Age-Related Macular DegenerationPhase 1/2NCT05672121
Safety and Tolerability of KH631 Gene Therapy in Subjects With Neovascular Age-related Macular Degeneration (nAMD)
A Phase 1/2 study testing KH631 in people with Age-Related Macular Degeneration, run by Chengdu Origen Biotechnology Co., Ltd. at 1 site in China.
Trial summary
KH631 is a adeno-associated virus (AAV) vector-based gene therapy for subretinal injection. The long-term, stable therapeutic protein after one time injection for nAMD could potentially reduce the treatment burden and maintain vision. 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
- 42 participants
- Where
- 1 siteChina
- Study dates
- 6 Feb 2023to 28 Dec 2026
- Phase
- Phase 1/2
- 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.Are willing and able to sign the study written informed consent form (ICF); 2. Men and women ≥ 50 and ≤85 years of age, diagnosed with nAMD at the Screening visit; 3. Subjects must be under active anti-VEGF treatment for nAMD and received a minimum of 3 injections within 6 months prior to screening; 4. Response to anti-VEGF therapy(Response is defined as reduction in CRT≥50μm or at least 30% reduction in fluid by OCT compared to disease at the worst); 5. BCVA between ≤20/63 and ≥20/400(≤63 and ≥19 Early Treatment Diabetic Retinopathy Study [ETDRS] letters) for the first patient in each cohort followed by BCVA between ≤20/40 and ≥20/400(≤73 and ≥19 ETDRS letters) for the rest of the cohort; 6. Must be pseudophakic(at least 3 months after intraocular lens implantation) in the study eye; 7.Female subjects must have been postmenopausal for at least 1 year.
Who cannot join
- 1.Any other cause of CNV, including pathologic myopia, etc, or other diseases except nAMD have an influence on the test of macular or affect the central visual acuity; 2.Presence of an implant, refractive media opacity affects fundus examination or narrow pupil of the study eye; 3.Active or history of retinal detachment in the study eye; 4.Uncontrolled glaucoma or ocular hypertension; 5.Have taken the drug known to have retinal toxicity; 6.History of intraocular surgery; 7.Uncontrolled hypertension despite medication at the screening visit.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
China1 site, 1 recruiting
- Recruiting
Beijing Tongren Hospital, Capital Medical University
Beijing
Wenbin Wei, PhD, 86 13701255115, tr_weiwenbin@163.com
About this study
Official title: A Phase I/II, Open-label, Multiple-cohort, Dose-escalation Study to Evaluate the Safety and Tolerability of KH631 Gene Therapy in Subjects With Neovascular Age-related Macular Degeneration (nAMD)
KH631 is a adeno-associated virus (AAV) vector-based gene therapy for subretinal injection. The long-term, stable therapeutic protein after one time injection for nAMD could potentially reduce the treatment burden and maintain vision.
- 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 | dose1:Administered by Subretinal injection. Dosage form: injection solution. Dose: 200uL. Frequency of administration: one time injection. Receives: Drug: KH631 |
| KH631 Dose 2 | Experimental | dose2:Administered by Subretinal injection. Dosage form: injection solution. Dose: 200uL. Frequency of administration: one time injection. Receives: Drug: KH631 |
| KH631 Dose 3 | Experimental | dose3:Administered by Subretinal injection. Dosage form: injection solution. Dose: 200uL. Frequency of administration: one time injection. Receives: Drug: KH631 |
| KH631 Dose 4 | Experimental | dose4:Administered by Subretinal injection. Dosage form: injection solution. Dose: 200uL. Frequency of administration: one time injection. Receives: Drug: KH631 |
| KH631 Dose 5 | Experimental | dose5:Administered by Subretinal injection. Dosage form: injection solution. Dose: 200uL. Frequency of administration: one time injection. Receives: Drug: KH631 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Safety
incidence of AEs and SAEs
Measured: 24 weeks
Change in best corrected visual acuity
BCVA
Measured: 52 weeks
Secondary outcomes (5)
Safety
incidence of AEs and SAEs
Measured: 104 weeks
Change in best corrected visual acuity
BCVA
Measured: 104 weeks
Change in central retinal thickness
CRT
Measured: 104 weeks
Change in area of retinal leakage
Leakage measured by FFA
Measured: 104 weeks
Rescue injections
Mean number of rescue injections
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 posted5 Jan 2023
- Study start6 Feb 2023
- Primary completion28 Dec 2026 (estimated)
- Study completion28 Dec 2026 (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 1/2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 42 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Chengdu Origen Biotechnology Co., Ltd.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Wenbin Wei, PhD, Principal investigator, Beijing Tongren Hospital Affiliated to Capital Medical University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05672121
- Sponsor's protocol ID
- KH631-40101
- Status last verified
- Nov 2024
- First submitted
- 21 Dec 2022
- Last updated
- 27 Nov 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 27 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase I/II, Open-label, Multiple-cohort, Dose-escalation Study to Evaluate the Safety and Tolerability of KH631 Gene Therapy in Subjects With Neovascular Age-related Macular Degeneration (nAMD)
- ClinicalTrials.gov ID
- NCT05672121
- Acronym / sponsor ID
- KH631-40101
- Conditions
- Age-Related Macular Degeneration
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 42 participants (estimated)
- Lead sponsor
- Chengdu Origen Biotechnology Co., Ltd.Industry (pharma, biotech, devices)
- Study start
- 6 Feb 2023 (actual)
- Primary completion
- 28 Dec 2026 (estimated)
- Study completion
- 28 Dec 2026 (estimated)
- First posted
- 5 Jan 2023 (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.
KH631 is a adeno-associated virus (AAV) vector-based gene therapy for subretinal injection. The long-term, stable therapeutic protein after one time injection for nAMD could potentially reduce the treatment burden and maintain vision.
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: KH631dose1:Administered by Subretinal injection. Dosage form: injection solution. Dose: 200uL. Frequency of administration: one time injection. |
| KH631 Dose 2 | Experimental | Drug: KH631dose2:Administered by Subretinal injection. Dosage form: injection solution. Dose: 200uL. Frequency of administration: one time injection. |
| KH631 Dose 3 | Experimental | Drug: KH631dose3:Administered by Subretinal injection. Dosage form: injection solution. Dose: 200uL. Frequency of administration: one time injection. |
| KH631 Dose 4 | Experimental | Drug: KH631dose4:Administered by Subretinal injection. Dosage form: injection solution. Dose: 200uL. Frequency of administration: one time injection. |
| KH631 Dose 5 | Experimental | Drug: KH631dose5:Administered by Subretinal injection. Dosage form: injection solution. Dose: 200uL. Frequency of administration: one time injection. |
Who may qualify
- Ages
- 50 to 85 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- 1.Are willing and able to sign the study written informed consent form (ICF); 2. Men and women ≥ 50 and ≤85 years of age, diagnosed with nAMD at the Screening visit; 3. Subjects must be under active anti-VEGF treatment for nAMD and received a minimum of 3 injections within 6 months prior to screening; 4. Response to anti-VEGF therapy(Response is defined as reduction in CRT≥50μm or at least 30% reduction in fluid by OCT compared to disease at the worst); 5. BCVA between ≤20/63 and ≥20/400(≤63 and ≥19 Early Treatment Diabetic Retinopathy Study [ETDRS] letters) for the first patient in each cohort followed by BCVA between ≤20/40 and ≥20/400(≤73 and ≥19 ETDRS letters) for the rest of the cohort; 6. Must be pseudophakic(at least 3 months after intraocular lens implantation) in the study eye; 7.Female subjects must have been postmenopausal for at least 1 year.
Key exclusion criteria (1 listed)
- 1.Any other cause of CNV, including pathologic myopia, etc, or other diseases except nAMD have an influence on the test of macular or affect the central visual acuity; 2.Presence of an implant, refractive media opacity affects fundus examination or narrow pupil of the study eye; 3.Active or history of retinal detachment in the study eye; 4.Uncontrolled glaucoma or ocular hypertension; 5.Have taken the drug known to have retinal toxicity; 6.History of intraocular surgery; 7.Uncontrolled hypertension despite medication at the screening visit.
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
Time frame: 24 weeks
incidence of AEs and SAEs
Change in best corrected visual acuity
Time frame: 52 weeks
BCVA
5 secondary outcomes
Safety
Time frame: 104 weeks
Change in best corrected visual acuity
Time frame: 104 weeks
Change in central retinal thickness
Time frame: 104 weeks
Change in area of retinal leakage
Time frame: 104 weeks
Rescue injections
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 27 Nov 2024.
- Individual participant dataSharing planned: No
Related trials for Age-Related Macular Degeneration
Active, not recruitingObservational study
Retinal Pigment Epithelium Safety Study For Patients In B4711001
Age Related Macular Degeneration
Active, not recruitingObservational study
Real World Evidence in China: Faricimab Use in Diabetic Macular Edema, Retinal Vein Occlusion, and Neovascular Age-Related Macular Degeneration (The Farseeing Study)
Diabetic Macular Edema, Neovascular Age-related Macular Degeneration
RecruitingPhase 3
A Study of 36-Week Refill Exchanges of Port Delivery System (PDS) With Ranibizumab in nAMD
Neovascular Age-related Macular Degeneration
RecruitingObservational study
Pivotal Trial of an Automated AI-based System for Early Diagnosis and Prediction of Late Age-related Macular Degeneration
Age-related Macular Degeneration (AMD)
Active, not recruitingObservational study
Age-Related Eye Disease Study (AREDS) Follow-Up
Age-Related Macular Degeneration, Cataract
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.
Wenbin Wei, PhD principal investigator
Beijing Tongren Hospital Affiliated to Capital Medical University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing KH631 in people with Age-Related Macular Degeneration, run by Chengdu Origen Biotechnology Co., Ltd. at 1 site 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 1 inclusion and 1 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 27 Nov 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site 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 combined 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.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
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 Qiang Zheng (86 13880331037, zhengqiang@cnkh.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Safety (time frame: 24 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 27 Nov 2024; AzTrials last refreshed its copy on 8 Nov 2026.