Safety and efficacy of sodium phenylbutyrate eye drops for paediatric myopia progression

The primary objective is to evaluate the ocular safety of T10430 in paediatric myopia, which is clinically relevant for assessing tolerability of an ophthalmic treatment intended for use in a pediatric population. The secondary objectives are to evaluate preliminary efficacy data of T10430 and to further assess its safety, providing additional information on both potential clinical effect and safety profile.

The trial population comprised 180 participants with paediatric myopia. The study included male and female participants aged ≥ 6 and < 12 years, and the population was described as vulnerable. Participants were selected from patients meeting the specified entry requirements, including signed and dated informed consent, a spherical equivalent refractive error between -1.00 D and -6.00 D in each eye, intraocular pressure below 21 mmHg in each eye, and distant best-corrected visual acuity of at least 0.1 LogMAR in each eye. No information was provided on general health status beyond these criteria, and no lifestyle considerations such as diet, physical activity, or habits were reported.

The trial is a randomized, controlled study evaluating the safety and efficacy of T10430 eye drops in paediatric myopia. The investigational treatment is sodium phenylbutyrate administered by ocular use, with a preservative-free ophthalmic solution used as placebo. The overall trial duration is planned from July 2026 to November 2027. Participant involvement is expected to last up to 12 months. The study sequence includes an inclusion screening visit to confirm eligibility criteria, followed by post-baseline follow-up visits at 1 month, 6 months, and 12 months, with the 12-month assessment serving as the end-of-study visit. These visits are used to assess ocular safety, treatment-emergent adverse events, visual function, ocular surface findings, intraocular pressure, and structural eye measurements. Early termination may occur for ocular or systemic treatment-emergent adverse events, serious adverse events, events related to the investigational product, or adverse events leading to premature discontinuation of the investigational product.

The investigational treatment consisted of sodium phenylbutyrate administered as eye drops, solution in single-dose container for ocular use. The study product was given by the ocular route. The source data specify a dose of 00 µl microlitre(s) and do not provide a frequency of administration. Three test product entries were listed for the study.

The comparator treatment was a preservative-free, ophthalmic solution used as placebo. The source data do not specify a pharmaceutical form beyond ophthalmic solution, nor do they provide route, dose, or dosing frequency. No additional information on drug administration, dosing schedule, or participant compliance monitoring is provided in the source data.

Efficacy will be assessed by change from baseline in axial length at 6 months and 12 months in both eyes, and by change from baseline in cycloplegic spherical equivalent refractive error at 6 months and 12 months in both eyes. Additional efficacy assessments will include change from baseline in distant and near best-corrected visual acuity at 1 month, 6 months, and 12 months in both eyes, as well as changes from baseline in conjunctival hyperemia, corneal fluorescein staining, conjunctival fluorescein staining, tear break-up time, intraocular pressure, central corneal thickness, thinnest corneal thickness, biometry keratometry anterior chamber depth, lens thickness, K1, K2, Kmax, central corneal endothelial cell density, hexagonality ratio of central corneal endothelial cells, and coefficient of variation of central corneal endothelial cell size at the specified post-baseline timepoints. Ocular symptoms will be evaluated throughout the day and immediately after instillation, with scores and changes from baseline recorded at all visits and phone calls. Ocular tolerance will be assessed by the investigator at 1 month, 6 months, and 12 months, and by the participant or relatives at all post-baseline visits and phone calls. Global satisfaction with the use of the single-dose container will also be assessed by the participant or legal guardian(s) at all post-baseline visits and phone calls.