Pediatric myopia is rising globally, with high myopia associated with sight-threatening complications later in life. Low-dose atropine has been shown to slow myopia progression, with most studies conducted in Asian populations using compounded formulations. We evaluated the efficacy and safety of atropine sulfate ophthalmic solution at two concentrations for slowing myopia progression in a predominantly white population. STAR was a randomized, double-masked, vehicle-controlled, phase 3 clinical study conducted in Europe and the USA. Children (N = 847) aged 3–14 years with myopia between − 0.50 and − 6.00 diopters (D) were dosed after 1:1:1 randomization to vehicle, atropine sulfate 0.01% or 0.03% once daily before bedtime for 48 months. The primary endpoint was mean annual progression rate (APR) of myopia, measured by cycloplegic autorefraction, through month 24. Mean APR at 24 months was − 0.44 D/year (vehicle), − 0.31 D/year (atropine sulfate 0.01%), and − 0.32 D/year (atropine sulfate 0.03%), with differences versus vehicle of 0.13 D (95% CI 0.06–0.20; p = 0.0003) and 0.12 D (95% CI 0.05–0.19; p = 0.0009), respectively. In Fast Progressor Subgroup 1 (progression − 0.50 D/year or worse at baseline), differences versus vehicle were 0.21 D (p < 0.0001) for atropine sulfate 0.01% and 0.15 D (p = 0.0023) for atropine sulfate 0.03%. In Fast Progressor Subgroup 2 (progression − 0.75 D/year or worse at baseline), differences versus vehicle were 0.19 D (p = 0.0008) for atropine sulfate 0.01% and 0.11 D (p = 0.0397) for atropine sulfate 0.03%. TEAEs were similar across groups. Photophobia was the most common ocular event (16.7% vehicle, 24.1% atropine sulfate 0.01%, 30.4% atropine sulfate 0.03%). Treatment discontinuation was similar across groups (18.7% vehicle, 19.7% atropine sulfate 0.01%, 18.6% atropine sulfate 0.03%). Atropine sulfate 0.01% and 0.03% effectively slowed myopia progression in children, with greatest efficacy in Fast Progressors Subgroup 1. Both concentrations were well tolerated with manageable side effects, supporting atropine sulfate as a treatment option for pediatric myopia. Clinical Trial Registration: ClinicalTrials.gov identifier NCT03918915. Myopia (near-sightedness) is becoming increasingly common worldwide, especially in children. When left untreated, it can worsen over time and lead to serious eye complications later in life. This study tested whether low-dose atropine sulfate eye drops could safely slow myopia worsening (progression) in children aged 3–14 years. The STudy of Atropine to Reduce (STAR) myopia progression was a randomized study of atropine sulfate 0.01% and 0.03%. A total of 847 participants received either atropine sulfate eye drops (at concentrations of 0.01% or 0.03%) or a vehicle (eye drops without active medication) once daily before bedtime for 48 months. Results showed that both atropine concentrations significantly slowed myopia progression compared with vehicle. Children receiving atropine 0.01% had 0.13 diopters less myopia worsening at 2 years than those receiving vehicle, while those using atropine 0.03% had 0.12 diopters less worsening. The treatment worked particularly well for children who had a history of faster myopia progression (− 0.50 and − 0.75 diopters per year or worse). Both atropine concentrations were well tolerated, with few children stopping treatment due to side effects. The most common side effect across all groups was increased sensitivity to light, which was generally mild. On the basis of these results, atropine 0.01% received marketing authorization by the European Medicines Agency on June 2, 2025, for slowing myopia progression in children. This study provides important evidence that atropine eye drops can be an effective option for managing childhood myopia, potentially reducing the risk of serious eye complications later in life.
Korenfeld et al. (Mon,) studied this question.