NCT06196827

Brief Summary

The purpose of the study is to evaluate the safety, tolerability and efficacy of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy.

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
19mo left

Started Jul 2022

Longer than P75 for phase_1

Geographic Reach
1 country

2 active sites

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 Progress71%
Jul 2022Dec 2027

Study Start

First participant enrolled

July 2, 2022

Completed
1.4 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 6, 2023

Completed
19 days until next milestone

First Submitted

Initial submission to the registry

December 25, 2023

Completed
15 days until next milestone

First Posted

Study publicly available on registry

January 9, 2024

Completed
3.9 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 1, 2027

Expected
Last Updated

March 6, 2026

Status Verified

March 1, 2026

Enrollment Period

1.4 years

First QC Date

December 25, 2023

Last Update Submit

March 4, 2026

Conditions

Inherited Retinal Dystrophy Associated With RPE65 Mutations

Outcome Measures

Primary Outcomes (2)

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

    Incidence of ocular and non-ocular AEs and SAEs following LX101 subretinal injection

    12 months

  • Incidence of dose-limiting toxicity (DLT)

    Incidence of DLT following LX101 subretinal injection at different doses

    1 month

Secondary Outcomes (2)

  • Efficacy of LX101 in study eye

    12 months

  • Efficacy of LX101 in study eye

    12 months

Study Arms (2)

LX101 Dose 1

EXPERIMENTAL
Genetic: LX101

LX101 Dose 2

EXPERIMENTAL
Genetic: LX101

Interventions

LX101GENETIC

Subretinal Administration

LX101 Dose 1LX101 Dose 2

Eligibility Criteria

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

You may qualify if:

  • Subject and/or their guardian signing a written informed consent.
  • Diagnosed with biallelic RPE65 mutation-associated inherited retinal dystrophy.
  • Subjects are 6 years of age or older.
  • Visual acuity of ≤ 20/63 or visual field less than 20 degrees in the eye to be injected.

You may not qualify if:

  • Prior gene therapy for IRD and other hereditary eye diseases.
  • Pre-existing eye conditions that would interfere with interpretation of study endpoints.
  • Active intraocular or periocular infections in the study eye.
  • Lacking of sufficient surviving retinal cells.
  • Prior ocular surgery within six months.
  • Complicating systemic diseases or clinically significant abnormal baseline laboratory values.
  • Pre-existing systemic diseases that should not discontinue the use of any retinal toxic compounds.

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (2)

Beijing Tongren Hospital, Capital Medical University

Beijing, China

Location

Shanghai General Hospital

Shanghai, China

Location

Study Design

Study Type
interventional
Phase
phase 1
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
PARALLEL
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

December 25, 2023

First Posted

January 9, 2024

Study Start

July 2, 2022

Primary Completion

December 6, 2023

Study Completion (Estimated)

December 1, 2027

Last Updated

March 6, 2026

Record last verified: 2026-03

Locations

CN(2)