Table 2:
Current Ongoing Clinical Trials for Mitochondrial Allotopic Gene Replacement Therapy for LHON
| Clinical Trial | Study | Disease | Gene/Treatment | Phase | Endpoint | Sponsor |
|---|---|---|---|---|---|---|
| NCT02652767 | Efficacy Study of GS010 for the Treatment of Vision Loss up to 6 Months From Onset in LHON Due to the ND4 Mutation (RESCUE) | LHON | rAAV2/2-ND4, intravitreal injection | Phase 3 | Visual Acuity, High Resolution Spectral Domain Optical Coherence Tomography to Measure Optic Nerve RNFL Thickness/Volume, Color Hue Vision Test, Immune Response | Gensight Biologics, Paris, France |
| NCT03293524 | Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year (REFLECT) | LHON | GS010 | Phase 3 | Visual acuity, Adverse Effects | Gensight Biologics, Paris, France |
| NCT02064569 | Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients | LHON | GS010 (rAAV2/2-ND4) | Phase 1 | Safety and Tolerability | Gensight Biologics, Paris, France |
| NCT03406104 | RESCUE and REVERSE Long-term Follow-up (RESCUE/REVERSE) | LHON | GS010 | Phase 3 | Adverse Events, Visual Acuity, GCL Thickness/Volume | Gensight Biologics, Paris, France |
| NCT02161380 | Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber’s Hereditary Optic Neuropathy (LHON) | LHON | scAAV2-P1ND4v2 (various titers) | Phase 1 | Toxicity | University of Miami, Miami, FL USA |
| NCT03153293 | Safety and Efficacy Study of Gene Therapy for The Treatment of Leber’s Hereditary Optic Neuropathy | LHON | rAAV2-ND4 | Phase 2/3 | BCVA, RNFL Thickness/Volume, Liver and Kidney Function in Plasma | Huazhong University of Science and Technology, Wuhan, China |
| NCT03153293 | Safety and Efficacy Study of Gene Therapy for The Treatment of Leber’s Hereditary Optic Neuropathy | LHON | rAAV2-ND4 | Phase 2 | Visual acuity, RNFL Thickness/Volume, VEP, Liver and Kidney Function in Plasma | Huazhong University of Science and Technology, Wuhan, China |
LHON: Leber’s Hereditary Optic Neuropathy; RNFL: Retinal Nerve Fiber Layer; GCL: Ganglion Cell Layer; BCVA: Best Corrected Visual Acuity; VEP: Visual Evoked Potential