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Safety and Tolerability of Anti-microRNA-328 Ophthalmic Solution, SHJ002, in Pediatric Subjects: First-in-Human
Jiunn-Liang Chen1, Wei-Yu Lai2, Reuy-Tay Lin3
1Department of Ophthalmology, Kaohsiung Veterans General Hospital, Kaohsiung, Taiwan; Center for General Education, Cheng Shiu University, Kaohsiung, Taiwan.
Purpose:
microRNA-328 has been reported as a risk factor for myopia development. SHJ002 is an antisense for microRNA-328, and SHJ002 was formulated as ophthalmic solution for a novel microRNA therapy. We aimed to investigate the safety and tolerability of SHJ002 ophthalmic solution in children.
Methods:
This was a single-center, open-label, first-in-human trial in healthy children (NCT04928144). All subjects received the study medication. The trial had 2 stages. Stage 1 was an intrasubject dose-escalation study, and stage 2 was the highest tolerable dose study. The SHJ002 ophthalmic solution was instilled in a randomly selected study eye in each participant, whereas the other untreated eye served as a negative control. Three participants were assigned to stage 1, and they received eye drops of 3 concentrations (0.025%, 0.08%, and 0.25%), each of which was used for 3 consecutive days. The highest tolerable dose from stage 1 was used in stage 2 where another 9 participants were recruited for 28-day treatment. Ocular assessments, physical examination, and vital signs were measured to evaluate safety and tolerability.
Findings:
There were 4 boys and 8 girls with a mean age of 12.3 years and a SD of 1.56. All participants were Asians. All 3 concentrations used in stage 1 were well tolerated, and the dose of 0.25% was used in stage 2. There were no reports of discomfort. There was only 1 mild adverse event (punctate keratitis) in the untreated eye in 1 participant, which was deemed as "unrelated to study drug."
Implications:
SHJ002 is a novel microRNA therapy that uses eye drop instillation. SHJ002 ophthalmic solution is generally safe and tolerable, which warrants further investigations in Phase II and III trials.
Clinicaltrials:
gov identifier: NCT04928144.
Insights
SHJ002 ophthalmic solution, a novel microRNA therapy for myopia, demonstrated excellent safety and tolerability in a pediatric clinical trial. Further research is warranted to confirm its efficacy in treating myopia.
Area of Science:
- Ophthalmology
- Genetics
- Pharmacology
Background:
- MicroRNA-328 is implicated as a risk factor in myopia development.
- SHJ002, an antisense agent targeting microRNA-328, has been developed as an ophthalmic solution for myopia therapy.
Purpose of the Study:
- To evaluate the safety and tolerability of SHJ002 ophthalmic solution in a pediatric population.
- To establish the highest tolerable dose for future clinical trials.
Main Methods:
- A single-center, open-label, first-in-human trial (NCT04928144) involving healthy children.
- Two-stage study: Stage 1 (dose escalation) and Stage 2 (highest tolerable dose for 28 days).
- Ocular assessments, physical examinations, and vital signs were monitored.
Main Results:
- All tested concentrations (0.025%, 0.08%, 0.25%) were well-tolerated in Stage 1.
- The 0.25% concentration was advanced to Stage 2 without reported discomfort.
- One mild, unrelated adverse event (punctate keratitis) occurred in an untreated eye.
Conclusions:
- SHJ002 ophthalmic solution is generally safe and well-tolerated in children.
- The findings support further investigation of SHJ002 in Phase II and III clinical trials for myopia.
- This novel microRNA therapy shows promise for myopia treatment via eye drop administration.
