N/A N=9 Treatment

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

Leber Hereditary Optic Neuropathy

Bottom Line

View on ClinicalTrials.gov: NCT01267422 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

May 2016

Primary outcome: Primary: The Best Corrected Visual Acuity(BCVA) — 1.40; 1.69; 1.36; 1.58 logMAR

Study Design & Population

Study type: Interventional
Phase: N/A
Interventions: rAAV2-ND4 (Drug)
Age: Pediatric, Adult · 8+ yrs
Sex: All
Sponsor: Bin Li
Primary completion: Nov 2015

Outcome Measures

Outcome	Result	p-value
PRIMARY The Best Corrected Visual Acuity(BCVA)	1.40; 1.69; 1.36; 1.58; 1.31; 1.38	—
PRIMARY Results of CD3/CD4/CD8 Test	51; 53; 26; 20; 20; 13	—
SECONDARY Intraocular Pressure;	14; 16	—
SECONDARY Neutralizing Antibody Assay	0.9221; 0.8723	—
SECONDARY Average RNFL Thickness Througth Optical Coherence Tomography(OCT) Test	47.3125; 46.7813; 50.1875; 47.9688	—
SECONDARY Computerized Visual Field(MD: Mean Deviation, the Value Close to 0 Regarded Normal)	-29.377; -23.813; -28.701; -23.427	—
SECONDARY Computerized Visual Field(VFI: Visual Field Index ,the Value Close to 100% Regarded Normal)	11; 28; 10.5; 25	—

Summary

This study is meant to assess the safety and efficacy of rAAV2-ND4 treatment of Leber hereditary optic neuropathy with 11778 LHON mutation.

Eligibility Criteria

Inclusion Criteria

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

Exclusion Criteria

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.
Are participating in other clinical studies of patients.
Patients with mental disorders.

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT01267422). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.