Clinical trial NCT06458595: Safety and Tolerability of KH658 Gene Therapy in Subjects With Neovascular Age-related Macular Degeneration (nAMD)
Official title: A Phase I/II Study to Evaluate the Tolerability, Safety and Efficacy of KH658 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: 2024-06-13
Enrollment: 44
Sex: ALL
Minimum age (years): 50
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SEQUENTIAL
Primary purpose: TREATMENT

Brief summary: KH658 is a adeno-associated virus (AAV) vector-based gene therapy for suprachoroidal space 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 informed consent form (ICF);
2. Female and male aged 50 to 85 years (inclusive) with nAMD;
3. Have previously received anti-VEGF treatment for nAMD, with documented response to anti-VEGF therapy and a minimum of 2 anti-VEGF IVT injections in the study eye in the 6 months prior to screening;
4. Have a ETDRS BCVA letter score of 83 to 19 (approximately 20/25 to 20/400 Snellen equivalent) in the study eye at Screening;
5. Have a ETDRS BCVA letter score of ≥19 in the fellow eye at Screening;
6. Females must be at least 1 year postmenopausal.

Exclusion Criteria:

1. Have any other cause of CNV (such as pathological myopia, etc.) in the study eye; or non-nAMD disease in the study eye judged by the investigator that could impact macular examination or affect central vision (such as diabetic retinopathy, central retinal vein occlusion, etc.);
2. Any condition preventing visual acuity improvement in the study eye, e.g. fovea scar, fibrosis or atrophy;
3. Have a subretinal hemorrhage that is either 50% or more of the total lesion area, or blood is greater than 1.0 mm2 under the fovea in the study eye at screening;
4. Have any vitreous hemorrhage or history of vitreous hemorrhage in the study eye;
5. Presence of an implant (excluding intraocular lens), opacification of the refractive medium or miosis that affects fundus examination;
6. Have a history of or presence of retinal detachment or choroidal detachment in the study eye.

Primary outcomes:
measure: Tolerability and safety of KH658 in different dose levels
timeFrame: Week 4， Week 26
measure: Change in BCVA letter
timeFrame: Week 26
Secondary outcomes:
measure: Incidence of adverse events and adverse reactions
timeFrame: Week 4， 26， 38， 54
measure: Efficacy (BCVA, CRT)
timeFrame: Week 38， 54
measure: Number of supplemental injections
timeFrame: Week 54
measure: Annualized rate of supplemental injections
timeFrame: Week 54
measure: Percentage of subjects free of rescue IVT therapy
timeFrame: Week 54
Other outcomes:
Locations:
city: Tianjin
country: China
facility: Tianjin Medical University General Hospital
Study officials:
References: