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

Primary Purpose

Age-Related Macular Degeneration

Status
Recruiting
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
KH631
Sponsored by
Chengdu Origen Biotechnology Co., Ltd.
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Age-Related Macular Degeneration

Eligibility Criteria

50 Years - 85 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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: Have had any prior ocular or systemic treatment (investigational or approved) or surgery for the treatment of neovascular AMD except IVT anti-VEGF Retinal pigment epithelial tears or rips at screening Any history or presence of vitreous hemorrhage; Have any condition preventing visual acuity improvement; 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; History of intraocular or periocular surgery in the prior 3 months; Prior trabeculectomy or other filtration surgery ; Any use of long-acting intraocular steroids, including implants, within six months prior;

Sites / Locations

  • Kanghong Investigative SiteRecruiting

Arms of the Study

Arm 1

Arm 2

Arm 3

Arm 4

Arm 5

Arm Type

Experimental

Experimental

Experimental

Experimental

Experimental

Arm Label

KH631 Dose 1

KH631 Dose 2

KH631 Dose 3

KH631 Dose 4

KH631 Dose 5

Arm Description

KH631 One-Time Intraocular Injection Dose Level 1

KH631 One-Time Intraocular Injection Dose Level 2

KH631 One-Time Intraocular Injection Dose Level 3

KH631 One-Time Intraocular Injection Dose Level 4

KH631 One-Time Intraocular Injection Dose Level 5

Outcomes

Primary Outcome Measures

Safety (Type, Severity and Incidence of ocular and systemic AEs and SAEs)
type, severity and incidence of ocular and systemic AEs and SAEs
Best Corrected Visual Acuity
Change in Best Corrected Visual Acuity

Secondary Outcome Measures

Safety (type, severity and incidence of ocular and systemic AEs and SAEs)
type, severity and incidence of ocular and systemic AEs and SAEs
Rescue Injections
Mean Number of Rescue Injections
Best Corrected Visual Acuity
Change in Best Corrected Visual Acuity

Full Information

First Posted
December 10, 2022
Last Updated
August 21, 2023
Sponsor
Chengdu Origen Biotechnology Co., Ltd.
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1. Study Identification

Unique Protocol Identification Number
NCT05657301
Brief Title
Safety and Tolerability of KH631 Gene Therapy in Participants With Neovascular Age-related Macular Degeneration
Official Title
A Phase I, Open-label, Multicenter, Dose-Escalating Study to Evaluate the Safety and Tolerability of KH631 Gene Therapy in Participants With Neovascular Age-related Macular Degeneration
Study Type
Interventional

2. Study Status

Record Verification Date
August 2023
Overall Recruitment Status
Recruiting
Study Start Date
August 2023 (Anticipated)
Primary Completion Date
September 2026 (Anticipated)
Study Completion Date
September 2027 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Chengdu Origen Biotechnology Co., Ltd.

4. Oversight

Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

Brief Summary
VAN-2201 is Phase I clinical trial to assess the safety and tolerability of KH631 in subjects with neovascular AMD. KH631 is gene therapy designed to deliver a protein which targets and blocks VEGF via an adeno-associated viral vector. The standard of care for patients with neovascular AMD are anti-VEGF treaments, which have demonstrated improvement in vision and reduction in fluid. A one time placement of a product which inhibits VEGF has the potential to reduce the patient burden of regular intraocular injections.
Detailed Description
The VAN-2201 clinical trial is a multicenter, open-label, dose-escalating clinical study. The primary objective of the study will be to establish a safe and tolerable dose range in subjects with neovascular AMD. Five dose cohorts are planned to be included in the study, with approximately five subjects per cohort. Subjects who meet the eligibility criteria (i.e. all the inclusion and no exclusion criteria) will be enrolled to receive KH631. KH631 will be delivered at the dose level according to the cohort via intraocular injection. Subjects will be seen monthly until the primary outcome measurement at 24 weeks and through week 52. The subjects will be continued to followed with regular visits until they complete the 104 week visit.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Age-Related Macular Degeneration

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
25 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
KH631 Dose 1
Arm Type
Experimental
Arm Description
KH631 One-Time Intraocular Injection Dose Level 1
Arm Title
KH631 Dose 2
Arm Type
Experimental
Arm Description
KH631 One-Time Intraocular Injection Dose Level 2
Arm Title
KH631 Dose 3
Arm Type
Experimental
Arm Description
KH631 One-Time Intraocular Injection Dose Level 3
Arm Title
KH631 Dose 4
Arm Type
Experimental
Arm Description
KH631 One-Time Intraocular Injection Dose Level 4
Arm Title
KH631 Dose 5
Arm Type
Experimental
Arm Description
KH631 One-Time Intraocular Injection Dose Level 5
Intervention Type
Drug
Intervention Name(s)
KH631
Intervention Description
KH631: AAV vector containing a coding sequence for an anti-VEGF protein
Primary Outcome Measure Information:
Title
Safety (Type, Severity and Incidence of ocular and systemic AEs and SAEs)
Description
type, severity and incidence of ocular and systemic AEs and SAEs
Time Frame
52 Weeks
Title
Best Corrected Visual Acuity
Description
Change in Best Corrected Visual Acuity
Time Frame
52 Weeks
Secondary Outcome Measure Information:
Title
Safety (type, severity and incidence of ocular and systemic AEs and SAEs)
Description
type, severity and incidence of ocular and systemic AEs and SAEs
Time Frame
104 Weeks
Title
Rescue Injections
Description
Mean Number of Rescue Injections
Time Frame
104 Weeks
Title
Best Corrected Visual Acuity
Description
Change in Best Corrected Visual Acuity
Time Frame
104 Weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
50 Years
Maximum Age & Unit of Time
85 Years
Accepts Healthy Volunteers
No
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: Have had any prior ocular or systemic treatment (investigational or approved) or surgery for the treatment of neovascular AMD except IVT anti-VEGF Retinal pigment epithelial tears or rips at screening Any history or presence of vitreous hemorrhage; Have any condition preventing visual acuity improvement; 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; History of intraocular or periocular surgery in the prior 3 months; Prior trabeculectomy or other filtration surgery ; Any use of long-acting intraocular steroids, including implants, within six months prior;
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Sponsor Clinical Contact
Phone
+1 267-644-6708
Email
van2201@cnkh.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Avner Ingerman, MD, MSc
Organizational Affiliation
Vanotech Ltd.
Official's Role
Study Director
Facility Information:
Facility Name
Kanghong Investigative Site
City
Katy
State/Province
Texas
ZIP/Postal Code
77494
Country
United States
Individual Site Status
Recruiting

12. IPD Sharing Statement

Plan to Share IPD
No

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

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