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

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

ACTIVE_NOT_RECRUITING

Clinical Phase

PHASE1

Total Enrollment

25 participants

Study Classification

INTERVENTIONAL

Study Start Date

2023-10-16

Study Completion Date

2027-09-30

Brief Summary

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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

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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.

Conditions

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Age-Related Macular Degeneration

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Intervention Type DRUG

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

Interventions

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KH631

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

Intervention Type DRUG

Eligibility Criteria

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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;

Minimum Eligible Age

50 Years

Maximum Eligible Age

85 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Avner Ingerman, MD, MSc

Role: STUDY_DIRECTOR

Vanotech Ltd.

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United States

Other Identifiers

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VAN-2201

Identifier Type: -

Identifier Source: org_study_id

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

Study Results

Basic Information

Brief Summary

Detailed Description

Conditions

Study Design

Study Groups

KH631 Dose 1

KH631

KH631 Dose 2

KH631

KH631 Dose 3

KH631

KH631 Dose 4

KH631

KH631 Dose 5

KH631

Interventions

KH631

Eligibility Criteria

Inclusion Criteria

Exclusion Criteria

Sponsors

Chengdu Origen Biotechnology Co., Ltd.

Responsible Party

Principal Investigators

Avner Ingerman, MD, MSc

Locations

Kanghong Investigative Site

Kanghong Investigative Site

Kanghong Investigative Site

Kanghong Investigative Site

Kanghong Investigative Site

Kanghong Investigative Site

Kanghong Investigative Site

Countries

Other Identifiers

VAN-2201