4D-150 in Patients With Diabetic Macular Edema
Study Details
Study Description
Brief Summary
Phase 2 randomized, active-controlled, double-masked, dose-ranging trial in adults with Diabetic Macular Edema (DME).
Condition or Disease | Intervention/Treatment | Phase |
---|---|---|
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Phase 2 |
Detailed Description
This Phase 2 trial is a prospective, multicenter, randomized, active-controlled, double-masked dose-ranging trial to evaluate the safety and efficacy of 4D-150 in adults with DME. The trial will be conducted in two parts: Dose Confirmation and Dose Expansion.
Study Design
Arms and Interventions
Arm | Intervention/Treatment |
---|---|
Experimental: 4D-150 Part 1 Dose Confirmation Dose Level 1 4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day1. |
Biological: 4D-150 IVT
4D-150: AAV-based gene therapy comprised of miRNA targeting VEGF-C and codon-optimized sequence encoding aflibercept.
|
Experimental: 4D-150 Part 1 Dose Confirmation Dose Level 2 4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day1. |
Biological: 4D-150 IVT
4D-150: AAV-based gene therapy comprised of miRNA targeting VEGF-C and codon-optimized sequence encoding aflibercept.
|
Active Comparator: 4D-150 Part 1 Dose Confirmation Control Aflibercept will be administered PRN. |
Biological: Aflibercept IVT
Commercially available Active Comparator
Other Names:
|
Experimental: 4D-150 Part 2 Dose Expansion Dose Level 1 4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day1. |
Biological: 4D-150 IVT
4D-150: AAV-based gene therapy comprised of miRNA targeting VEGF-C and codon-optimized sequence encoding aflibercept.
|
Experimental: 4D-150 Part 2 Dose Expansion Dose Level 2 4D-150 will be administered at the assigned dose level as a single dose IVT injection on Day1. |
Biological: 4D-150 IVT
4D-150: AAV-based gene therapy comprised of miRNA targeting VEGF-C and codon-optimized sequence encoding aflibercept.
|
Active Comparator: 4D-150 Part 2 Dose Expansion Control Aflibercept at a fixed regimen will be administered. |
Biological: Aflibercept IVT
Commercially available Active Comparator
Other Names:
|
Outcome Measures
Primary Outcome Measures
- Annualized number of aflibercept injections in the study eye [52 weeks]
Secondary Outcome Measures
- Mean cumulative number of aflibercept injections over time [52 weeks]
- Change from baseline in BCVA as assessed using the ETDRS Visual Acuity Chart [104 weeks]
- Change from baseline in CST measured by spectral domain optical coherence tomography (SD-OCT) [104 weeks]
- Percentage of subjects with a ≥2 and ≥3-Step Diabetic Retinopathy Severity (DRS) improvement from baseline on the ETDRS-DRSS [104 weeks]
Other Outcome Measures
- Incidence and severity of treatment-emergent adverse events and serious adverse events, including clinically significant changes in safety parameters [104 weeks]
Eligibility Criteria
Criteria
Inclusion Criteria:
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≥18 years of age
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Type I or Type II diabetes mellitus with macular thickening secondary to DME involving the center of the fovea
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Demonstrate clinical response to on-study aflibercept injection in the study eye.
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Decreased visual acuity attributable primarily to DME
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BCVA in the study eye between 25 and 83 ETDRS letters, inclusive (~20/320 and 20/25, respectively) at Screening
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Study eye amenable to IVT injection
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Sufficient clear ocular media, pupil dilation and fixation in the study eye to permit adequate imaging; ability to perform tests of visual and retinal function and structure; and ability to comply with other protocol-specified procedures
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Provide written informed consent
Exclusion Criteria:
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Macular edema in the study eye considered to be secondary to a cause other than DME
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Systemic anti-VEGF treatment (e.g. sunitinib, bevacizumab, pazopanib) within 6 months, or anticipated need for systemic anti-VEGF therapy during study participation
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Systemic corticosteroids (oral, intravenous, intramuscular, intra-articular) or other immunosuppressive medications within 3 months
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Received an investigational drug, agent, device, or therapy (ocular or non-ocular) in the 6 months or at least 5 half-lives (whichever is longer) prior to Screening
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Prior gene therapy (ocular or non-ocular) and/or ocular stem cell therapy in either eye
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Any concurrent ocular condition in the study eye that is likely to require surgical intervention (e.g. cataract surgery) during the 2 year (104 week) study duration
Note: Other inclusion/exclusion criteria apply.
Contacts and Locations
Locations
No locations specified.Sponsors and Collaborators
- 4D Molecular Therapeutics
Investigators
- Study Director: Schonmei Lee, MD, 4D Molecular Therapeutics
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 4D-150-C002