Contact SCGE




Gene Therapy Trial Report

Summary

Safety and Tolerability of KH631 Gene Therapy in Participants With Neovascular Age-related Macular Degeneration


NCTID NCT05657301 (View at clinicaltrials.gov)
Description
Development Status Active
Indication Neovascular (Wet) Age-Related Macular Degeneration (nAMD)
Disease Ontology Term DOID:10871
Compound Name KH631
Sponsor Chengdu Origen Biotechnology Co., Ltd.
Funder Type Industry
Recruitment Status
Active not recruiting
Enrollment Count 25 (ESTIMATED)
Results Posted Not Available

Therapy Information


Target Gene/Variant VEGFR1/R2 fusion protein
Therapy Type Gene transfer
Therapy Route In-vivo
Mechanism of Action Genetic delivery of therapeutic protein
Route of Administration Intravitreal
Drug Product Type Viral vector
Delivery System Viral transduction
Vector Type AAV8
Editor Type none
Dose 1 5 undisclosed dose levels (200ul/dose)

Study Record Dates


Current Stage Phase1
Submit Date 2022-12-10
Completion Date 2027-09
Last Update 2025-12-31

Participation Criteria


Eligible Age 50 Years - 85 Years
Standard Ages Adult, Older adult
Sexes Eligible for Study ALL
Eligibility Criteria
Inclusion Criteria: 1\. Males and Females ages 50 to 85 (inclusive) with a study eye which meets the following criteria: a. Previously received IVT treatment of anti-VEGF for neovascular AMD, with documented response to anti-VEGF therapy during the first 2 weeks of screening b. active macular CNV lesion secondary to AMD evidenced by SD-OCT c. Have a ETDRS BCVA letter score of 63 to 19 (approximately 20/63 to 20/400 Snellen equivalent) in the study eye at Screening for the first subject in each cohort (sentinel subject), followed by ETDRS BCVA letter score of 73 to 19 (approximately 20/40 to 20/400 Snellen equivalent) for the rest of the subjects each cohort; d. Pseudophakia in the study eye, with ocular media to permit high quality fundus imaging at screening and allow planned vitrectomy and subretinal injection; e. Are willing and able to sign the study written informed consent form (ICF). Exclusion Criteria: 1. Have had any prior ocular or systemic treatment (investigational or approved) or surgery for the treatment of neovascular AMD except IVT anti-VEGF 2. Retinal pigment epithelial tears or rips at screening 3. Any history or presence of vitreous hemorrhage; 4. Have any condition preventing visual acuity improvement; 5. Have any other cause of CNV; prior pars plana vitrectomy or scleral buckling or retinal detachment surgery; macular hole, Epiretinal membrane or vitreo-macular traction; full thickness macular hole; 6. History of intraocular or periocular surgery in the prior 3 months; 7. Prior trabeculectomy or other filtration surgery ; 8. Any use of long-acting intraocular steroids, including implants, within six months prior;
View Inclusion and Exclusion Criteria at ClinicalTrials.gov

Locations


No.of Trial Sites 7
Locations United States

Regulatory Information


Has US IND True
FDA Designations
Recent Updates 1st patient dosed 11/20/23

Resources/Links