NCT06212297

Brief Summary

Up to nine subjects who have participated in the earlier LX101 clinical study, and who meet all study eligibility criteria, will receive LX101 administration in the previously uninjected, contralateral eye to evaluate the safety of bilateral, sequential subretinal administration of LX101.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
9

participants targeted

Target at below P25 for phase_1

Timeline
35mo left

Started Sep 2023

Longer than P75 for phase_1

Geographic Reach
1 country

1 active site

Status
active not recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress49%
Sep 2023Mar 2029

Study Start

First participant enrolled

September 12, 2023

Completed
3 months until next milestone

First Submitted

Initial submission to the registry

December 25, 2023

Completed
24 days until next milestone

First Posted

Study publicly available on registry

January 18, 2024

Completed
1.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

February 6, 2025

Completed
4.1 years until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2029

Expected
Last Updated

June 29, 2025

Status Verified

June 1, 2025

Enrollment Period

1.4 years

First QC Date

December 25, 2023

Last Update Submit

June 27, 2025

Conditions

Inherited Retinal Dystrophy

Keywords

RPE65 Mutations

Outcome Measures

Primary Outcomes (1)

  • Safety: Incidence of adverse events (AEs) and serious adverse events (SAEs)

    Incidence of ocular and non-ocular AEs and SAEs following LX102 subretinal injection

    6 months

Secondary Outcomes (3)

  • Full-field Light Sensitivity Threshold (FST) Test

    6 months、12 months

  • Visual Acuity

    6 months、12 months

  • Mobility Test

    6 months、12 months

Study Arms (1)

LX101 Subretinal Administration

EXPERIMENTAL
Genetic: LX101

Interventions

LX101GENETIC

Subretinal administration of LX101 to the contralateral, previously uninjected eye

LX101 Subretinal Administration

Eligibility Criteria

Age6 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

  • Prior subretinal administration of LX101 (unilateral)
  • ≥ 6 years old Signed written informed consent

You may not qualify if:

  • Prior gene therapy except LX101 Active intraocular or periocular infections Lacking of sufficient surviving retinal cells Prior ocular surgery within six months Retinoid like compounds or precursors were taken within three months Complicating systemic diseases Clinically significant abnormal baseline laboratory values Using of any retinal toxic compounds

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Shanghai General Hospital

Shanghai, Shanghai Municipality, 200080, China

Location

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 25, 2023

First Posted

January 18, 2024

Study Start

September 12, 2023

Primary Completion

February 6, 2025

Study Completion (Estimated)

March 1, 2029

Last Updated

June 29, 2025

Record last verified: 2025-06

Locations

CN(1)