Status:
ACTIVE_NOT_RECRUITING
Safety and Tolerability of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations
Lead Sponsor:
Innostellar Biotherapeutics Co.,Ltd
Conditions:
Inherited Retinal Dystrophy Associated With RPE65 Mutations
Eligibility:
All Genders
6+ years
Phase:
PHASE1
PHASE2
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.
Eligibility Criteria
Inclusion
- 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
- 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.
Key Trial Info
Start Date :
July 2 2022
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 1 2027
Estimated Enrollment :
9 Patients enrolled
Trial Details
Trial ID
NCT06196827
Start Date
July 2 2022
End Date
December 1 2027
Last Update
July 1 2025
Active Locations (2)
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1
Beijing Tongren Hospital, Capital Medical University
Beijing, China
2
Shanghai General Hospital
Shanghai, China