Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON)

Plain-language summary

This study assessed the safety and efficacy of rAAV2-ND4 gene therapy in patients with Leber hereditary optic neuropathy (LHON) carrying the 11778 mutation, an inherited condition affecting the optic nerve. Participants had to meet the diagnostic criteria for LHON, be between 8 and 60 years old, be in good general health, be able to tolerate the procedure under local anesthesia, and provide informed consent.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

Not Applicable

Status

Completed

Sponsor

Bin Li

Intervention

rAAV2-ND4 (rAAV2-ND4 gene therapy)

Start date

4/1/2011

Estimated completion

11/1/2015

Countries

China

Locations

• Wuhan, China

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

Inclusion Criteria:

1. comply with Leber hereditary optic neuropathy diagnostic criteria.
2. in patients with informed consent, voluntary participation.
3. signed informed consent.
4. 8 ≤ Age ≤ 60 years old, good health, the patient can tolerate local anesthesia surgery.
5. to comply with doctor's instructions, can in the time of referral.

Exclusion Criteria:

1. Cardiopulmonary and renal function in severe weakness, cancer, a variety of bleeding disorders, acute sensing disease, high fever, high fever disease, women during pregnancy, heart disease, such as post-operative recovery period.
2. Are participating in other clinical studies of patients.
3. Patients with mental disorders.