Safety and Tolerability of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations

I

Innostellar Biotherapeutics Co.,Ltd

Status and phase

Active, not recruiting
Phase 2
Phase 1

Conditions

Inherited Retinal Dystrophy Associated With RPE65 Mutations

Treatments

Genetic: LX101

Study type

Interventional

Funder types

Industry

Identifiers

NCT06196827
INNOSTELLAR-LX101-1 (phase 1)

Details and patient eligibility

About

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.

Enrollment

9 patients

Sex

All

Ages

6+ years old

Volunteers

No Healthy Volunteers

Inclusion criteria

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.

Exclusion criteria

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.

Trial design

Primary purpose

Treatment

Allocation

Non-Randomized

Interventional model

Parallel Assignment

Masking

None (Open label)

9 participants in 2 patient groups

LX101 Dose 1
Experimental group
Treatment:
Genetic: LX101
LX101 Dose 2
Experimental group
Treatment:
Genetic: LX101

Trial contacts and locations

0

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Data sourced from clinicaltrials.gov

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