incidence of ocular and systemic AEs and SAEs
Measured: 52 Weeks
Best Corrected Visual Acuity
Change in Best Corrected Visual Acuity
Measured: 52 Weeks
Secondary outcomes (3)
Safety (type, severity and incidence of ocular and systemic AEs and SAEs)
type, severity and incidence of ocular and systemic AEs and SAEs
Measured: 104 Weeks
Rescue Injections
Mean Number of Rescue Injections
Measured: 104 Weeks
Best Corrected Visual Acuity
Change in Best Corrected Visual Acuity
Measured: 104 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Dec 2022
- Study start16 Oct 2023
- Primary completionSep 2026 (estimated)
- Study completionSep 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Age-Related Macular DegenerationStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
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
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chengdu Origen Biotechnology Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Avner Ingerman, MD, MSc, Study director, Vanotech Ltd.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05657301
- Sponsor's protocol ID
- VAN-2201
- Status last verified
- Dec 2025
- First submitted
- 10 Dec 2022
- Last updated
- 31 Dec 2025
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 31 Dec 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I, Open-label, Multicenter, Dose-Escalating Study to Evaluate the Safety and Tolerability of KH631 Gene Therapy in Participants With Neovascular Age-related Macular Degeneration
- ClinicalTrials.gov ID
- NCT05657301
- Acronym / sponsor ID
- VAN-2201
- Conditions
- Age-Related Macular Degeneration
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Active, not recruiting
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- Chengdu Origen Biotechnology Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 16 Oct 2023 (actual)
- Primary completion
- Sep 2026 (estimated)
- Study completion
- Sep 2027 (estimated)
- First posted
- 20 Dec 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
VAN-2201 is Phase I clinical trial to assess the safety and tolerability of KH631 in subjects with neovascular AMD. KH631 is gene therapy designed to deliver a protein which targets and blocks VEGF via an adeno-associated viral vector. The standard of care for patients with neovascular AMD are anti-VEGF treaments, which have demonstrated improvement in vision and reduction in fluid. A one time placement of a product which inhibits VEGF has the potential to reduce the patient burden of regular intraocular injections.
Detailed description
The VAN-2201 clinical trial is a multicenter, open-label, dose-escalating clinical study. The primary objective of the study will be to establish a safe and tolerable dose range in subjects with neovascular AMD. Five dose cohorts are planned to be included in the study, with approximately five subjects per cohort. Subjects who meet the eligibility criteria (i.e. all the inclusion and no exclusion criteria) will be enrolled to receive KH631. KH631 will be delivered at the dose level according to the cohort via intraocular injection. Subjects will be seen monthly until the primary outcome measurement at 24 weeks and through week 52. The subjects will be continued to followed with regular visits until they complete the 104 week visit.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 5
| Arm | Type | Receives |
|---|---|---|
| KH631 Dose 1 | Experimental | Drug: KH631KH631 One-Time Intraocular Injection Dose Level 1 |
| KH631 Dose 2 | Experimental | Drug: KH631KH631 One-Time Intraocular Injection Dose Level 2 |
| KH631 Dose 3 | Experimental | Drug: KH631KH631 One-Time Intraocular Injection Dose Level 3 |
| KH631 Dose 4 | Experimental | Drug: KH631KH631 One-Time Intraocular Injection Dose Level 4 |
| KH631 Dose 5 | Experimental | Drug: KH631KH631 One-Time Intraocular Injection Dose Level 5 |
Who may qualify
- Ages
- 50 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- 1. Males and Females ages 50 to 85 (inclusive) with a study eye which meets the following criteria:
- Previously received IVT treatment of anti-VEGF for neovascular AMD, with documented response to anti-VEGF therapy during the first 2 weeks of screening b. active macular CNV lesion secondary to AMD evidenced by SD-OCT c. Have a ETDRS BCVA letter score of 63 to 19 (approximately 20/63 to 20/400 Snellen equivalent) in the study eye at Screening for the first subject in each cohort (sentinel subject), followed by ETDRS BCVA letter score of 73 to 19 (approximately 20/40 to 20/400 Snellen equivalent) for the rest of the subjects each cohort; d. Pseudophakia in the study eye, with ocular media to permit high quality fundus imaging at screening and allow planned vitrectomy and subretinal injection; e. Are willing and able to sign the study written informed consent form (ICF).
Key exclusion criteria (8 listed)
- Have had any prior ocular or systemic treatment (investigational or approved) or surgery for the treatment of neovascular AMD except IVT anti-VEGF
- Retinal pigment epithelial tears or rips at screening
- Any history or presence of vitreous hemorrhage;
- Have any condition preventing visual acuity improvement;
- Have any other cause of CNV; prior pars plana vitrectomy or scleral buckling or retinal detachment surgery; macular hole, Epiretinal membrane or vitreo-macular traction; full thickness macular hole;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| KH631KH631: AAV vector containing a coding sequence for an anti-VEGF protein | Drug | KH631 Dose 1, KH631 Dose 2, KH631 Dose 3, KH631 Dose 4, KH631 Dose 5 |
Primary outcome
Safety (Type, Severity and Incidence of ocular and systemic AEs and SAEs)
Time frame: 52 Weeks
type, severity and incidence of ocular and systemic AEs and SAEs
Best Corrected Visual Acuity
Time frame: 52 Weeks
Change in Best Corrected Visual Acuity
3 secondary outcomes
Safety (type, severity and incidence of ocular and systemic AEs and SAEs)
Time frame: 104 Weeks
Rescue Injections
Time frame: 104 Weeks
Best Corrected Visual Acuity
Time frame: 104 Weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Dec 2025.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Avner Ingerman, MD, MSc study director
Vanotech Ltd.
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing KH631 in people with Age-Related Macular Degeneration, run by Chengdu Origen Biotechnology Co., Ltd. at 7 sites in United States.
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 2 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: under way, not looking for participants, as of the sponsor's last update on 31 Dec 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites in United States. 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?
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?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety (Type, Severity and Incidence of ocular and systemic AEs and SAEs) (time frame: 52 Weeks). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 31 Dec 2025.