Map Active LHON Clinical Trials in Asia-Pacific
A comprehensive database of 10 LHON clinical trials across 5 Asia-Pacific countries, with 611+ total patients enrolled.
10
Total trials
611+
Patients enrolled
5
Countries
6
Gene therapy trials
Clinical Trials Database
| Trial | Country | Phase | Status | Enrollment | Intervention |
|---|---|---|---|---|---|
| GOLD Trial — NR082 Gene Therapy NCT04912843 · Wuhan Neurophth Biotechnology | China | 1/2/3 | Recruiting | 102 | NR082 (AAV2-ND4 gene therapy) ND4 (G11778A) |
| NR082 Phase 3 Bilateral IVT NCT07406854 · Wuhan Neurophth Biotechnology | China | 3 | Active, not recruiting | 95 | NR082 bilateral intravitreal injection ND4 (G11778A) |
| rAAV2-ND4 for LHON NCT03153293 · Huazhong University of Science and Technology | China | 2/3 | Unknown | 159 | rAAV2-ND4 gene therapy ND4 (G11778A) |
| rAAV2-ND4 for Acute LHON NCT03428178 · Bin Li / Huazhong University | China | 2/3 | Unknown | 120 | rAAV2-ND4 gene therapy (acute onset) ND4 (G11778A) |
| NFS-02 for ND1 LHON NCT05820152 · Neurophth Therapeutics Inc | China / United States | 1/2 | Terminated | 11 | NFS-02 (ND1-targeting gene therapy) ND1 (G3460A) |
| First LHON Gene Therapy Trial NCT01267422 · Bin Li / Huazhong University | China | 1/2 | Completed | 9 | rAAV2-ND4 gene therapy (world's first) ND4 (G11778A) |
| REFLECT Trial — GS010/LUMEVOQ NCT03293524 · GenSight Biologics | Taiwan + 6 countries | 3 | Completed | 98 | GS010 (lenadogene nolparvovec / LUMEVOQ) ND4 (G11778A) |
| Curcumin for LHON NCT00528151 · Ramathibodi Hospital, Thailand | Thailand | 2 | Completed | 70 | Curcumin (neuroprotective supplement) Multiple |
| Idebenone for Japanese LHON Patients UMIN000017939 · Japanese LHON Group | Japan | 3 | Completed | 57 | Idebenone Multiple |
| DdCBE Mitochondrial Base Editing SNU-2025 · Seoul National University | South Korea | Preclinical | Preclinical | — | DdCBE (dual-deaminase base editor) for mtDNA correction Multiple mtDNA mutations |
Sources & Methodology
Data compiled from ClinicalTrials.gov API, UMIN Clinical Trials Registry (Japan), PubMed cross-references, and Nature Communications publications. All ClinicalTrials.gov entries were verified against the official registry.