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

NCT05672121 | Recruiting Phase 1 DMC

• 1 other identifier: KH631-40101
• Study Type: interventional
• Target: 42
• Locations: 1 country
• Sites: 1

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.

Conditions

Age-Related Macular Degeneration

Outcome Measures

Primary Outcomes (2)

• Safety

  incidence of AEs and SAEs

  24 weeks

• Change in best corrected visual acuity

  BCVA

  52 weeks

Secondary Outcomes (5)

• Safety

  104 weeks

• Change in best corrected visual acuity

  104 weeks

• Change in central retinal thickness

  104 weeks

• Change in area of retinal leakage

  104 weeks

• Rescue injections

  104 weeks

Other Outcomes (2)

• KH631 protein in aqueous fluid and blood

  104 weeks

• VEGF-A in aqueous fluid and blood

  104 weeks

Study Arms (5)

KH631 Dose 1

EXPERIMENTAL

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

Drug: KH631

KH631 Dose 2

EXPERIMENTAL

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

Drug: KH631

KH631 Dose 3

EXPERIMENTAL

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

Drug: KH631

KH631 Dose 4

EXPERIMENTAL

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

Drug: KH631

KH631 Dose 5

EXPERIMENTAL

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

Drug: KH631

Interventions

KH631 DRUG

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

KH631 Dose 1; KH631 Dose 2; KH631 Dose 3; KH631 Dose 4; KH631 Dose 5

Eligibility Criteria

• Age: 50 Years - 85 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Chengdu Origen Biotechnology Co., Ltd.: lead

Study Sites (1)

Beijing Tongren Hospital, Capital Medical University

Beijing, China

RECRUITING

MeSH Terms

Conditions

Macular Degeneration

Condition Hierarchy (Ancestors)

Retinal Degeneration; Retinal Diseases; Eye Diseases

Study Officials

• Wenbin Wei, PhD

  Beijing Tongren Hospital Affiliated to Capital Medical University

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Qiang Zheng

CONTACT

86 13880331037; zhengqiang@cnkh.com

Shanshan Ji

CONTACT

86 18188058101; 022078@cnkh.com

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: December 21, 2022
• First Posted: January 5, 2023
• Study Start: February 6, 2023
• Primary Completion (Estimated): December 28, 2026
• Study Completion (Estimated): December 28, 2026
• Last Updated: November 27, 2024

Record last verified: 2024-11

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)