Leber Hereditary Optic NeuropathyrAAV2-ND4NCT01267422
Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON)
A study testing rAAV2-ND4 in people with Leber Hereditary Optic Neuropathy, run by Bin Li at 1 site in China.
Trial summary
This study is meant to assess the safety and efficacy of rAAV2-ND4 treatment of Leber hereditary optic neuropathy with 11778 LHON mutation. Read more about this study
- Who is it for?
- Ages 8 to 60 years
- Conditions the study is for
- Leber Hereditary Optic Neuropathy
- What is being tested?
- rAAV2-ND4
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 9 participants
- Where
- 1 siteChina
- Study dates
- Apr 2011to Nov 2015
- Phase
- Interventional
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 8 to 60 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
China1 site
Department of Ophthalmology ，Tongji Hospital, Tongji Medical College, Huazhong University of Science and Technology
Wuhan, Hubei, 430030
About this study
Official title: Safety and Efficacy Study of a Single Intravitreal Injection of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy
This study is meant to assess the safety and efficacy of rAAV2-ND4 treatment of Leber hereditary optic neuropathy with 11778 LHON mutation.
Read the 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)（0.05ml).The dose was 5 × 10^9 vg/0.05 mL for patients younger than 12 years old, and 1 × 10^10 vg/0.05 mL for patients older than 12 years old. The visual acuity, visual evoked potential （VEP）,optical coherence tomography（ OCT）, computerized visual field, electroretinograms(ERG), retinal nerve fiber layer(RNFL)and Liver and kidney function in plasma were compared before and after treatment at 1,3,and 6, months interval.
- Conditions
- Leber Hereditary Optic Neuropathy
- Being tested
- rAAV2-ND4
- Keywords
- Leber's Hereditary Optic Neuropathy,11778 LHON mutation,, rAAV2-ND4,triamcinolone acetonide
- Medical subject headings
- Optic Atrophy, Hereditary, Leber
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
rAAV2-ND4Drug
Also called rAAV2-ND4 gene therapy
injection
| Study group | Role | What they receive |
|---|---|---|
| rAAV2-ND4 | Experimental | injection Receives: Drug: rAAV2-ND4 |
What is being measured
The primary outcome is the main question the study is designed to answer.
The Best Corrected Visual Acuity(BCVA)
Measured: Up to 3 years
Results of CD3/CD4/CD8 Test
The mean percentage of CD3+/CD4+/CD8+ test before and after treatment
Measured: up to 6 months
Secondary outcomes (5)
Intraocular Pressure;
Measured: Up to 3 years
Neutralizing Antibody Assay
The mean of Neutralizing antibody assay of 8 patients before and after treatment
Measured: up to 3 years
Average RNFL Thickness Througth Optical Coherence Tomography(OCT) Test
Average RNFL thickness of 8 patients througth Optical coherence tomography(OCT) test before and after treatment
Measured: Up to 3 years
Computerized Visual Field(MD: Mean Deviation, the Value Close to 0 Regarded Normal)
MD: mean deviation, the value Close to 0 regarded normal.VFI/MD：The bigger one was more close to the normal value.
Measured: up to 3 years
Computerized Visual Field(VFI: Visual Field Index ,the Value Close to 100% Regarded Normal)
VFI: visual field index ,the value Close to 100% regarded normal. VFI/MD：The bigger one was more close to the normal value.
Measured: up to 3 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted28 Dec 2010 (estimated)
- Study startApr 2011
- Primary completionNov 2015
- Study completionNov 2015
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (32)Papers the sponsor has linked to this study.
- Other trials for Leber Hereditary Optic NeuropathyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- Bin Li
- Sponsor type
- University, hospital or other
- Collaborators
- Huazhong University of Science and Technology
- Responsible party
- Sponsor-investigator, Bin Li, Huazhong University of Science and Technology
- Investigators
- bin Li, PhD,MD, Study chair, Deputy Director of Ophthalmology
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01267422
- Sponsor's protocol ID
- RAVCT-2
- Acronym
- rAAV2-ND4
- Status last verified
- Jan 2018
- First submitted
- 27 Dec 2010
- Last updated
- 31 Jan 2018
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Carelli V, Ross-Cisneros FN, Sadun AA. Mitochondrial dysfunction as a cause of optic neuropathies. Prog Retin Eye Res. 2004 Jan;23(1):53-89. doi: 10.1016/j.preteyeres.2003.10.003.
PubMed 14766317Mackey DA, Oostra RJ, Rosenberg T, Nikoskelainen E, Bronte-Stewart J, Poulton J, Harding AE, Govan G, Bolhuis PA, Norby S. Primary pathogenic mtDNA mutations in multigeneration pedigrees with Leber hereditary optic neuropathy. Am J Hum Genet. 1996 Aug;59(2):481-5. No abstract available.
PubMed 8755941Wallace DC, Singh G, Lott MT, Hodge JA, Schurr TG, Lezza AM, Elsas LJ 2nd, Nikoskelainen EK. Mitochondrial DNA mutation associated with Leber's hereditary optic neuropathy. Science. 1988 Dec 9;242(4884):1427-30. doi: 10.1126/science.3201231.
PubMed 3201231Oguchi Y. [Past, present, and future in Leber's hereditary optic neuropathy]. Nippon Ganka Gakkai Zasshi. 2001 Dec;105(12):809-27. Japanese.
PubMed 11802455Cui G, Ding H, Xu Y, Li B, Wang DW. Applications of the method of high resolution melting analysis for diagnosis of Leber's disease and the three primary mutation spectrum of LHON in the Han Chinese population. Gene. 2013 Jan 1;512(1):108-12. doi: 10.1016/j.gene.2012.09.110. Epub 2012 Oct 9.
PubMed 23063736Mashima Y, Kigasawa K, Wakakura M, Oguchi Y. Do idebenone and vitamin therapy shorten the time to achieve visual recovery in Leber hereditary optic neuropathy? J Neuroophthalmol. 2000 Sep;20(3):166-70. doi: 10.1097/00041327-200020030-00006.
PubMed 11001192Carelli V, Barboni P, Zacchini A, Mancini R, Monari L, Cevoli S, Liguori R, Sensi M, Lugaresi E, Montagna P. Leber's Hereditary Optic Neuropathy (LHON) with 14484/ND6 mutation in a North African patient. J Neurol Sci. 1998 Oct 8;160(2):183-8. doi: 10.1016/s0022-510x(98)00239-1.
PubMed 9849804Barnils N, Mesa E, Munoz S, Ferrer-Artola A, Arruga J. [Response to idebenone and multivitamin therapy in Leber's hereditary optic neuropathy]. Arch Soc Esp Oftalmol. 2007 Jun;82(6):377-80. doi: 10.4321/s0365-66912007000600012. Spanish.
PubMed 17573650Angebault C, Gueguen N, Desquiret-Dumas V, Chevrollier A, Guillet V, Verny C, Cassereau J, Ferre M, Milea D, Amati-Bonneau P, Bonneau D, Procaccio V, Reynier P, Loiseau D. Idebenone increases mitochondrial complex I activity in fibroblasts from LHON patients while producing contradictory effects on respiration. BMC Res Notes. 2011 Dec 22;4:557. doi: 10.1186/1756-0500-4-557.
PubMed 22192149Klopstock T, Yu-Wai-Man P, Dimitriadis K, Rouleau J, Heck S, Bailie M, Atawan A, Chattopadhyay S, Schubert M, Garip A, Kernt M, Petraki D, Rummey C, Leinonen M, Metz G, Griffiths PG, Meier T, Chinnery PF. A randomized placebo-controlled trial of idebenone in Leber's hereditary optic neuropathy. Brain. 2011 Sep;134(Pt 9):2677-86. doi: 10.1093/brain/awr170. Epub 2011 Jul 25.
PubMed 21788663Carelli V, La Morgia C, Valentino ML, Rizzo G, Carbonelli M, De Negri AM, Sadun F, Carta A, Guerriero S, Simonelli F, Sadun AA, Aggarwal D, Liguori R, Avoni P, Baruzzi A, Zeviani M, Montagna P, Barboni P. Idebenone treatment in Leber's hereditary optic neuropathy. Brain. 2011 Sep;134(Pt 9):e188. doi: 10.1093/brain/awr180. Epub 2011 Aug 2. No abstract available.
PubMed 21810891Sadun AA, Chicani CF, Ross-Cisneros FN, Barboni P, Thoolen M, Shrader WD, Kubis K, Carelli V, Miller G. Effect of EPI-743 on the clinical course of the mitochondrial disease Leber hereditary optic neuropathy. Arch Neurol. 2012 Mar;69(3):331-8. doi: 10.1001/archneurol.2011.2972.
PubMed 22410442Newman NJ. Treatment of Leber hereditary optic neuropathy. Brain. 2011 Sep;134(Pt 9):2447-50. doi: 10.1093/brain/awr192. Epub 2011 Aug 22. No abstract available.
PubMed 21859767Maguire AM, Simonelli F, Pierce EA, Pugh EN Jr, Mingozzi F, Bennicelli J, Banfi S, Marshall KA, Testa F, Surace EM, Rossi S, Lyubarsky A, Arruda VR, Konkle B, Stone E, Sun J, Jacobs J, Dell'Osso L, Hertle R, Ma JX, Redmond TM, Zhu X, Hauck B, Zelenaia O, Shindler KS, Maguire MG, Wright JF, Volpe NJ, McDonnell JW, Auricchio A, High KA, Bennett J. Safety and efficacy of gene transfer for Leber's congenital amaurosis. N Engl J Med. 2008 May 22;358(21):2240-8. doi: 10.1056/NEJMoa0802315. Epub 2008 Apr 27.
PubMed 18441370Bainbridge JW, Smith AJ, Barker SS, Robbie S, Henderson R, Balaggan K, Viswanathan A, Holder GE, Stockman A, Tyler N, Petersen-Jones S, Bhattacharya SS, Thrasher AJ, Fitzke FW, Carter BJ, Rubin GS, Moore AT, Ali RR. Effect of gene therapy on visual function in Leber's congenital amaurosis. N Engl J Med. 2008 May 22;358(21):2231-9. doi: 10.1056/NEJMoa0802268. Epub 2008 Apr 27.
PubMed 18441371
Study at a glance
From the registry record, last updated by the sponsor on 31 Jan 2018. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Safety and Efficacy Study of a Single Intravitreal Injection of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy
- ClinicalTrials.gov ID
- NCT01267422
- Acronym / sponsor ID
- rAAV2-ND4 / RAVCT-2
- Conditions
- Leber Hereditary Optic Neuropathy
- Keywords
- Leber's Hereditary Optic Neuropathy,11778 LHON mutation,, rAAV2-ND4,triamcinolone acetonide
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- Bin LiUniversity, hospital or other
- Collaborators
- Huazhong University of Science and Technology
- Study start
- Apr 2011
- Primary completion
- Nov 2015 (actual)
- Study completion
- Nov 2015 (actual)
- First posted
- 28 Dec 2010 (estimated)
- Results
- Posted 2 May 2016
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
This study is meant to assess the safety and efficacy of rAAV2-ND4 treatment of Leber hereditary optic neuropathy with 11778 LHON mutation.
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)（0.05ml).The dose was 5 × 10^9 vg/0.05 mL for patients younger than 12 years old, and 1 × 10^10 vg/0.05 mL for patients older than 12 years old. The visual acuity, visual evoked potential （VEP）,optical coherence tomography（ OCT）, computerized visual field, electroretinograms(ERG), retinal nerve fiber layer(RNFL)and Liver and kidney function in plasma were compared before and after treatment at 1,3,and 6, months interval.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| rAAV2-ND4 | Experimental | Drug: rAAV2-ND4injection |
Who may qualify
- Ages
- 8 to 60 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (3 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| rAAV2-ND4Also: rAAV2-ND4 gene therapyinjection | Drug | rAAV2-ND4 |
Primary outcome
The Best Corrected Visual Acuity(BCVA)
Time frame: Up to 3 years
Results of CD3/CD4/CD8 Test
Time frame: up to 6 months
The mean percentage of CD3+/CD4+/CD8+ test before and after treatment
5 secondary outcomes
Intraocular Pressure;
Time frame: Up to 3 years
Neutralizing Antibody Assay
Time frame: up to 3 years
Average RNFL Thickness Througth Optical Coherence Tomography(OCT) Test
Time frame: Up to 3 years
Computerized Visual Field(MD: Mean Deviation, the Value Close to 0 Regarded Normal)
Time frame: up to 3 years
Computerized Visual Field(VFI: Visual Field Index ,the Value Close to 100% Regarded Normal)
Time frame: up to 3 years
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 Jan 2018.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Background: PubMed 14766317Carelli V, Ross-Cisneros FN, Sadun AA. Mitochondrial dysfunction as a cause of optic neuropathies. Prog Retin Eye Res. 2004 Jan;23(1):53-89. doi: 10.1016/j.preteyeres.2003.10.003.
- Background: PubMed 8755941Mackey DA, Oostra RJ, Rosenberg T, Nikoskelainen E, Bronte-Stewart J, Poulton J, Harding AE, Govan G, Bolhuis PA, Norby S. Primary pathogenic mtDNA mutations in multigeneration pedigrees with Leber hereditary optic neuropathy. Am J Hum Genet. 1996 Aug;59(2):481-5. No abstract available.
- Background: PubMed 3201231Wallace DC, Singh G, Lott MT, Hodge JA, Schurr TG, Lezza AM, Elsas LJ 2nd, Nikoskelainen EK. Mitochondrial DNA mutation associated with Leber's hereditary optic neuropathy. Science. 1988 Dec 9;242(4884):1427-30. doi: 10.1126/science.3201231.
- Background: PubMed 11802455Oguchi Y. [Past, present, and future in Leber's hereditary optic neuropathy]. Nippon Ganka Gakkai Zasshi. 2001 Dec;105(12):809-27. Japanese.
- Background: PubMed 23063736Cui G, Ding H, Xu Y, Li B, Wang DW. Applications of the method of high resolution melting analysis for diagnosis of Leber's disease and the three primary mutation spectrum of LHON in the Han Chinese population. Gene. 2013 Jan 1;512(1):108-12. doi: 10.1016/j.gene.2012.09.110. Epub 2012 Oct 9.
- Background: PubMed 11001192Mashima Y, Kigasawa K, Wakakura M, Oguchi Y. Do idebenone and vitamin therapy shorten the time to achieve visual recovery in Leber hereditary optic neuropathy? J Neuroophthalmol. 2000 Sep;20(3):166-70. doi: 10.1097/00041327-200020030-00006.
- Background: PubMed 9849804Carelli V, Barboni P, Zacchini A, Mancini R, Monari L, Cevoli S, Liguori R, Sensi M, Lugaresi E, Montagna P. Leber's Hereditary Optic Neuropathy (LHON) with 14484/ND6 mutation in a North African patient. J Neurol Sci. 1998 Oct 8;160(2):183-8. doi: 10.1016/s0022-510x(98)00239-1.
- Background: PubMed 17573650Barnils N, Mesa E, Munoz S, Ferrer-Artola A, Arruga J. [Response to idebenone and multivitamin therapy in Leber's hereditary optic neuropathy]. Arch Soc Esp Oftalmol. 2007 Jun;82(6):377-80. doi: 10.4321/s0365-66912007000600012. Spanish.
- Background: PubMed 22192149Angebault C, Gueguen N, Desquiret-Dumas V, Chevrollier A, Guillet V, Verny C, Cassereau J, Ferre M, Milea D, Amati-Bonneau P, Bonneau D, Procaccio V, Reynier P, Loiseau D. Idebenone increases mitochondrial complex I activity in fibroblasts from LHON patients while producing contradictory effects on respiration. BMC Res Notes. 2011 Dec 22;4:557. doi: 10.1186/1756-0500-4-557.
- Background: PubMed 21788663Klopstock T, Yu-Wai-Man P, Dimitriadis K, Rouleau J, Heck S, Bailie M, Atawan A, Chattopadhyay S, Schubert M, Garip A, Kernt M, Petraki D, Rummey C, Leinonen M, Metz G, Griffiths PG, Meier T, Chinnery PF. A randomized placebo-controlled trial of idebenone in Leber's hereditary optic neuropathy. Brain. 2011 Sep;134(Pt 9):2677-86. doi: 10.1093/brain/awr170. Epub 2011 Jul 25.
- Background: PubMed 21810891Carelli V, La Morgia C, Valentino ML, Rizzo G, Carbonelli M, De Negri AM, Sadun F, Carta A, Guerriero S, Simonelli F, Sadun AA, Aggarwal D, Liguori R, Avoni P, Baruzzi A, Zeviani M, Montagna P, Barboni P. Idebenone treatment in Leber's hereditary optic neuropathy. Brain. 2011 Sep;134(Pt 9):e188. doi: 10.1093/brain/awr180. Epub 2011 Aug 2. No abstract available.
- Background: PubMed 22410442Sadun AA, Chicani CF, Ross-Cisneros FN, Barboni P, Thoolen M, Shrader WD, Kubis K, Carelli V, Miller G. Effect of EPI-743 on the clinical course of the mitochondrial disease Leber hereditary optic neuropathy. Arch Neurol. 2012 Mar;69(3):331-8. doi: 10.1001/archneurol.2011.2972.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
bin Li, PhD,MD study chair
Deputy Director of Ophthalmology
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing rAAV2-ND4 in people with Leber Hereditary Optic Neuropathy, run by Bin Li at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 31 Jan 2018. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Yes. Results are posted in the registry (first posted 2 May 2016). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The Best Corrected Visual Acuity(BCVA) (time frame: Up to 3 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01267422, which also lists 32 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 Jan 2018.