| NCT Number | Trial Name | Therapy | Type | Phase | Enrollment | Status | Sponsor |
|---|---|---|---|---|---:|---|---|
| NCT03872479 | BRILLIANCE | EDIT-101 | CRISPR gene editing | Phase 1/2 | 34 | Unknown / later reported as paused in secondary sources | Editas Medicine, Inc. (pqac-00000021, pqac-00000024, pqac-00000027) |
| NCT03140969 | PQ-110-001 | Sepofarsen (QR-110) | Antisense oligonucleotide (ASO) | Phase 1b/2 | 12 | Completed | ProQR Therapeutics (pqac-00000018, pqac-00000025) |
| NCT03913143 | ILLUMINATE | Sepofarsen (QR-110) | Antisense oligonucleotide (ASO) | Phase 2/3 | 36 | Active, not recruiting | ProQR Therapeutics (pqac-00000024) |
| NCT03913130 | Extension study to PQ-110-001 | Sepofarsen | Antisense oligonucleotide (ASO) | Phase 1/2 | 9 | Terminated | Laboratoires Théa (pqac-00000025) |
| NCT04855045 | Pediatric sepofarsen study | Sepofarsen | Antisense oligonucleotide (ASO) | Phase 2/3 | 15 | Unknown | ProQR Therapeutics (pqac-00000025) |
| NCT06891443 | HYPERION | Sepofarsen | Antisense oligonucleotide (ASO) | Phase 3 | 32 | Recruiting | Laboratoires Théa (pqac-00000025) |
| NCT03396042 | Natural History Study of CEP290-Related Retinal Degeneration | None (observational) | Natural history / observational | N/A | 26 | Completed | Editas Medicine, Inc. (pqac-00000025) |


*Table: This table summarizes the main registered clinical studies for CEP290-associated Leber congenital amaurosis 10, including interventional therapy trials and the natural history study that informed trial design. It is useful for comparing modalities, development stage, recruitment status, and sponsors across the current LCA10 treatment landscape.*