Safety and Efficacy Study of LX103 Treatment of X-Linked Retinoschisis (XLRS)

An Open Label Dose Escalation Clinical Trial to Evaluate the Safety and the Efficacy of LX103 in Patients With X-Linked Retinoschisis (XLRS)

Status

Recruiting

Phases

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT05814952

Enrollment

Registered

2023-04-18

Start date

2022-12-13

Completion date

2028-08-31

Last updated

2023-04-18

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

X-linked Retinoschisis

Brief summary

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

GENETICLX103 Injection

Qualified subjects will receive a single unilateral intravitreal injection of LX103 at Day 0 in the trial.

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

MALE

Age

6 Years to No maximum

Healthy volunteers

Inclusion criteria

1. Willing to sign the informed consent, and willing to attend follow-up visits. 2. Male individual at least 6 years of age with a diagnosis of XLRS and documented mutations in the RS1 gene. 3. The study eye must have a best-corrected E-ETDRS visual acuity letter score of less than or equal to 63.

Exclusion criteria

1. Any eye with disease that would interfere with the fundus examinations. 2. The study eye is receiving topical carbonic anhydrase inhibitor, or has received topical carbonic anhydrase inhibitors in the past three months. 3. The study eye has undergone intraocular surgery within six months prior to enrollment. 4. Participant has uncontrolled hypertension or diabetes.

Design outcomes

Primary

Measure	Time frame	Description
Incidence of adverse events (AEs)	24 weeks	Incidence of adverse events (AEs) within 24 weeks of LX103 intravitreal injection at different doses
Incidence of serious adverse events (SAEs)	24 weeks	Incidence of serious adverse events (SAEs) within 24 weeks of LX103 intravitreal injection at different doses

Secondary

Measure	Time frame	Description
Mean change from baseline in BCVA (LogMAR) in the study eye	24 weeks, 52 weeks	ETDRS visual acuity charts will be used to assess BCVA
Mean change from baseline in central subfield thickness (CST) in the study eye	24 weeks, 52 weeks	Mean change of CST measured using Optical Coherence Tomography (OCT)
Change from baseline in Electroretinogram (ERG) parameters in the study eye	24 weeks, 52 weeks	Change in dark-adapted 3.0 B-wave amplitude (μV)

Countries

China

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: Feb 4, 2026