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Long-Term Follow-Up After Unilateral Intravitreal Gene Therapy for Leber Hereditary Optic Neuropathy: The RESTORE Study
Abstract
Background: RESCUE and REVERSE were 2 Phase 3 clinical trials that assessed the efficacy and safety of intravitreal gene therapy with lenadogene nolparvovec (rAAV2/2- ND4 ) for the treatment of Leber hereditary optic neuropathy (LHON). RESTORE is the long-term follow-up study of subjects treated in the RESCUE and REVERSE trials. Methods: In RESCUE and REVERSE, 76 subjects with LHON because of the m.11778 G>A mutation in the mitochondrial gene ND4 received a single unilateral intravitreal injection of lenadogene nolparvovec. After 96 weeks, 61 subjects were enrolled in the long-term follow-up study RESTORE. The best-corrected visual acuity (BCVA) was assessed over a period of up to 52 months after onset of vision loss. A locally estimated scatterplot smoothing regression model was used to analyze changes in BCVA over time. Vision-related quality of life was reported using the visual function questionnaire-25 (VFQ-25). Results: The population of MT-ND4 subjects enrolled in RESTORE was representative of the combined cohorts of RESCUE and REVERSE for mean age (35.1 years) and gender distribution (79% males). There was a progressive and sustained improvement of BCVA up to 52 months after the onset of vision loss. The final mean BCVA was 1.26 logarithm of the minimal angle of resolution 48 months after the onset of vision loss. The mean VFQ-25 composite score increased by 7 points compared with baseline. Conclusion: The treatment effect of lenadogene nolparvovec on BCVA and vision-related quality of life observed 96 weeks (2 years) after treatment in RESCUE and REVERSE was sustained at 3 years in RESTORE, with a maximum follow-up of 52 months (4.3 years) after the onset of vision loss.
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“GenSight Biologics has also completed two phase 3 clinical trials to treat blindness caused by mtND4 mutations (ClinicalTrials.gov, Identifiers NCT03293524 and NCT02064569) and recently reported sustained positive results from the RESTORE study [177], though at the time of this writing neither study has reported results in any clinical trial registries.”
From The Mitochondrial Genome in Aging and Disease and the Future of Mitochondrial Therapeutics (2022) · cites this paper as [177]
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Cites
A randomized placebo-controlled trial of idebenone in Leber’s hereditary optic neuropathy.
Klopstock, T., Yu-Wai-Man, P., Dimitriadis, K., Rouleau, J., Heck, S., Bailie, M., Atawan, A., Chattopadhyay, S., Schubert, M., Garip, A., et al. (2011) · Brain · doi:10.1093/brain/awr170 · Cited by 578
Self-citationYu-Wai-Man, P., Newman, N. J., Carelli, V., Moster, M. L., Biousse, V., Sadun, A. A., Klopstock, T., Vignal-Clermont, C., Sergott, R. C., Rudolph, G., et al. (2020) · Science Translational Medicine · doi:10.1126/scitranslmed.aaz7423 · Cited by 232
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Self-citationNewman, N. J., Carelli, V., Taiel, M., & Yu-Wai-Man, P. (2020) · Journal of Neuro-Ophthalmology · doi:10.1097/wno.0000000000001045 · Cited by 111
Self-citationCwerman-Thibault, H., Augustin, S., Lechauve, C., Ayache, J., Ellouze, S., Sahel, J.-A., & Corral-Debrinski, M. (2015) · Molecular Therapy - Methods & Clinical Development · doi:10.1038/mtm.2015.3 · Cited by 86 · cited 1×
“In rodent models of LHON with ND4-induced complex I deficiency, lenadogene nolparvovec prevented optic atrophy and vision loss (8).”
BackgroundSelf-citationVignal-Clermont, C., Girmens, J.-F., Audo, I., Said, S. M., Errera, M.-H., Plaine, L., O’Shaughnessy, D., Taiel, M., & Sahel, J.-A. (2021) · BioDrugs · doi:10.1007/s40259-021-00468-9 · Cited by 51
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Newman, N. J., Yu-Wai-Man, P., Biousse, V., & Carelli, V. (2023) · The Lancet Neurology · doi:10.1016/s1474-4422(22)00174-0 · Cited by 97
Self-citationRandomized trial of bilateral gene therapy injection for m.11778G>A MT-ND4 Leber optic neuropathy
Newman, N. J., Yu-Wai-Man, P., Subramanian, P. S., Moster, M. L., Wang, A.-G., Donahue, S. P., Leroy, B. P., Carelli, V., Biousse, V., Vignal-Clermont, C., et al. (2023) · Brain · doi:10.1093/brain/awac421 · Cited by 87
“Patients who completed the RESCUE and REVERSE studies are being followed in the ongoing extension study, RESTORE, and results at 3 years posttreatment have been reported.(20)”
InfluentialSelf-citationUpdate on viral gene therapy clinical trials for retinal diseases
Cheng, S.-Y., & Punzo, C. (2022) · Human Gene Therapy · doi:10.1089/hum.2022.159 · Cited by 49
Biodistribution of intravitreal lenadogene nolparvovec gene therapy in nonhuman primates
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Self-citationVIGNAL-CLERMONT, C., YU-WAI-MAN, P., NEWMAN, N. J., CARELLI, V., MOSTER, M. L., BIOUSSE, V., SUBRAMANIAN, P. S., WANG, A.-G., DONAHUE, S. P., LEROY, B. P., et al. (2023) · American Journal of Ophthalmology · doi:10.1016/j.ajo.2022.11.026 · Cited by 25
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Metadata
- Authors
- Biousse, V., Newman, N. J., Yu-Wai-Man, P., Carelli, V., Moster, M. L., Vignal-Clermont, C., Klopstock, T., Sadun, A. A., Sergott, R. C., Hage, R., Esposti, S., La Morgia, C., Priglinger, C., Karanja, R., Blouin, L., Taiel, M., Sahel, J.-A., & for the LHON Study Group
- Venue
- Journal of Neuro-Ophthalmology
- Published
- 2020-08-28
- Volume
- 41
- Issue
- 3
- Pages
- 309-315
- ISSN
- 1070-8022
- PMC
- PMC8366761
- Cited by
- 78
- References
- 8
- Language
- en
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