A Single Intravitreal Injection of rAAV2-ND4 for the Treatment of Leber's Hereditary Optic Neuropathy

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT03153293
• Date of registration: 10/05/2017
• Prospective Registration: Yes
• Primary sponsor: Huazhong University of Science and Technology
• Public title: A Single Intravitreal Injection of rAAV2-ND4 for the Treatment of Leber's Hereditary Optic Neuropathy
• Scientific title: A Single Intravitreal Injection of rAAV2-ND4 for the Treatment of Leber's Hereditary Optic Neuropathy
• Date of first enrolment: December 27, 2017
• Target sample size: 159
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT03153293
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2/Phase 3

Countries of recruitment

China

Contacts

• Name: Bin Li, PhD,MD
• Affiliation: Deputy Director of Ophthalmology,Tongji Hospital

• Name: Yong Zhang, PhD,MD
• Affiliation: Director of Ophthalmology,Shiyan Taihe Hospital

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Patients carry the mitochondrial point mutation at 11778, which is consistent with the
diagnostic criteria for LHON.

2. No apparent eye sight improvement in LHON patients or any other treatment within the
past year.

3. Eyesight of both eyes is below 0.3.

4. Patients signed written informed consent.

5. Patients are between the ages of 10 and 65 years old and able to tolerate the gene
therapy procedure which includes local anesthesia.

6. Patients are willing to follow the doctor's instructions and to consult the doctor at
prescribed times.

7. Patient's physical examination results are all normal, including liver function,
kidney function, routine blood test, routine urine test, complete immunological test,
and humoral immune response.

Exclusion Criteria:

1. Patients who are wearing a cardiac pacemaker, suffering from severe heart, lung or
kidney function failure, various hemorrhagic diseases, acute infectious diseases, high
fever, or convalescing after heart surgery or who are pregnant are excluded.

2. Patients who are participating in other clinical studies are excluded.

3. Patients who suffer from a diagnosed mental problem are excluded.

4. Patients who suffer from chronic diseases such as diabetes and hypertension are
excluded.

5. Patients who show abnormal test results such as positive AAV2 humoral immune response
(positive means that the AAV2 neutralizing antibody assay of patient was significant
different when comparing free serum with 1:20 serum concentrations) and abnormal human
T lymphocyte subsets CD3+, CD3+/CD4+ and CD3+/CD8+ prior to gene therapy surgery are
excluded.



Age minimum: 10 Years
Age maximum: 65 Years
Gender: All

Health Condition(s) or Problem(s) studied

Leber Hereditary Optic Neuropathy

Intervention(s)

Drug: rAAV2-ND4

Primary Outcome(s)

Computerized Visual Field [Time Frame: Change from Baseline at 12 months .]

BCVA [Time Frame: Change from Baseline at 12 months .]

Secondary Outcome(s)

Liver function in plasma [Time Frame: Change from Baseline at 12 months .]

RNFL [Time Frame: Change from Baseline at 12 months .]

kidney function in plasma [Time Frame: Change from Baseline at 12 months .]

VEP [Time Frame: Change from Baseline at 12 months .]

Secondary ID(s)

Leber 2

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Shiyan Taihe Hospital