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Safety and Efficacy Study of Novel Gene Therapy AGX-08 for Geographic Atrophy

ConditionGeographic Atrophy
Trial statusNot yet recruiting
Trial phaseEarly Phase 1
Trial typeInterventional
Biological sexAll
Age50+
SponsorZhongmou Therapeutics

About this trial

This is AGX-08's safety, tolerability, and efficacy in Geographic Atrophy first-in-human study. This trial is meant to evaluate the safety and efficacy of AGX-08 in Geographic Atrophy patients. Unilateral intravitreal injections (IVT) will be given into the subject's Study Eye.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Clinically confirmed diagnosis of geographic atrophy (GA) secondary to age-related macular degeneration (AMD);

Age ≥ 50 years, regardless of sex;

Best-corrected visual acuity (BCVA) in the study eye measured using the ETDRS chart at a starting distance of 4 meters must be ≤35 letters (Snellen equivalent ≤20/200), with vision no worse than light perception; the fellow eye must not have better visual acuity than the study eye;

GA lesion size between 2.5 and 17.5 mm² with clearly defined borders. For multifocal GA, at least one lesion must be ≥1.25 mm² (0.5 disc area) to ensure measurable efficacy assessment;

Disqualifiers

GA caused by conditions other than AMD (e.g., Stargardt disease, cone-rod dystrophy, or other inherited macular dystrophies);

Aphakia in the study eye, or cataract surgery/YAG capsulotomy within 3 months prior to screening;

Refractive status or axial length outside the following range: spherical equivalent between -6.00D and +5.00D, and axial length between 21 mm and 26 mm;

Two baseline BCVA measurements (ETDRS) taken at least 14 days apart during screening differ by >30%;

Trial design

Design model

Parallel

Treatments tested in this trial

  • AGX-08-L

    Drug

    rAAV2.AG-eNCRV intravitreal injection of low dose

  • AGX-08-H

    Drug

    rAAV2.AG-eNCRV intravitreal injection of high dose

  • AGX-08-S

    Procedure/Surgery

    sham intravitreal injection of AGX-08 (not actual injection)

Treatment groups

24 Participants
are divided into 3 treatment groups
Group A: Low DoseExperimental treatment 1 intervention
Group B: High DoseExperimental treatment 1 intervention
Group C: ControlSham comparator 1 intervention

Trial outcomes

Primary outcomes

1

Incidence of treatment-emergent adverse events,adverse events, serious adverse events and dose-limiting toxicities

Incidence of severity of ocular and systemic treatment-emergent adverse events, (TEAEs), adverse events (AEs) , serious adverse events (SAEs) and dose-limiting toxicities(DLTs) following a single intravitreal injection of AGX-08.

Time frame
baseline to Week 52

Secondary outcomes

1

Change in Fundus Autofluorescence (FAF)

FAF is a non-invasive imaging technique that provides critical information about the health of the retina, which is vital for the functioning of the retina.

Time frame
baseline to Week 52
2

Change in best corrected visual acuity (BCVA)

BCVA of both eyes will be assessed using the early treatment of diabetic retinopathy study (ETDRS) chart or tumbling "E" chart or other avaliable measurement. This approach was chosen to facilitate visual acuity testing in subject who cannot recognize letters.

Time frame
baseline to Week 52

Other outcomes

Locations

This trial has no locations.

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Zhongmou Therapeutics

Lead sponsor

Source ClinicalTrials.gov

This trial is not recruiting at the moment. You can still explore other options: