NCT06196827Active, not recruitingPhase 1
Safety and Tolerability of LX101 for Inherited Retinal Dystrophy Associated With RPE65 Mutations
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
Source: ClinicalTrials.gov · Last updated 6 Mar 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Active, not recruiting
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 9 (actual)
- First submitted
- 25 Dec 2023
- Start date
- 2 Jul 2022
- Primary completion date
- 6 Dec 2023
- Lead sponsor
- Innostellar Biotherapeutics Co.,Ltd
- Diseases
- Retinitis pigmentosa
- NCT number
- NCT06196827
What you commit to
- Study type: Interventional · Phase 1
- Participants enrolled: 9
- From 2 Jul 2022 to the primary completion date, 6 Dec 2023
- 2 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.
Prior treatment
- Prior gene therapy for IRD and other hereditary eye diseases.
- Prior ocular surgery within six months.
Other illness
- Pre-existing eye conditions that would interfere with interpretation of study endpoints.
- Active intraocular or periocular infections in the study eye.
- Pre-existing systemic diseases that should not discontinue the use of any retinal toxic compounds.
Lab values
- Complicating systemic diseases or clinically significant abnormal baseline laboratory values.
Other
- Lacking of sufficient surviving retinal cells.
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 (2)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Beijing Tongren Hospital, Capital Medical University Coordinator of this site? Confirm its status | Beijing | China | Unknown status |
| Shanghai General Hospital Coordinator of this site? Confirm its status | Shanghai | China | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.