Clinical Trials in China / NCT06196827
Active, not recruiting
Phase 1
Safety and Tolerability of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations
NCT06196827 · tracked via the Priya Life Science China tracker
Condition(s) studied
Inherited Retinal Dystrophy Associated With RPE65 Mutations
Investigational drug(s) / intervention(s)
LX101
LX101: Subretinal Administration
Study 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.
Eligibility
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.
Primary outcome measure(s)
- Incidence of adverse events (AEs) and serious adverse events (SAEs) — 12 months
Incidence of ocular and non-ocular AEs and SAEs following LX101 subretinal injection - Incidence of dose-limiting toxicity (DLT) — 1 month
Incidence of DLT following LX101 subretinal injection at different doses
Trial sites (2)
| Facility | City | Region | Status |
|---|---|---|---|
| Beijing Tongren Hospital, Capital Medical University | Beijing | China | |
| Shanghai General Hospital | Shanghai | China |
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Efficacy and Safety of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations
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Other trials for the same condition
Efficacy and Safety of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations
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Official registry record
This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT06196827 on ClinicalTrials.gov ↗ ← All trials in China