CompletedEarly Phase 1

Safety and Efficacy Study of Novel Gene Therapy ZM-01 for X-linked Retinoschisis Patients

China2 participantsStarted 2022-09-25

Plain-language summary

This trial is meant to evaluate the safety and efficacy of ZM-01 of X-linked retinoschisis. Unilateral intravitreal injections (IVT) will be given into the subject's Study Eye.

Who can participate

Age range

3 Years – 18 Years

Sex

MALE

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Diagnosis of X-linked retinoschisis consistent with the presence of RS1 gene mutation
. Male, aged between 3 and 18 years old, in overall good health except for XLRS condition
. Capable of undergoing visual and retinal function assessment.
. The visual acuity of the study eye not better than: 0.4 (68 ETDRS letters equivalent)
. No carbonic anhydrase inhibitors have been used at present and for 3 months before treatment
. Laboratory tests meet the following criteria:
. Hemoglobin ≥ 11.0 g/dL
. White blood cell counts ranged from 3,300 to 12,000 cells /mm³;

Exclusion criteria

. Previously received any AAV gene therapy
. The following mutations in RS1 gene: R141H, C59S or C223S
. Pre-existing eye conditions that cause severe vision loss or increase the risk of intravitreal injections (e.g., advanced glaucoma, uveitis, or severe retinal detachment)

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Incidence of adverse events and serious adverse events

Timeframe: baseline to day 7, month 1, 2

Change in best corrected visual acuity (BCVA)

Timeframe: baseline to day 7, month 1, 2

Trial details

NCT IDNCT06066008

SponsorZhongmou Therapeutics

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2023-10-31

View on ClinicalTrials.gov More X-linked Retinoschisis trials