Safety and Efficacy Study of LX103 Treatment of X-Linked Retinoschisis (XLRS)
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: LX103 Injection.
- Who it may be relevant to
- Registry conditions: X-linked Retinoschisis. Basic parameters: from 6 years · Male.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- China
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Efficacy of LX103 in Patients With X-Linked Retinoschisis (XLRS)
Overview
The goal of this study is to evaluate the safety and efficacy of LX103 treatment of X-linked retinoschisis. This study will enroll subjects aged ≥ 6 years old to receive a single unilateral intravitreal (IVT) injection of LX103 to evaluate its safety and efficacy.
Interventions
- Genetic LX103 Injection
Qualified subjects will receive a single unilateral intravitreal injection of LX103 at Day 0 in the trial.
Primary outcome measures
- Incidence of adverse events (AEs) [Time frame: 24 weeks]
- Incidence of serious adverse events (SAEs) [Time frame: 24 weeks]
Secondary outcome measures (3)
- Mean change from baseline in BCVA (LogMAR) in the study eye [Time frame: 24 weeks, 52 weeks]
- Mean change from baseline in central subfield thickness (CST) in the study eye [Time frame: 24 weeks, 52 weeks]
- Change from baseline in Electroretinogram (ERG) parameters in the study eye [Time frame: 24 weeks, 52 weeks]
Eligibility criteria
Inclusion criteria
- Willing to sign the informed consent, and willing to attend follow-up visits.
- Male individual at least 6 years of age with a diagnosis of XLRS and documented mutations in the RS1 gene.
- The study eye must have a best-corrected E-ETDRS visual acuity letter score of less than or equal to 63.
Exclusion criteria
- Any eye with disease that would interfere with the fundus examinations.
- The study eye is receiving topical carbonic anhydrase inhibitor, or has received topical carbonic anhydrase inhibitors in the past three months.
- The study eye has undergone intraocular surgery within six months prior to enrollment.
- Participant has uncontrolled hypertension or diabetes.
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Allocation
- N/A
- Model
- Single group
- Masking
- Open label
- Primary purpose
- Treatment
Study locations
China · 1 center
- Shanghai General Hospital, Shanghai Jiao Tong University — Shanghai
Identifiers
NCT: NCT05814952 · SHGH-LX103