Table 1.

Study schedule for patients participating from phase I

	Entry	Study period												Follow-up	Cessation
Visit	V1*	V2	V3	V4	V5	V6	V7	V8	V9	V10	V11	V12	V13	V14	–
Timing (D=day, W=week)	D0	D1	D2	D4	D7	D8	W2	W3	W4	W6	W8	W10	W12	W16	–
Entry
Informed consent	○
Eligibility screen	○	○
Registration		○
Intervention
Phase I		○	○	○	○†	○	○	○
Phase II								○‡	○	○	○	○	○
Assessments
Vital signs	○	○	○	○	○	○	○	○	○	○	○	○	○	○	○
Height, weight and head circumference	○								○		○		○	○	○
Slit-lamp examination	○	○	○	○	○	○	○	○	○	○	○	○	○	○	○
Fundus examination	○	○			○		○	○	○	○	○	○	○	○	○
Fundus camera	○											○	○	○	○
CBC/blood chemistry test	○												○	○	○
Pharmacokinetics			○			○		○
Urinalysis	○				○				○				○	○	○
Imaging evaluation of the CNS	○													○	○
AEs and ADRs		○	○	○	○	○	○	○	○	○	○	○	○	○	○

*V1 and V2 can be performed on the same day.

†After approval of DSMB, the dose of the eye drops will be increased from one to two times per day.

‡The principal investigator decides whether to continue the trial.

ADR, adverse drug reaction; AE, adverse event; CBC, complete blood cell count; CNS, central nervous system; DSMB, data and safety monitoring board.