Study of OT-101 in Treating Myopia

Study Overview
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Brief Summary
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This is a phase III, randomized, double-masked, placebo-controlled, parallel-group, multicenter safety, tolerability, and efficacy study of atropine sulfate 0.01% (OT-101) as an investigational treatment for myopia in pediatric subjects

Detailed Description
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This is a phase III, randomized, double-masked, placebo-controlled, parallel-group, multi-center study to evaluate the safety tolerability and efficacy of OT-101 (Atropine 0.01%) Ophthalmic Solution in treating the progression of myopia in pediatric subjects that will enroll approximately 678 pediatric subjects with myopia in total. Subjects will be stratified by age and refractive error. This study consists of 11 visits over the course of approximately 4 years and can be broken down in the 3 stages: Screening (Days -14 to -1), Stage 1 (Day 1 to Year 3), and Stage 2 (Year 3 to Year 4). In stage 1, subjects will be randomized in a 2:1 manner to OT-101 Ophthalmic Solution: Placebo. Subjects will be dosed with one drop of study treatment per eye once daily at bedtime for 3 years. In stage 2, subjects will be re-randomization 1:1 will occur at Visit 9 (Month 36). At re-randomization, subjects who had been assigned to OT-101 Ophthalmic Solution will be randomized in a 1:1 ratio to continue on OT-101 Ophthalmic Solution or switch to placebo. Subjects who had been assigned to placebo will continue with placebo.

Official Title
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A Phase III, Randomized, Double-masked, Placebo-controlled, Parallel-group, Multicenter Study of the Safety and Efficacy of OT-101 (Atropine Sulfate 0.01%) in Treating the Progression of Myopia in Pediatric Subjects

Conditions
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Myopia, Progressive

Intervention / Treatment
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* Drug: OT-101 Ophthalmic Solution
* Drug: Vehicle


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Have refractive error by cycloplegic autorefraction at baseline (Visit 1) in the study eye of: myopia greater or equal to -1.00D of spherical equivalent astigmatism less than or equal to 1.50DC progression of at least -0.50D of spherical equivalent in the last 12 months; Exclusion Criteria: Have active or a history of chronic or recurrent episodes of ocular inflammation (e.g. moderate to severe blepharitis, allergic conjunctivitis, peripheral ulcerative keratitis, scleritis) in either eye; Have undergone any myopia control treatment including orthokeratology, rigid gas-permeable contact lenses, bifocal contact lenses, progressive addition spectacle lenses, or other lenses to reduce myopia progression in the previous 6 months. Myopic correction in the form of single-vision eyeglasses and/or single-vision soft contact lenses are allowed; Have undergone any form of refractive eye surgery including incisional keratotomy, photorefractive keratectomy [PRK], laser in situ keratomileusis [LASIK], laser-assisted sub- epithelial keratectomy [LASEK]), corneal inlay procedures, conductive keratoplasty, small incision lenticule extraction (SMILE), cataract extraction, or any form of intraocular lens implantation;

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 15 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
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: OT-101 alone<br>Atropine Sulfate 0.01% Ophthalmic Solution through year 4 | Drug: OT-101 Ophthalmic Solution<br>* Atropine 0.01%<br>* Other names: Investigational Product;|
| Experimental: OT-101 plus vehicle<br>Atropine Sulfate 0.01% Ophthalmic Solution through year 3 followed by vehicle for 1 year | Drug: OT-101 Ophthalmic Solution<br>* Atropine 0.01%<br>* Other names: Investigational Product;Drug: Vehicle<br>* Investigational Product minus active ingredient<br>* Other names: Placebo;|
| Placebo Comparator: Vehicle<br>Vehicle (Investigational Product minus active ingredient) through year 4 | Drug: Vehicle<br>* Investigational Product minus active ingredient<br>* Other names: Placebo;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Percentage of study eyes with a -0.75D of progressive myopia defined as an increase in spherical equivalent of -0.75D or greater as assessed by cycloplegic autorefraction. | Efficacy of OT-101 Ophthalmic Solution | At month 36 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in spherical equivalent (D) in the study eye as assessed by cycloplegic autorefraction | Efficacy of OT-101 Ophthalmic Solution | Baseline to Month 36 | 
| Change in study eye axial length as measured by cycloplegic biometry (a standardized device will be selected for this study) | Efficacy of OT-101 Ophthalmic Solution | Baseline to Month 36 |