Myopia
Conditions
Keywords
child, myopia, nearsightedness
Brief summary
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
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 follow-up 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)
Interventions
DRUGNVK-002 Concentration 1
Study medication will be administered, once daily (QD)
DRUGNVK-002 Concentration 2
Study medication will be administered, once daily (QD)
DRUGPlacebo
Vehicle (placebo) will be administered, once daily (QD)
Sponsors
Syneos Health
Vyluma, Inc.
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
DOUBLE (Subject, Investigator)
Masking description
If subjects meet eligibility criteria at the Screening/Baseline visit (Day 0), subjects will be randomly assigned to masked study medication. The study will be double masked. The study medication will be provided in identical-appearing laminated pouches with no labeling indicating the identity of the study group or the contents of the ampules. The laminated pouches will contain identical-appearing ampules. Study subjects and study management personnel will be masked to the identity of treatment until after the final database lock.
Intervention model description
This will be a 3-arm randomized, multicenter, double-masked, placebo-controlled study conducted in 2 stages. Stage 1 is to assess the primary and secondary endpoints. Stage 2 is an extension stage for exploratory endpoints. 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.
Eligibility
Sex/Gender
ALL
Age
3 Years to 17 Years
Healthy volunteers
No
Inclusion criteria
1. Children aged 3 to ≤ 17.0 years. 2. Myopia SER of at least -0.50 D and no worse than -6.00 D myopia in each eye as measured by cycloplegic autorefraction.
Exclusion criteria
1. If present, astigmatism more than -1.50 D in either eye. 2. Current or history of amblyopia or strabismus. 3. History of any disease or syndrome that predisposes the subject to severe myopia (e.g., Marfan syndrome, Stickler syndrome, retinopathy of prematurity). 4. History in either eye of abnormal ocular refractive anatomy (e.g., keratoconus, lenticonus, spherophakia). 5. Serious systemic illness that, in the Investigator's opinion, would render the subject ineligible. 6. Chronic use (more than 3 days per week) of any topical ophthalmic medications (prescribed or over the-counter) other than the assigned study medication.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Number of Subjects' Eyes That Show < 0.50 D Myopia Progression (SER, Spherical Equivalent Refraction) at the Month 36 Visit | 36 Months | Number of subjects' eyes that show \< 0.50 D myopia progression (SER, spherical equivalent refraction) at the Month 36 visit. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Change From Baseline in SER (Spherical Equivalent Refraction) | Month 36 | Change from baseline in SER (spherical equivalent refraction). |
Countries
Hungary, Ireland, Netherlands, Spain, United Kingdom, United States
Participant flow
Pre-assignment details
Enrolled subjects who did not meet the inclusion criteria or met the exclusion criteria were not randomized.
Participants by arm
| Arm | Count |
|---|---|
| NVK-002 Concentration 1 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.
NVK-002 Concentration 1: Study medication will be administered, once daily (QD) | 164 |
| NVK-002 Concentration 2 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.
NVK-002 Concentration 2: Study medication will be administered, once daily (QD) | 247 |
| Vehicle (Placebo) 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
Placebo: Vehicle (placebo) will be administered, once daily (QD) | 165 |
| Total | 576 |
Withdrawals & dropouts
| Period | Reason | FG000 | FG001 | FG002 |
|---|---|---|---|---|
| Stage 2 | Adverse Event | 0 | 1 | 1 |
| Stage 2 | Lack of Efficacy | 0 | 2 | 0 |
| Stage 2 | Lost to Follow-up | 1 | 2 | 2 |
| Stage 2 | unable to visit (1); used prohibited concomitant medication / withdrew consent (4); relocation (1). | 2 | 4 | 0 |
Baseline characteristics
| Characteristic | Vehicle (Placebo) | Total | NVK-002 Concentration 1 | NVK-002 Concentration 2 |
|---|---|---|---|---|
| Age, Continuous | 8.8 years STANDARD_DEVIATION 1.8 | 8.9 years STANDARD_DEVIATION 2 | 9.0 years STANDARD_DEVIATION 2.1 | 9.0 years STANDARD_DEVIATION 2.1 |
| Race (NIH/OMB) American Indian or Alaska Native | 1 Participants | 7 Participants | 3 Participants | 3 Participants |
| Race (NIH/OMB) Asian | 26 Participants | 109 Participants | 29 Participants | 54 Participants |
| Race (NIH/OMB) Black or African American | 27 Participants | 81 Participants | 23 Participants | 31 Participants |
| Race (NIH/OMB) More than one race | 8 Participants | 30 Participants | 15 Participants | 7 Participants |
| Race (NIH/OMB) Native Hawaiian or Other Pacific Islander | 3 Participants | 4 Participants | 1 Participants | 0 Participants |
| Race (NIH/OMB) Unknown or Not Reported | 7 Participants | 36 Participants | 9 Participants | 20 Participants |
| Race (NIH/OMB) White | 93 Participants | 309 Participants | 84 Participants | 132 Participants |
| Region of Enrollment Europe | 29 participants | 111 participants | 34 participants | 48 participants |
| Region of Enrollment United States | 136 participants | 465 participants | 130 participants | 199 participants |
| SER | -2.41 Diopter | -2.33 Diopter | -2.25 Diopter | -2.33 Diopter |
| Sex: Female, Male Female | 94 Participants | 315 Participants | 85 Participants | 136 Participants |
| Sex: Female, Male Male | 71 Participants | 261 Participants | 79 Participants | 111 Participants |
Adverse events
| Event type | EG000 affected / at risk | EG001 affected / at risk | EG002 affected / at risk | EG003 affected / at risk | EG004 affected / at risk | EG005 affected / at risk |
|---|---|---|---|---|---|---|
| deaths Total, all-cause mortality | 0 / 164 | 0 / 247 | 0 / 162 | 0 / 159 | 0 / 162 | 0 / 99 |
| other Total, other adverse events | 56 / 164 | 92 / 247 | 75 / 162 | 43 / 159 | 29 / 162 | 17 / 99 |
| serious Total, serious adverse events | 1 / 164 | 8 / 247 | 4 / 162 | 3 / 159 | 2 / 162 | 2 / 99 |
Outcome results
Primary
Number of Subjects' Eyes That Show < 0.50 D Myopia Progression (SER, Spherical Equivalent Refraction) at the Month 36 Visit
Number of subjects' eyes that show \< 0.50 D myopia progression (SER, spherical equivalent refraction) at the Month 36 visit.
Time frame: 36 Months
Population: ITT
| Arm | Measure | Value (NUMBER) |
|---|---|---|
| NVK-002 Concentration 1 | Number of Subjects' Eyes That Show < 0.50 D Myopia Progression (SER, Spherical Equivalent Refraction) at the Month 36 Visit | 79 participants' eyes |
| NVK-002 Concentration 2 | Number of Subjects' Eyes That Show < 0.50 D Myopia Progression (SER, Spherical Equivalent Refraction) at the Month 36 Visit | 106 participants' eyes |
| Vehicle (Placebo) | Number of Subjects' Eyes That Show < 0.50 D Myopia Progression (SER, Spherical Equivalent Refraction) at the Month 36 Visit | 61 participants' eyes |
Secondary
Change From Baseline in SER (Spherical Equivalent Refraction)
Change from baseline in SER (spherical equivalent refraction).
Time frame: Month 36
Population: ITT
| Arm | Measure | Value (MEDIAN) |
|---|---|---|
| NVK-002 Concentration 1 | Change From Baseline in SER (Spherical Equivalent Refraction) | -0.85 Diopter |
| NVK-002 Concentration 2 | Change From Baseline in SER (Spherical Equivalent Refraction) | -1.02 Diopter |
| Vehicle (Placebo) | Change From Baseline in SER (Spherical Equivalent Refraction) | -1.10 Diopter |