Clinical trial
A Multi-center Clinical Study to Evaluate the Safety, Tolerability, and Efficacy of rAAV2-RPE65 Gene Therapy (LX101) in Subjects With Biallelic RPE65 Mutation-associated Inherited Retinal Dystrophy
Name
INNOSTELLAR-LX101-1 (phase 1)
Description
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 arms
Trial start
2022-07-02
Estimated PCD
2023-12-06
Trial end
2027-12-01
Status
Active (not recruiting)
Phase
Early phase I
Treatment
LX101
Subretinal Administration
Arms:
LX101 Dose 1, LX101 Dose 2
Size
9
Primary endpoint
Incidence of adverse events (AEs) and serious adverse events (SAEs)
12 months
Incidence of dose-limiting toxicity (DLT)
1 month
Eligibility criteria
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.
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE1', 'PHASE2'], 'designInfo': {'allocation': 'NON_RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 9, 'type': 'ACTUAL'}}
Updated at
2024-01-09
1 organization
1 product
1 indication
Product
LX101Organization
Innostellar Biotherapeutics