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

Innostellar Biotherapeutics

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