The CHAMP clinical trial and NVK002 for controlling paediatric myopia

Thus far, the drug’s efficacy has been maintained out to 3 years, but additional studies are needed. Image credit: ©Mangostar – stock.adobe.com

Findings from the CHAMP phase 3 study (NCT03350620) show that NVK002 atropine 0.01% (Vyluma, Inc) “meaningfully slowed” myopia progression in paediatric patients with myopia over a 36-month period,¹ according to Rupa K. Wong, MD, FAAP. Wong presented the study findings at the 49th Annual Meeting of the American Association for Pediatric Ophthalmology and Strabismus in Austin, Texas.

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 several studies.^2,3

Wong explains 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 drawbacks emphasise the need for a shelf-stable, preservative-free, Good Manufacturing Practices (GMP)–grade, low-dose atropine option for treatment of myopia progression in paediatric patients, she says.

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 long-term administration; it is packaged in sterile, single-dose ampoules; and it is formulated with standard topical ophthalmic excipients, Wong explains.

The CHAMP study

CHAMP was 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 one dose of NVK002 for treating myopia progression in paediatric patients. The study evaluated 2 doses, 0.01% and 0.02%, in 164 and 247 children, respectively.

The study participants were aged 3 to 16 years and had a spherical equivalent refractive (SER) error ranging from –0.5 D to –6 D in each eye, low astigmatism, and distance vision that was correctable to at least 20/25 visual acuity in each eye. The investigators assessed the effects of the 2 doses of NVK002 using mean change from baseline in axial length, mean change from the baseline SER, and the proportion of patients who had myopia progression less than 0.5 SER.

CHAMP results

Wong reports that NVK002 0.01% demonstrated efficacy across all key end points at 36 months. The mean difference in the axial length between the patients treated actively and those who received the vehicle was –0.13 mm (95% CI, –0.191 to –0.069; P < .001).

The mean change in the refractive error between those treated actively and those who received the vehicle was a difference of 0.24 D (95% CI, –0.106 to 0.374; P < .001). More patients actively treated with NVK002 had myopia progression less than 0.5 D compared with those who received the vehicle (P = .031).

The results suggest that 3 years of dosing with the lower dose of NVK002, 0.01%, provides myopia control. The 0.02% dose of NVK002 achieved significant and clinically meaningful differences from the vehicle only in the axial length.

A consideration

Wong points out that an important effect that needs to be determined is whether tachyphylaxis occurs with long-term dosing of NVK002. Thus far, the drug’s efficacy has been maintained out to 3 years. She notes that 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(10):990-999. doi:10.1001/jamaophthalmol.2023.2097

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(1):113-124. doi:10.1016/j.ophtha.2018.05.029

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;128(8):1180-1187. doi:10.1016/j.ophtha.2020.12.036

Rupa Wong, MD, FAAP | E: rupawong@honolulueyeclinic.com

Wong is from the Honolulu Eye Clinic in Hawaii and the John A. Burns School of Medicine at the University of Hawaii at Mānoa in Honolulu. She is a consultant/adviser to Vyluma, Inc. The CHAMP study was sponsored by Vyluma, Inc. This article is based on Wong's presentation, “The Efficacy of NVK002 in Treating Paediatric Myopia Progression Is Maintained Each Year Over 36 Months in the CHAMP Phase 3 Trial,” given at the 49th Annual Meeting of the American Association for Pediatric Ophthalmology and Strabismus, 7 to 11 April, 2024, in Austin, Texas.