Clinical trial NCT05672121: Safety and Tolerability of KH631 Gene Therapy in Subjects With Neovascular Age-related Macular Degeneration (nAMD)
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)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE1
PHASE2
Conditions: Age-Related Macular Degeneration
Lead sponsor: Chengdu Kanghong Pharmaceutical Group Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2023-01-05
Enrollment: 42
Sex: ALL
Minimum age (years): 50
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief 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.


Eligibility criteria: Inclusion Criteria:

* 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.

Exclusion Criteria:

* 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.

Primary outcomes:
measure: Safety
timeFrame: 24 weeks
measure: Change in best corrected visual acuity
timeFrame: 52 weeks
Secondary outcomes:
measure: Safety
timeFrame: 104 weeks
measure: Change in best corrected visual acuity
timeFrame: 104 weeks
measure: Change in central retinal thickness
timeFrame: 104 weeks
measure: Change in area of retinal leakage
timeFrame: 104 weeks
measure: Rescue injections
timeFrame: 104 weeks
Other outcomes:
measure: KH631 protein in aqueous fluid and blood
timeFrame: 104 weeks
measure: VEGF-A in aqueous fluid and blood
timeFrame: 104 weeks
Locations:
city: Beijing
country: China
facility: Beijing Tongren Hospital, Capital Medical University
Study officials:
affiliation: Beijing Tongren Hospital Affiliated to Capital Medical University
name: Wenbin Wei, PhD
role: PRINCIPAL_INVESTIGATOR
References: