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

Primary Purpose

X-linked Retinoschisis

Status

Recruiting

Phase

Not Applicable

Locations

China

Study Type

Interventional

Intervention

LX103 Injection

About this trial

This is an interventional treatment trial for X-linked Retinoschisis

Eligibility Criteria

6 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria: 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. Exclusion Criteria: 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.

Sites / Locations

Shanghai General Hospital, Shanghai Jiao Tong UniversityRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

LX103 Injection

Arm Description

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

Outcomes

Primary Outcome Measures

Incidence of adverse events (AEs)

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

Incidence of serious adverse events (SAEs)

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

Secondary Outcome Measures

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

ETDRS visual acuity charts will be used to assess BCVA

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

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

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

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

Full Information

NCT ID

NCT05814952

First Posted

April 4, 2023

Last Updated

April 4, 2023

Sponsor

Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

Collaborators

Innostellar Biotherapeutics

1. Study Identification

Unique Protocol Identification Number

NCT05814952

Brief Title

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

Official Title

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

Study Type

Interventional

2. Study Status

Record Verification Date

April 2023

Overall Recruitment Status

Recruiting

Study Start Date

December 13, 2022 (Actual)

Primary Completion Date

August 2024 (Anticipated)

Study Completion Date

August 2028 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine

Collaborators

Innostellar Biotherapeutics

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

X-linked Retinoschisis

7. Study Design

Primary Purpose

Treatment

Study Phase

Not Applicable

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

12 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

LX103 Injection

Arm Type

Experimental

Arm Description

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

Intervention Type

Genetic

Intervention Name(s)

LX103 Injection

Intervention Description

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

Primary Outcome Measure Information:

Title

Incidence of adverse events (AEs)

Description

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

Time Frame

24 weeks

Title

Incidence of serious adverse events (SAEs)

Description

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

Time Frame

24 weeks

Secondary Outcome Measure Information:

Title

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

Description

ETDRS visual acuity charts will be used to assess BCVA

Time Frame

24 weeks, 52 weeks

Title

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

Description

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

Time Frame

24 weeks, 52 weeks

Title

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

Description

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

Time Frame

24 weeks, 52 weeks

10. Eligibility

Sex

Male

Minimum Age & Unit of Time

6 Years

Accepts Healthy Volunteers

Eligibility Criteria

Facility Information:

Facility Name

Shanghai General Hospital, Shanghai Jiao Tong University

City

Shanghai

ZIP/Postal Code

200080

Country

China

Individual Site Status

Recruiting

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Junran Sun, MD

Phone

+86 13661995933

emiliesun@sina.com

First Name & Middle Initial & Last Name & Degree

Xiaodong Sun, PhD

12. IPD Sharing Statement

Plan to Share IPD

Learn more about this trial

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

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