21 completed, 7 recruiting, 5 terminated, 3 active not recruiting. 15 Phase 2, 11 Phase 3, 9 Phase 1, 5 Not applicable, 1 Phase 4.
Follow Optic Atrophy, Hereditary, Leber: new trials by email Literature Review of Optic Atrophy, Hereditary, Leber trials Research Report on Optic Atrophy, Hereditary, Leber
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Pilot Study of the Efficacy of Nicotinamide (Vitamin B3) in Leber's Hereditary Optic Neuropathy Nicotinamide treatment - Leber Hereditary Optic Neuropathy (LHON), Leber's Hereditary Optic Neuropathy (LHON) | Phase 1 | Not yet recruiting | University Hospital, Angers | 13 | 2026-04-01 | 2025-12-18 |
| Efficacy and Safety Study of Bilateral IVT Injection of GS010 at Two Dose Levels in LHON Patients GS010 High dose, GS010 Low dose - Leber Hereditary Optic Disease | Phase 2 | Recruiting | GenSight Biologics | 14 | 2026-01-22 | 2026-03-02 |
| Optic Nerve Head Evaluation Through Multimodal Blood Flow Analysis: a Prospective Observational, Multi-center Study Neuro-Ophthalmology, Optic Neuropathies | Not yet recruiting | Barboni | 70 | 2025-10-01 | 2025-07-20 | |
| Study to Evaluate Sepofarsen in Subjects With Leber Congenital Amaurosis (LCA) Type 10 (HYPERION) sepofarsen, Placebo IVT - Leber Congenital Amaurosis 10, Blindness | Phase 3 | Recruiting | Laboratoires Thea | 32 | 2025-06-04 | 2026-10-21 |
| A Phase 3, Multicenter, Randomized, Double-Masked, Sham-Controlled Clinical Trial for Leber's Hereditary Optic Neuropathy (LHON) Associated With ND4 Mutation rAAV2-ND4, Sham (No Treatment) - Leber Hereditary Optic Neuropathy (LHON) | Phase 3 | Active not recruiting | Wuhan Neurophth Biotechnology Limited Company | 95 | 2024-09-19 | 2026-02-12 |
| Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND1 Mutations NFS-02 Injection - Leber Hereditary Optic Neuropathy (LHON) | Phase 1 / Phase 2 | Terminated | Neurophth Therapeutics Inc | 11 | 2023-08-15 | 2024-09-20 |
| Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND4 Mutations NR082 Injection, Injection needle - Leber Hereditary Optic Neuropathy (LHON) | Phase 1 / Phase 2 | Active not recruiting | Neurophth Therapeutics Inc | 12 | 2023-05-22 | 2024-09-04 |
| Metabolomics Analysis According to the Retinal Nerve Fiber Layer in Patients With NOHL Mutations (MétabOCT) Optical coherent tomography - Healthy Subjects, Leber Hereditary Optic Neuropathy | Not applicable | Recruiting | Hôpital Necker-Enfants Malades | 90 | 2023-03-10 | 2025-11-18 |
| Evaluation of Impact of Disease and Visual Disability on Quality of Life and Loss of Independence of Patients Living in France With Leber's Hereditary Optic Neuropathy (LHON) Through Qualitative and Quantitative Data Collection Leber's Hereditary Optic Neuropathy (LHON) | Completed | Argo Sante | 12 | 2022-08-04 | 2024-02-06 | |
| Gene Therapy Clinical Trial for the Treatment Of Leber's HereDitary Optic Neuropathy NR082 injection, Sham Injection - Leber's Hereditary Optic Neuropathy (LHON) | Phase 2 / Phase 3 | Recruiting | Wuhan Neurophth Biotechnology Limited Company | 102 | 2021-06-18 | 2022-11-14 |
| Extension Study to Study PQ-110-001 (NCT03140969) QR-110 - Leber Congenital Amaurosis 10, Blindness | Phase 1 / Phase 2 | Terminated | Laboratoires Thea | 9 | 2019-05-13 | 2024-10-15 |
| New Non-invasive Modalities for Assessing Retinal Structure and Function LSFG-NAVI - Ischemic Optic Neuropathy, Branch Retinal Artery Occlusion | Not applicable | Recruiting | Randy Kardon | 500 | 2019-05-06 | 2026-09-08 |
| A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE) sepofarsen, Sham - Leber Congenital Amaurosis 10, Blindness | Phase 2 / Phase 3 | Active not recruiting | ProQR Therapeutics | 36 | 2019-04-04 | 2022-03-17 |
| Pupil Dynamics and Color Vision for the Detection of Eye Diseases Dynamic pupillometry sessions - Retinitis Pigmentosa, Leber's Hereditary Optic Neuropathy | Not applicable | Completed | Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts | 60 | 2018-03-22 | 2021-06-01 |
| Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year GS010, Placebo - Leber Hereditary Optic Neuropathy | Phase 3 | Completed | GenSight Biologics | 98 | 2018-03-12 | 2026-04-16 |
| RESCUE and REVERSE Long-term Follow-up GS010, Sham Intravitreal Injection - Leber Hereditary Optic Neuropathy | Phase 3 | Completed | GenSight Biologics | 62 | 2018-01-09 | 2026-03-02 |
| REALITY LHON Registry Patient-reported outcomes (PROs) - Leber Hereditary Optic Neuropathy | Completed | GenSight Biologics | 44 | 2018-01-03 | 2026-03-02 | |
| A Single Intravitreal Injection of rAAV2-ND4 for the Treatment of Leber's Hereditary Optic Neuropathy rAAV2-ND4 - Leber Hereditary Optic Neuropathy | Phase 2 / Phase 3 | Unknown | Huazhong University of Science and Technology | 159 | 2017-12-27 | 2020-09-17 |
| Study to Evaluate QR-110 in Leber's Congenital Amaurosis (LCA) Due to the c.2991+1655A>G Mutation (p.Cys998X) in the CEP290 Gene QR-110 - Leber's Congenital Amaurosis | Phase 1 / Phase 2 | Completed | Laboratoires Thea | 11 | 2017-10-16 | 2024-10-15 |
| Biomechanics of Optic Neuropathy magnetic resonance image (MRI), Optical Coherence Tomography (OCT) - Optic Neuropathy | Terminated | University of Miami | 10 | 2016-09-01 | 2020-10-23 |
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Eye Diseases (11) · Mitochondrial Diseases (10) · Eye Diseases, Hereditary (9) · Genetic Diseases, Inborn (6) · Neurodegenerative Diseases (6) · Nervous System Diseases (5) · Optic Nerve Diseases (5) · Optic Neuropathy, Ischemic (5) · Retinal Diseases (5) · Blindness (4) · Meckel Syndrome, Type 4 (4) · Vision Disorders (4) · Eye Abnormalities (3) · Leber Congenital Amaurosis (3)
Congenital, Hereditary, and Neonatal Diseases and Abnormalities (44) · Cranial Nerve Diseases (44) · Heredodegenerative Disorders, Nervous System (44) · Metabolic Diseases (44) · Nutritional and Metabolic Diseases (44) · Optic Atrophies, Hereditary (43) · Optic Atrophy (42) · Nervous System Diseases (39) · Optic Nerve Diseases (39) · Genetic Diseases, Inborn (38) · Neurodegenerative Diseases (38) · Eye Diseases, Hereditary (35) · Mitochondrial Diseases (34) · Eye Diseases (33) · Pathological Conditions, Signs and Symptoms (9)
idebenone (5) · salicylhydroxamic acid (3) · Cysteamine (2) · Curcumin (1) · Cyclosporine (1) · Magnetic Resonance Imaging (1) · Matrilin Proteins (1) · Needles (1) · Ophthalmic Solutions (1) · Patient Reported Outcome Measures (1) · RNA, Small Interfering (1) · Syringes (1) · Tablets (1) · Therapeutics (1) · Tomography, Optical Coherence (1)
GenSight Biologics (8) · Santhera Pharmaceuticals (8) · Laboratoires Thea (3) · Amgen (2) · Neurophth Therapeutics Inc (2) · University Hospital, Angers (2) · Wuhan Neurophth Biotechnology Limited Company (2) · Argo Sante (1) · Barboni (1) · Bin Li (1) · Byron Lam (1) · Centre Hospitalier National d'Ophtalmologie des Quinze-Vingts (1) · Columbia University (1) · Edison Pharmaceuticals Inc (1) · Huazhong University of Science and Technology (1)
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Nancy Newman, MD (2) · Alfredo Sadun, MD, PhD (1) · Bin Li, MD (1) · Bin Li, PhD,MD (1) · Byron Lam, MD (1) · Byron R Lam, MD (1) · Christophe Orssaud, MD (1) · Harry T Whelan, MD (1) · Jeffrey Weiss, MD (1) · Jose-Alain Sahel, MD (1) · Marieke Podevin, PhD (1) · Michio Hirano, MD (1)
PMID 36449262, PMID 34108929, PMID 26606867, PMID 26892229, PMID 1463007, PMID 1674640, PMID 18441370, PMID 18441371, PMID 18774912, PMID 1928099, PMID 21788663, PMID 22612072, PMID 22644094, PMID 23063736, PMID 23263355, PMID 23388409, PMID 24396955, PMID 24398093, PMID 24457989, PMID 25342621, PMID 25950380, PMID 26199618, PMID 26604914, PMID 27386292, PMID 27426279, PMID 27631475, PMID 27749593, PMID 27857759, PMID 27904503, PMID 28647203, PMID 29270420, PMID 29454364, PMID 30050918, PMID 30194931, PMID 30559420, PMID 30878986, PMID 31119146, PMID 31215818, PMID 31237206, PMID 31620478, PMID 3201231, PMID 32039263, PMID 32231088, PMID 33451738, PMID 33546345, PMID 33795869, PMID 35271811, PMID 35344016, PMID 35379979, PMID 37716450
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