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

Sponsor

Chengdu Origen Biotechnology Co., Ltd. (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05672121

Collaborator

(none)

Enrollment

Location

Arms

Anticipated Duration (Months)

0.9

Patients Per Site Per Month

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Age-Related Macular Degeneration	Drug: KH631	Phase 1/Phase 2

Study Design

Study Type:

Interventional

Anticipated Enrollment :

42 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Treatment

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)

Anticipated Study Start Date :

Jan 1, 2023

Anticipated Primary Completion Date :

Dec 1, 2023

Anticipated Study Completion Date :

Dec 1, 2026

Arms and Interventions

Arm	Intervention/Treatment
Experimental: KH631 Dose 1 dose1:Administered by Subretinal injection. Dosage form: injection solution. Dose: 200uL. Frequency of administration: one time injection.	Drug: KH631 KH631: AAV vector containing a coding sequence for an anti-VEGF protein
Experimental: KH631 Dose 2 dose2:Administered by Subretinal injection. Dosage form: injection solution. Dose: 200uL. Frequency of administration: one time injection.	Drug: KH631 KH631: AAV vector containing a coding sequence for an anti-VEGF protein
Experimental: KH631 Dose 3 dose3:Administered by Subretinal injection. Dosage form: injection solution. Dose: 200uL. Frequency of administration: one time injection.	Drug: KH631 KH631: AAV vector containing a coding sequence for an anti-VEGF protein
Experimental: KH631 Dose 4 dose4:Administered by Subretinal injection. Dosage form: injection solution. Dose: 200uL. Frequency of administration: one time injection.	Drug: KH631 KH631: AAV vector containing a coding sequence for an anti-VEGF protein
Experimental: KH631 Dose 5 dose5:Administered by Subretinal injection. Dosage form: injection solution. Dose: 200uL. Frequency of administration: one time injection.	Drug: KH631 KH631: AAV vector containing a coding sequence for an anti-VEGF protein

Outcome Measures

Primary Outcome Measures

Safety [24 weeks]
incidence of AEs and SAEs
Change in best corrected visual acuity [52 weeks]
BCVA

Secondary Outcome Measures

Safety [104 weeks]
incidence of AEs and SAEs
Change in best corrected visual acuity [104 weeks]
BCVA
Change in central retinal thickness [104 weeks]
CRT
Change in area of retinal leakage [104 weeks]
Leakage measured by FFA
Rescue injections [104 weeks]
Mean number of rescue injections

Other Outcome Measures

KH631 protein in aqueous fluid and blood [104 weeks]
Exploratory
VEGF-A in aqueous fluid and blood [104 weeks]
Exploratory

Eligibility Criteria

Criteria

Ages Eligible for Study:

50 Years to 85 Years

Sexes Eligible for Study:

All

Accepts 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.

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Beijing Tongren Hospital, Capital Medical University	Beijing		China

Sponsors and Collaborators

Chengdu Origen Biotechnology Co., Ltd.

Investigators

Principal Investigator: Wenbin Wei, PhD, Beijing Tongren Hospital Affiliated to Capital Medical University

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Chengdu Origen Biotechnology Co., Ltd.

ClinicalTrials.gov Identifier:

NCT05672121

Other Study ID Numbers:

KH631-40101

First Posted:

Jan 5, 2023

Last Update Posted:

Jan 5, 2023

Last Verified:

Jan 1, 2023

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Macular Degeneration

Study Results

No Results Posted as of Jan 5, 2023