CHAMP: Study of NVK-002 in Children With Myopia

Study Overview
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Brief Summary
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Stage 1: To evaluate the safety and efficacy of 2 concentrations of NVK-002 compared to Vehicle (placebo) for slowing the progression of myopia in children over a 3 year treatment period. Stage 2: To observe safety and efficacy in subjects re-randomized to one (1) year of treatment with NVK-002 or Vehicle following 3 years of treatment in children with progressive myopia.

Detailed Description
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This will be a 3-arm randomized, multicenter, double-masked, placebo-controlled study conducted in 2 stages. Stage 1 is a safety and efficacy phase of 3 years in duration, during which subjects will be allocated to 1 of 3 study medications. Stage 2 is a randomized cross-over phase of 1 year in duration, during which subjects will be re-randomized to receive 1 of the 3 study medications, with subjects initially randomized to Vehicle only eligible for randomization to low or high-dose NVK-002. Treatment arms are: NVK-002 low dose concentration NVK-002 high dose concentration Vehicle (placebo)

Official Title
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A 3-Arm Randomized, Double-Masked, Placebo-Controlled, Phase 3 Study of NVK-002 in Children With Myopia

Conditions
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Myopia

Intervention / Treatment
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* Drug: NVK-002 Concentration 1
* Drug: NVK-002 Concentration 2
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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Inclusion Criteria: Children aged 3 to ≤ 17.0 years. Myopia SER of at least -0.50 D and no greater than -6.00 D myopia in each eye as measured by cycloplegic autorefraction. Exclusion Criteria: If present, astigmatism more than -1.50 D in either eye. Current or history of amblyopia or strabismus. History of any disease or syndrome that predisposes the subject to severe myopia (e.g., Marfan syndrome, Stickler syndrome, retinopathy of prematurity). History in either eye of abnormal ocular refractive anatomy (e.g., keratoconus, lenticonus, spherophakia). Serious systemic illness that, in the Investigator's opinion, would render the subject ineligible. Chronic use (more than 3 days per week) of any topical ophthalmic medications (prescribed or over the-counter) other than the assigned study medication.

Ages Eligible for Study
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Minimum Age: 3 Years
Maximum Age: 17 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: Crossover Assignment
Interventional Model Description: This will be a 3-arm randomized, multicenter, double-masked, placebo-controlled study conducted in 2 stages. Stage 1 is a safety and efficacy phase of 3 years in duration, during which subjects will be allocated to 1 of 3 study medications. Stage 2 is a randomized cross-over phase of 1 year in duration, during which subjects will be re-randomized to receive 1 of the 3 study medications with subjects initially randomized to Vehicle only eligible for randomization to low or high dose NVK-002.
Masking: Double


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: NVK-002 Concentration 1<br>Stage 1: Subjects will be randomized to NVK-002 Concentration 1 Stage 2: Subjects will be re-randomized to one of the three treatment arms. | Drug: NVK-002 Concentration 1<br>* Study medication will be administered, once daily (QD)<br>* Other names: NVK-002 Conc 1;|
| Experimental: NVK-002 Concentration 2<br>Stage 1: Subjects will be randomized to NVK-002 Concentration 2 Stage 2: Subjects will be re-randomized to one of the three treatment arms. | Drug: NVK-002 Concentration 2<br>* Study medication will be administered, once daily (QD)<br>* Other names: NVK-002 Conc 2;|
| Placebo Comparator: Vehicle (Placebo)<br>Stage 1: Subjects will be randomized to Vehicle (Placebo) Stage 2: Subjects will be re-randomized to one of the two experimental NVK-002 treatment arms | Drug: Placebo<br>* Vehicle (placebo) will be administered, once daily (QD)<br>* Other names: Vehicle;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| The overall between-group difference in proportion of subjects who show < -0.50 D myopia progression (SER) at the Month 36 visit. | The overall between-group difference in proportion of subjects who show < -0.50 D myopia progression (SER) at the Month 36 visit. | 36 Months | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Between-group difference in mean progression rates. | Between-group difference in mean progression rates. | Month 12, Month 24, Month 36 | 
| Between-group difference in proportion of subjects who show <-0.75 D progression and the between-group median time to a change in myopia of <-0.75 D. | Between-group difference in proportion of subjects who show <-0.75 D progression and the between-group median time to a change in myopia of <-0.75 D. | Month 36 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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child, myopia, nearsightedness