Autosomal dominant optic atrophy
Conditions
Interventions
Group 1:intravitreal injection of rAAV2-OPA1
Sponsors
Taihe Hospital
Eligibility
Sex/Gender
All
Age
8 Years to 70 Years
Inclusion criteria
Inclusion criteria: 1. Patients who meet the diagnostic criteria for autosomal dominant optic atrophy and have OPA1 gene mutation confirmed by gene sequencing, and have not received other treatment in the last 3 months, and their vision has not improved; 2. Patients with visual acuity below 0.3 in both eyes; 3. 8 years old <= Age <= 70 years old, male and female; 4. During treatment, agree not to use other drugs or treatments for the disease; 5. Patients and their legal representatives participate voluntarily and sign informed consent. 6. Follow the doctor's instructions and be able to return to the doctor at the prescribed time.
Exclusion criteria
Exclusion criteria: 1. Complicated with the following diseases: (1) Diabetes and other chronic diseases; (2) Serious cardiovascular, cerebrovascular, liver, kidney and other primary diseases; (3) Other systemic neurological diseases; (4) Patients in convalescence after heart surgery; (5) Patients with mental disorders; (6) Tumor patients; (7) Patients with other eye diseases. 2. Patients who participated in other clinical studies within the last 3 months; 3. Alcoholics, smokers, drug users or those with a history of drug abuse or exposure to toxic substances; 4. Pregnant women, breast-feeding women or those who plan to get pregnant within 12 months; 5. Abnormal preoperative immunoassay results (such as positive AAV2 humoral immune response, abnormal CD3/CD8, etc.); 6. Other reasons that the researcher considers inappropriate to participate in this study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| vision;visual field; | — |
Secondary
| Measure | Time frame |
|---|---|
| Thickness of the retinal nerve fiber layer;Visual Evoked Potential;Fundus; | — |
Countries
China
Contacts
Public ContactBin Li
Taihe Hospital
Outcome results
None listed