Gene Therapy for Diabetic Macular Edema

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

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.

Study Overview

• Status: Recruiting
• Conditions: Diabetic Macular Edema
• Intervention / Treatment: Genetic: FT-003

• Study Type: Interventional
• Enrollment (Estimated): 18
• Phase: Phase 1

Contacts and Locations

• Study Contact: Name: Xinyan Li; Phone Number: +862158206061; Email: Xinyan.li@fronteratherapeutics.com
• Study Contact Backup: Name: Minghui Xue; Phone Number: +862158206061; Email: minghui.xue@fronteratherapeutics.com

Study Locations

• China
  • Tianjin,China
    • Tianjin, Tianjin,China, China, 300392
      • Recruiting
      • Tianjin Medical University Eye Hospital
      • Contact: Xiaorong Li, Professor

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Adult; Older Adult
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Non-Randomized
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

3

Arms and Interventions

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

What is the study measuring?

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

Collaborators and Investigators

• Sponsor: Frontera Therapeutics
• Collaborators: Peking Union Medical College Hospital; The First Affiliated Hospital of Zhengzhou University; Tianjin Medical University Eye Hospital
• Investigators: Principal Investigator: Xiaorong Li, Professor, Tianjin Medical University Eye Hospital; Principal Investigator: Hanyi Min, Professor, Peking Union Medical College Hospital; Principal Investigator: Guangming Wan, Professor, The First Affiliated Hospital of Zhengzhou University

Study record dates

Study Major Dates

• Study Start (Actual): May 19, 2023
• Primary Completion (Estimated): May 1, 2025
• Study Completion (Estimated): May 1, 2028

Study Registration Dates

• First Submitted: June 14, 2023
• First Submitted That Met QC Criteria: June 14, 2023
• First Posted (Actual): June 23, 2023

Study Record Updates

• Last Update Posted (Actual): June 23, 2023
• Last Update Submitted That Met QC Criteria: June 14, 2023
• Last Verified: April 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Eye Diseases; Retinal Degeneration; Retinal Diseases; Macular Degeneration; Macular Edema; Edema
• Other Study ID Numbers: FT003DM-1

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No