Phase 2 Spectra Study to Evaluate the Safety and Efficacy of OPL-0401 in Patients With Diabetic Retinopathy

Study Overview
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Brief Summary
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OPL-0401-201 is a multicenter study to investigate the safety and efficacy of OPL-0401 in patients with diabetes mellitus (DM) with diabetic retinopathy.

Detailed Description
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OPL-0401-201 is a randomized, double-masked, placebo-controlled, multicenter study to investigate the efficacy and safety of OPL-0401 in patients with diabetes mellitus (DM) with NPDR or mild PDR with or without diabetic macular edema (DME).

Official Title
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Phase 2 Spectra Study to Evaluate the Safety and Efficacy of OPL-0401 in Patients With Diabetic Retinopathy

Conditions
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Non-proliferative Diabetic Retinopathy, Proliferative Diabetic Retinopathy

Intervention / Treatment
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* Drug: OPL-0401 Dose 1
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria Adults ≥ 18 years; Diabetes mellitus (type 1, type 2 or other forms); Females who are not a woman of childbearing potential (WOCBP) or who agree to use contraception; At least one eye with moderately severe to severe NPDR (DRSS levels 47 or 53) or mild PDR (DRSS level 61); Patients with or without diabetic macular edema (DME) may be eligible if they meet protocol specified eligibility criteria; Best corrected visual acuity (BCVA) early treatment of diabetes retinopathy study (ETDRS) letter score in the study at screening ≥69 letters (approximate Snellen equivalent of 20/40 or better) without CI-DME, or ≥75 letters when CI-DME is present (approximate Snellen equivalent 20/32 or better); Anti-vascular endothelial growth factor (VEGF) or any laser treatment is not required nor anticipated in the study eye for least 6 months. Exclusion Criteria: Body mass index ≥ 45 kg/m2 Uncontrolled diabetes mellitus such as hemoglobin A1c (HbA1C) > 11% or patients who are not currently treated for their diabetes; Uncontrolled hypertension defined as systolic > 160mmHg or diastolic > 100 mmHg (despite hypertensive medication); Proliferative Diabetes Retinopathy (PDR) with the exception of mild PDR (DRSS 61); Evidence of retinal neovascularization (with the exception of mild PDR); Any previous Diabetic Retinopathy treatment with focal or grid laser photocoagulation or Pan-Retinal Photocoagulation (PRP); History of previously treated DME with fluocinolone acetonide implant (Iluvien®) injection; Visual acuity loss due to an ocular condition that would not improve from treatment of DR or resolution of DME (i.e., foveal atrophy, pigment abnormalities, dense subfoveal hard exudates, nonretinal condition); History of vitreoretinal surgery; Intraocular surgery in the study eye within 3 months of randomization or anticipated over the course of the study; Uncontrolled glaucoma (e.g. visual field loss or defined as (IOP) ≥ 25 mmHg despite treatment with anti-glaucoma medication); Evidence of active infectious blepharitis, keratitis, scleritis, or conjunctivitis in either eye /any intraocular inflammation or infection in either eye within 3 months prior to randomization.

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: Randomized
Intervention Model: Parallel Assignment
Interventional Model Description: This study is a randomized, double-masked, placebo-controlled, multicenter study to investigate the efficacy and safety of OPL-0401 in patients with diabetes mellitus (DM) with non-proliferative diabetic retinopathy (NPDR) or mild PDR with or without diabetic macular edema (DME). Data from an interim analysis may be used to consider additional arms in the study.
Masking: Quadruple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: OPL-0401 Dose 1<br>Participants are randomized to OPL-0401 Dose 1 twice daily for 24 weeks | Drug: OPL-0401 Dose 1<br>* Pharmaceutical Form: Capsule; Route of Administration: Oral<br>|
| Placebo Comparator: Placebo<br>Participants are randomized to matching Placebo twice daily for 24 weeks | Drug: Placebo<br>* Pharmaceutical Form: Capsule; Route of Administration: Oral<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Improvement in Diabetic Retinopathy Severity Scale (DRSS) score | Proportion of patients with a ≥2-step improvement from baseline in DRSS | 24 weeks/168 days | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Proportion of patients with an improvement or worsening in DRSS | Proportion of patients with an improvement or worsening from baseline in DRSS of ≥ 1, ≥ 2 and ≥ 3 steps | 12 Weeks/84 days and 24 Weeks/168 days | 
| Safety and tolerability | Incidence of Adverse event (AE) and serious adverse events (SAE) | 198 days | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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diabetic retinopathy, diabetic macular edema, Non-proliferative Diabetic Retinopathy, Proliferative Diabetic Retinopathy