CHAMP Phase 3 trial: Efficacy of NVK002 for controlling pediatric myopia over 3 years

(Image Credit: AdobeStock/motortion)

Reviewed by Rupa K. Wong, MD, FAAP

The CHAMP phase 3 study showed that NVK002 atropine 0.01% (Vyluma Inc.) “meaningfully slowed” myopia progression in myopic pediatric patients over a 36-month period,¹ according to Rupa K. Wong, MD, who presented the study findings at the annual meeting of the American Association for Pediatric Ophthalmology and Strabismus.

Myopia has become a global public health threat and extensive efforts are underway worldwide to identify treatments to control progression. The use of atropine in various concentrations has been evaluated in a number of studies.^2,3

Wong explained that in the US, the treatment of myopia with low-dose atropine is an off label use of the drug, which must be compounded at a specialty pharmacy. This results in variability in the concentration, pH, osmolarity, and viscosity of the compounded low-dose formulation. These drawback emphasize the need for a shelf-stable, preservative-free, Good Manufacturing Practice (GMP)-grade low-dose atropine option for treatment of myopia progression in pediatric patients, she emphasized.

NVK002

This formulation of low-dose atropine is currently under development to treat myopia progression in children.

It has a number of advantages in that the formulation is shelf-stable at room temperature for at least 24 months; it is manufactured in accordance with GMP regulations and meets the quality standards for identity, strength, purity, and consistency; it is preservative-free and suitable for chronic administration; it is packaged in sterile, single-dose ampules, and it is formulated with standard topical ophthalmic excipients, Wong enumerated.

CHAMP Study

This is the first placebo-controlled 3-year, phase 3 trial in US and European patients, the goal of which was to determine the safety and efficacy of at least 1 dose of NVK002 for treating myopia progression in pediatric patients. The study evaluated 2 doses, 0.01% and 0.02% in 164 and 247 children, respectively.

The study participants were 3 to 16 years of age and had a spherical equivalent refractive error (SER) ranging from -0.5 to -6.0 diopters (D) in each eye, low astigmatism, and distance vision that was correctable to at least 20/25 in each eye.

The investigators assessed the effects of the 2 doses of NVK002 using 3 measures, ie, the mean change from baseline in the axial length, the mean change from the baseline SER, and the proportion of patients who had myopia progression less than 0.5 SER.

CHAMP results

Wong reported that NVK002 0.01% demonstrated efficacy across all key endpoints at 36 months.

The mean difference in the axial length between the patients treated actively and those that received vehicle was -0.130 mm (95% confidence [CI], -0.191, -0.069) p < 0.001).

The mean change in the refractive error between those treated actively and those who received vehicle was a difference of 0.240 D (95% CI, -0.106, 0.374; p < 0.001)

More patients actively treated with NVK002 had myopia progression less than 0.5 D compared with vehicle (p = 0.031).

The results suggested that 3 years of dosing with the lower dose of NVK002, 0.01%, provides myopia control.

The higher dose of NVK002, 0.02%, achieved significant and clinically meaningful differences from the vehicle only in the axial length.

A consideration

Wong pointed out that an important effect that needs to be determined is whether tachyphylaxis occurs with chronic dosing of NVK002. Thus far, the drug’s efficacy has been maintained out to 3 years. Additional studies are needed to determine the treatment benefits of low-dose atropine for longer than 3 years.

References:

1. Zadnik K, Schulman E, Flitcroft I, et al.; CHAMP Trial Group Investigators. Efficacy and safety of 0.01% and 0.02% atropine for the treatment of pediatric myopia progression over 3 years: a randomized clinical trial. JAMA Ophthalmol. 2023;141:990-999.

2. Yam JC, Jiang Y, Tang SM, et al. Low-Concentration Atropine for Myopia Progression (LAMP) study: a randomized, double-blinded, placebo-controlled trial of 0.05%, 0.025%, and 0.01% atropine eye drops in myopia control. Ophthalmology. 2019;126:113-124.

3. Li FF, Zhang Y, Zhang X, et al. Age effect on treatment responses to 0.05%, 0.025%, and 0.01% atropine: Low-Concentration Atropine for Myopia Progression Study. Ophthalmology. 2021; DOI:10.1016/j.ophtha.2020.12.036

4. Rong RK. The efficacy of NVK002 in treating pediatric myopia progression is maintained each year over 36 months in the CHAMP phase 3 trial. Presented at the 49th annual meeting of the American Association for Pediatric Ophthalmology and Strabismus, April 7-11, 2024, Austin Texas. Scientific session: Myopia