LX101

GENETIC4 trials

Sponsors

Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine, Innostellar Biotherapeutics Co.,Ltd

Conditions

Inherited Retinal DystrophyInherited Retinal Dystrophy Associated With RPE65 MutationsTo Evaluate the Scaling Clinical Study of AAV2-RPE65 Gene Therapy Agent (LX101) in Patients With Congenital Amaurosis (LCA)

Phase 1

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

Active, not recruitingNCT06196827

Innostellar Biotherapeutics Co.,LtdInherited Retinal Dystrophy Associated With RPE65 Mutations

Start: 2022-07-02End: 2027-12-01Updated: 2026-03-06

Fellow-eye Study (FE) of LX101 in Subjects With Inherited Retinal Dystrophy

Active, not recruitingNCT06212297

Innostellar Biotherapeutics Co.,LtdInherited Retinal Dystrophy

Start: 2023-09-12End: 2029-03-31Target: 9Updated: 2025-06-29

Phase 3

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

Active, not recruitingNCT07054632

Innostellar Biotherapeutics Co.,LtdInherited Retinal Dystrophy Associated With RPE65 Mutations

Start: 2023-09-13End: 2029-08-01Updated: 2026-03-05

Unknown Phase

An Expanded Clinical Study Evaluating the AAV2-RPE65 Gene Therapy(LX101) in Patients With LCA

Not yet recruitingNCT06024057

Shanghai General Hospital, Shanghai Jiao Tong University School of MedicineTo Evaluate the Scaling Clinical Study of AAV2-RPE65 Gene Therapy Agent (LX101) in Patients With Congenital Amaurosis (LCA)

Start: 2023-09-01End: 2038-09-30Target: 3Updated: 2023-09-05