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

Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine12 enrolled

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.

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

X-linked Retinoschisis

Interventions

LX103 InjectionGENETIC

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

Eligibility

Sex: MALEMin age: 6 Years

Medical Language ↔ Plain English

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.

Locations (1)

Shanghai General Hospital, Shanghai Jiao Tong University

Shanghai, China

RECRUITING

Outcomes

Primary Outcomes

Incidence of adverse events (AEs)

Incidence of adverse events (AEs) within 24 weeks of LX103 intravitreal injection at different doses

Time frame: 24 weeks

Incidence of serious adverse events (SAEs)

Incidence of serious adverse events (SAEs) within 24 weeks of LX103 intravitreal injection at different doses

Time frame: 24 weeks

Secondary Outcomes

Mean change from baseline in BCVA (LogMAR) in the study eye

ETDRS visual acuity charts will be used to assess BCVA

Time frame: 24 weeks, 52 weeks

Mean change from baseline in central subfield thickness (CST) in the study eye

Mean change of CST measured using Optical Coherence Tomography (OCT)

Time frame: 24 weeks, 52 weeks

Change from baseline in Electroretinogram (ERG) parameters in the study eye

Change in dark-adapted 3.0 B-wave amplitude (μV)

Time frame: 24 weeks, 52 weeks

Data from ClinicalTrials.gov

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