Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON)
This study is currently recruiting participants.
Verified December 2010 by Huazhong University of Science and Technology
Sponsor:
Bin Li
Collaborator:
Huazhong University of Science and Technology
Information provided by (Responsible Party):
Bin Li, Huazhong University of Science and Technology
ClinicalTrials.gov Identifier:
NCT01267422
First received: December 27, 2010
Last updated: April 20, 2013
Last verified: December 2010
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Purpose
This study is meant to assess the safety and efficacy of rAAV2-ND4 treatment of Leber hereditary optic neuropathy with 11778 LHON mutation.
| Condition | Intervention |
|---|---|
|
Leber Hereditary Optic Neuropathy |
Drug: rAAV2-ND4 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Safety and Efficacy Study of a Single Intravitreal Injection of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy |
Resource links provided by NLM:
Genetics Home Reference related topics:
ataxia neuropathy spectrum
Charcot-Marie-Tooth disease
childhood myocerebrohepatopathy spectrum
deoxyguanosine kinase deficiency
hereditary neuropathy with liability to pressure palsies
Leber hereditary optic neuropathy
Lenz microphthalmia syndrome
mitochondrial neurogastrointestinal encephalopathy disease
myoclonic epilepsy myopathy sensory ataxia
oculofaciocardiodental syndrome
Peters plus syndrome
U.S. FDA Resources
Further study details as provided by Huazhong University of Science and Technology:
Primary Outcome Measures:
- The visual acuity [ Time Frame: 6 month ] [ Designated as safety issue: No ]Vision testing before and after treatment
Secondary Outcome Measures:
- Intraocular pressure [ Time Frame: 6 month ] [ Designated as safety issue: Yes ]Intraocular pressure testing before and after treatment
- Visual evoked potential(VEP) [ Time Frame: 6 month ] [ Designated as safety issue: Yes ]VEP testing before and after treatment
- computerized visual field [ Time Frame: 6 month ] [ Designated as safety issue: No ]computerized visual field testing before and after treatment
- Optical coherence tomography(OCT) [ Time Frame: 6 month ] [ Designated as safety issue: No ]OCT testing before and after treatment
- Electroretinogram(ERG) [ Time Frame: 6 month ] [ Designated as safety issue: Yes ]ERG testing before and after treatment
- Liver and kidney function in plasma [ Time Frame: 6 month ] [ Designated as safety issue: Yes ]Liver and kidney function in plasma testing before and after treatment
- Enzyme-linked immunosorbent assay Enzyme-linked immunosorbent assay (ELISA) [ Time Frame: 6 month ] [ Designated as safety issue: Yes ]Antibodies against AAV2 capsid was analyzed by ELISA using serum samples collected before injection and different time points after the injection.
| Estimated Enrollment: | 6 |
| Study Start Date: | April 2011 |
| Estimated Study Completion Date: | October 2013 |
| Estimated Primary Completion Date: | September 2013 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
|
Experimental: rAAV2-ND4
injection
|
Drug: rAAV2-ND4
injection
|
Detailed Description:
Leber's Hereditary Optic Neuropathy (LHON) is a maternally inherited ocular disorder associated with a mutation in mtDNA . The common manifestation is visual loss which caused by the respiratory chain enzymes complex dysfunction resulting in increased oxidative stress enzymes production.
Material and Method Seven patients with 11778 LHON mutation were randomly treated with a Single IVT Injection of recombinant Adeno-Associated Virus-NADH dehydrogenase, subunit 4 (complex I)（rAAV2-ND4）（0.06ml/1.5×1010; The visual acuity, visual evoked potential （VEP）,optical coherence tomography（ OCT）, computerized visual field, Electroretinography（ERG） and Liver and kidney function in plasma were compared before and after treatment at 1,3,and 6, months interval.
Eligibility| Ages Eligible for Study: | 8 Years to 60 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
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.
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT01267422
Contacts
| Contact: Bin Li, PhD,MD | 8613638673626 | scotopsin@163.com |
| Contact: Han Pei, PhD | 8627-83663223 | libin-12@163.com |
Locations
| China, Hubei | |
| Department of Ophthalmology ，Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology | Recruiting |
| Wu Han, Hubei, China, 430030 | |
| Contact: Bin Li, PhD,MD 8613638673626 scotopsin@163.com | |
Sponsors and Collaborators
Bin Li
Huazhong University of Science and Technology
Investigators
| Study Chair: | bin Li, PhD,MD | Deputy Director of Ophthalmology |
More Information
No publications provided
| Responsible Party: | Bin Li, Safety and Efficacy Study of rAAV2-ND4 Treatment of LHON, Huazhong University of Science and Technology |
| ClinicalTrials.gov Identifier: | NCT01267422 History of Changes |
| Other Study ID Numbers: | RAVCT-2 |
| Study First Received: | December 27, 2010 |
| Last Updated: | April 20, 2013 |
| Health Authority: | China: Food and Drug Administration |
Keywords provided by Huazhong University of Science and Technology:
|
Leber's Hereditary Optic Neuropathy,11778 LHON mutation, rAAV2-ND4,triamcinolone acetonide |
Additional relevant MeSH terms:
|
Optic Nerve Diseases Demyelinating Diseases Polyneuropathies Nerve Compression Syndromes Neurologic Manifestations Neurotoxicity Syndromes Optic Atrophy, Hereditary, Leber Cranial Nerve Diseases Nervous System Diseases Eye Diseases Peripheral Nervous System Diseases Neuromuscular Diseases |
Signs and Symptoms Poisoning Substance-Related Disorders Optic Atrophies, Hereditary Optic Atrophy Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Eye Diseases, Hereditary Genetic Diseases, Inborn Mitochondrial Diseases Metabolic Diseases |
ClinicalTrials.gov processed this record on May 16, 2013