Safety and Tolerability of KH658 Gene Therapy in Subjects With Neovascular Age-related Macular Degeneration (nAMD)

Phase 1

Recruiting

• Conditions: Age-related Macular Degeneration
• Interventions: Drug: KH658

• Registration Number: NCT06458595
• Lead Sponsor: Chengdu Origen Biotechnology Co., Ltd.

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.

Detailed Description

Not available

Study Timeline

• Status Verified: 2024-06
• Study First Submitted: 2024-06-09
• Study First Submitted QC: 2024-06-09
• Study First Posted: 2024-06-13
• Study Start: 2024-06-28
• Last Update Submitted: 2024-11-26
• Last Update Posted: 2024-11-28
• Primary Completion: 2026-03-31
• Study Completion: 2026-12-28

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 44

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SEQUENTIAL

Arm && Interventions

Group	Intervention	Description
KH658	KH658	-

Primary Outcome Measures

Name	Time	Method
Change in BCVA letter	Week 26
Tolerability and safety of KH658 in different dose levels	Week 4， Week 26

Secondary Outcome Measures

Name	Time	Method
Number of supplemental injections	Week 54
Incidence of adverse events and adverse reactions	Week 4， 26， 38， 54
Annualized rate of supplemental injections	Week 54
Efficacy (BCVA, CRT)	Week 38， 54
Percentage of subjects free of rescue IVT therapy	Week 54

Trial Locations

Locations (1)

Tianjin Medical University General Hospital; 🇨🇳 Tianjin, China