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

NCT05814952 | Recruiting

• 1 other identifier: SHGH-LX103
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

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.

Conditions

X-linked Retinoschisis

Outcome Measures

Primary Outcomes (2)

• Incidence of adverse events (AEs)

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

  24 weeks

• Incidence of serious adverse events (SAEs)

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

  24 weeks

Secondary Outcomes (3)

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

  24 weeks, 52 weeks

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

  24 weeks, 52 weeks

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

  24 weeks, 52 weeks

Study Arms (1)

LX103 Injection

EXPERIMENTAL

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

Genetic: LX103 Injection

Interventions

LX103 Injection GENETIC

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

LX103 Injection

Eligibility Criteria

• Age: 6 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine: lead
• Innostellar Biotherapeutics: collaborator

Study Sites (1)

Shanghai General Hospital, Shanghai Jiao Tong University

Shanghai, 200080, China

RECRUITING

MeSH Terms

Conditions

Retinoschisis

Condition Hierarchy (Ancestors)

Retinal Degeneration; Retinal Diseases; Eye Diseases

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: PhD

Study Record Dates

• First Submitted: April 4, 2023
• First Posted: April 18, 2023
• Study Start: December 13, 2022
• Primary Completion: August 1, 2024
• Study Completion (Estimated): August 1, 2028
• Last Updated: April 18, 2023

Record last verified: 2023-04

Data Sharing

• IPD Sharing: Will not share

Locations

CN (1)