Safety and Tolerability of KH631 Gene Therapy in Subjects With Neovascular Age-related Macular Degeneration (nAMD)

Age-Related Macular Degeneration Clinical Trial

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)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05672121
• Other study ID #: KH631-40101
• Status: Recruiting
• Phase: Phase 1/Phase 2
• Start date: February 6, 2023
• Est. completion date: December 28, 2026

Study information

• Verified date: September 2023
• Source: Chengdu Kanghong Pharmaceutical Group Co., Ltd.
• Contact: Qiang Zheng
• Phone: 86 13880331037
• Email: zhengqiang[@]cnkh.com
• Is FDA regulated: No
• Study type: Interventional

Clinical 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 42
• Est. completion date: December 28, 2026
• Est. primary completion date: December 28, 2026
• Accepts healthy volunteers: No
• Gender: All
• Age group: 50 Years to 85 Years

Eligibility

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.

Related Conditions & MeSH terms

• Age-Related Macular Degeneration
• Macular Degeneration

Intervention

Drug:
• KH631
KH631: AAV vector containing a coding sequence for an anti-VEGF protein

Locations

Country	Name	City	State
China	Beijing Tongren Hospital, Capital Medical University	Beijing

Sponsors (1)

Lead Sponsor	Collaborator
Chengdu Origen Biotechnology Co., Ltd.

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Other	KH631 protein in aqueous fluid and blood	Exploratory	104 weeks
Other	VEGF-A in aqueous fluid and blood	Exploratory	104 weeks
Primary	Safety	incidence of AEs and SAEs	24 weeks
Primary	Change in best corrected visual acuity	BCVA	52 weeks
Secondary	Safety	incidence of AEs and SAEs	104 weeks
Secondary	Change in best corrected visual acuity	BCVA	104 weeks
Secondary	Change in central retinal thickness	CRT	104 weeks
Secondary	Change in area of retinal leakage	Leakage measured by FFA	104 weeks
Secondary	Rescue injections	Mean number of rescue injections	104 weeks