Actively Recruiting
An Exploratory Clinical Study to Evaluate LX107 Gene Therapy in Patients With AIPL1 Biallelic Mutation-related Inherited Retinal Dystrophy (AIPL1-IRD)
Led by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine · Updated on 2026-03-10
13
Participants Needed
1
Research Sites
208 weeks
Total Duration
On this page
Sponsors
S
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
Lead Sponsor
I
Innostellar Biotherapeutics Co.,Ltd
Collaborating Sponsor
AI-Summary
What this Trial Is About
Researchers are evaluating the use of LX107, a gene therapy drug, in patients with retinal dystrophy caused by mutations in the AIPL1 gene. This early phase 1 clinical trial aims to assess the safety and effectiveness of this treatment for individuals aged 4 years and older who have a confirmed diagnosis of this inherited retinal disease. The study is sponsored by Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine. Participants will receive a single subretinal injection of LX107 in one eye, designated as the study eye. The study includes three dose groups: low dose (3x10^9 viral genomes per eye), medium dose (1x10^10 viral genomes per eye), and high dose (3x10^10 viral genomes per eye). Treatment will be administered on Day 0, and patients will be monitored closely throughout the study. During the study, participants will undergo evaluations such as functional MRI scans, mobility tests, full-field sensitivity threshold tests, visual acuity assessments, and visual field measurements at 6 and 12 months after treatment. Safety will be closely monitored by tracking treatment-emergent adverse events over 6 months. Participants will also be followed long-term according to the study protocol to assess treatment effects and safety over time.
CONDITIONS
Brief Title
A Study of LX107 Gene Therapy in AIPL1-IRD Patients
Who Can Participate
Eligibility Criteria
You may qualify if you...
- The subject or their guardian signs informed consent and agrees to long-term follow-up.
- Patients aged 4 years or older diagnosed with AIPL1 inherited retinal dystrophy.
- Confirmed biallelic AIPL1 gene mutations by genetic testing.
- Study eye has best-corrected visual acuity of 58 letters or fewer (decimal visual acuity 4 0.3).
You will not qualify if you...
- History of ocular diseases that may interfere with treatment or study results (e.g., glaucoma, diabetic retinopathy, retinal vein occlusion, retinal detachment, severe inflammation).
- Prior gene therapy for inherited retinal diseases or other hereditary eye conditions.
- Insufficient viable retinal cells in the study eye.
- Active intraocular or periocular infection in the study eye.
- Intraocular surgery in the 6 months before screening.
- Uncontrolled hypertension (systolic 4 160 mmHg or diastolic 4 100 mmHg).
- Diabetic patients with macrovascular complications, HbA1c over 7.5%, or on multiple diabetes medications including insulin or GLP-1 receptor agonists.
- Other health conditions or lab abnormalities that increase risk or interfere with results (e.g., AIDS, syphilis, active hepatitis B or C, coagulation disorders, recent malignancy except certain skin or cervical cancers).
- Low platelet count or hemoglobin below specified levels.
- Elevated liver enzymes or kidney function tests.
- Use of other investigational drugs within 3 months prior to screening.
- Unwillingness to use effective contraception or pregnancy/lactation during the study period.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 6 months
Participants receive a single subretinal injection of LX107 gene therapy on Day 0.
Multiple follow-up visits over 6 months to monitor safety and treatment effects
Duration - Up to 6 additional months after treatment
Participants are monitored for safety and treatment outcomes including visual function and brain imaging up to 12 months after treatment.
Periodic visits up to 12 months post-treatment
Trial Site Locations
Total: 1 location
1
Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine
Shanghai, China
Actively Recruiting
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
Frequently Asked Questions
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