NCT07054632Active, not recruitingPhase 3
Efficacy and Safety of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations
Efficacy and Safety of Gene Therapy rAAV-RPE65 (LX101) in Biallelic RPE65 Mutation-associated Inherited Retinal Dystrophy: a Phase III, Multicenter, Randomized Controlled Trial (STAR)
Source: ClinicalTrials.gov · Last updated 5 Mar 2026
Brief summary (registry, in English)
The purpose of the study is to evaluate the efficacy and safety of LX101 in subjects with biallelic RPE65 mutation-associated inherited retinal dystrophy. This is an open-label, multicenter, randomized controlled Phase III clinical trial. Subjects were randomly assigned in a 1:1 ratio to either the intervention group or the control group. Subjects in the intervention group received subretinal injection of LX101, while those in the control group received no treatment.
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 3
- Study type
- Interventional
- Enrollment
- 30 (actual)
- First submitted
- 27 Jun 2025
- Start date
- Sept 2023
- Primary completion date
- Aug 2025
- Lead sponsor
- Innostellar Biotherapeutics Co.,Ltd
- Diseases
- Retinitis pigmentosa
- NCT number
- NCT07054632
What you commit to
- Study type: Interventional · Phase 3
- Participants enrolled: 30
- From Sept 2023 to the primary completion date, Aug 2025
- 4 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Lab values
- 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.
- Complicating systemic diseases or clinically significant abnormal baseline laboratory values.
Who the study is looking for
- 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.
Age in the registry: 6 years to - · Sex: All · Does not accept healthy volunteers
Sites (4)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Southwest Hospital of AMU Coordinator of this site? Confirm its status | Chongqing | China | Unknown status |
| Zhongshan Ophthalmic Center,Sun Yat-Sen University Coordinator of this site? Confirm its status | Guangzhou | China | Unknown status |
| Shanghai General Hospital Coordinator of this site? Confirm its status | Shanghai | China | Unknown status |
| Tianjin Medical University Ophthalmology Hospital Coordinator of this site? Confirm its status | Tianjin | China | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.