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

Shanghai Jiao Tong University

Status: Enrolling

Conditions: X-linked Retinoschisis

Treatments: Genetic: LX103 Injection

Study type: Interventional

Funder types: Other

Identifiers

NCT05814952

SHGH-LX103

Details and patient eligibility

About

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.

Enrollment: 12 estimated patients

Sex: Male

Ages: 6+ years old

Volunteers: No 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.

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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12 participants in 1 patient group

LX103 Injection

Experimental group

Description:

Potential doses: 5E10 vg, 0.1 mL/eye/dose (low dose) 1E11 vg, 0.05 mL/eye/dose (high dose)

Treatment:

Genetic: LX103 Injection