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

April 4, 2023 updated by: Xiaodong Sun, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

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

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 Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Anticipated)

12

Phase

  • Not Applicable

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • China
      • Shanghai, China, 200080
        • Recruiting
        • Shanghai General Hospital, Shanghai Jiao Tong University
        • Principal Investigator:
          • Xiaodong Sun, PhD
        • Contact:

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: LX103 Injection

Potential doses:

5E10 vg, 0.1 mL/eye/dose (low dose)

1E11 vg, 0.05 mL/eye/dose (high dose)
Genetic: LX103 Injection
Qualified subjects will receive a single unilateral intravitreal injection of LX103 at Day 0 in the trial.

What is the study measuring?

Primary Outcome Measures

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

Secondary Outcome Measures

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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

Collaborators

Innostellar Biotherapeutics

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

December 13, 2022

Primary Completion (Anticipated)

August 1, 2024

Study Completion (Anticipated)

August 1, 2028

Study Registration Dates

First Submitted

April 4, 2023

First Submitted That Met QC Criteria

April 4, 2023

First Posted (Actual)

April 18, 2023

Study Record Updates

Last Update Posted (Actual)

April 18, 2023

Last Update Submitted That Met QC Criteria

April 4, 2023

Last Verified

April 1, 2023

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SHGH-LX103

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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