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

Chengdu Origen Biotechnology

Status and phase: Enrolling; Phase 2; Phase 1

Conditions: Age-Related Macular Degeneration

Treatments: Drug: KH631

Study type: Interventional

Funder types: Industry

Identifiers

NCT05672121

KH631-40101

Details and patient eligibility

About

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.

Enrollment: 42 estimated patients

Sex: All

Ages: 50 to 85 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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42 participants in 5 patient groups

KH631 Dose 1

Experimental group

Description:

dose1:Administered by Subretinal injection. Dosage form: injection solution. Dose: 200uL. Frequency of administration: one time injection.

Treatment:

Drug: KH631

KH631 Dose 2

Experimental group

Description:

dose2:Administered by Subretinal injection. Dosage form: injection solution. Dose: 200uL. Frequency of administration: one time injection.

Treatment:

Drug: KH631

KH631 Dose 3

Experimental group

Description:

dose3:Administered by Subretinal injection. Dosage form: injection solution. Dose: 200uL. Frequency of administration: one time injection.

Treatment:

Drug: KH631

KH631 Dose 4

Experimental group

Description:

dose4:Administered by Subretinal injection. Dosage form: injection solution. Dose: 200uL. Frequency of administration: one time injection.

Treatment:

Drug: KH631

KH631 Dose 5

Experimental group

Description:

dose5:Administered by Subretinal injection. Dosage form: injection solution. Dose: 200uL. Frequency of administration: one time injection.

Treatment:

Drug: KH631