Gene Therapy for Diabetic Macular Edema

Study Overview
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Brief Summary
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FT-003 is a gene therapy product developed for the treatment of central involvement diabetic macular edema (CI-DME). Diabetic retinopathy is one of the most common microvascular complications of diabetes mellitus, and diabetic macular edema is the main cause of vision loss in patients with diabetic retinopathy. In the latest guidelines, anti-VEGF therapy is preferred for CI-DME. Administration of FT-003 has the potential to treat CI-DME by providing intraocular protein which is durable expressed in therapeutic level. FT-003 is designed to reduce the current treatment burden which often results in undertreatment and vision loss in patients with CI-DME receiving anti-VEGF therapy in clinical practice.

Official Title
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An Open-label, Multy-center, Dose-escalation Clinical Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of FT-003 in Subjects With Diabetic Macular Edema

Conditions
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Diabetic Macular Edema

Intervention / Treatment
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* Genetic: FT-003


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Subjects that are willing and able to follow study procedures; Female or male patients ≥18 years old at the time of signing the ICF; Clinically diagnosed with CI-DME HbA1c≤10% The best corrected visual acuity (BCVA) of the studied eye is ≤ 73 letters Exclusion Criteria: Presence of any other intraocular diseases other than CI-DME in the studied eye that would affect the improvement of visual acuity and require treatment during the study for prevention or treatment of visual loss, as judged by the investigator

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Interventional Model Description: Central Involvement Diabetic Macular Edema
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: FT003 Dose 1<br>Low dose of FT-003 | Genetic: FT-003<br>* Administration via intraocular injection<br>|
| Experimental: FT003 Dose 2<br>Mid dose of FT-003 | Genetic: FT-003<br>* Administration via intraocular injection<br>|
| Experimental: FT003 Dose 3<br>High dose of FT-003 | Genetic: FT-003<br>* Administration via intraocular injection<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Safety and tolerability after FT-003 injection | Incidence and severity of AE (Common Terminology Criteria for Adverse Events 5.0) | Whthin 52 weeks after administration | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Preliminary effectiveness after FT-003 injection | Changes in best-corrected visual acuity (BCVA) of the studied eye from baseline | Whthin 52 weeks after administration |