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| NCT04855045 | An Open-label, Dose Escalation and Double-masked, Randomized, Controlled Trial Evaluating Safety and Tolerability of Sepofarsen in Children (<8 Years of Age) With LCA10 Caused by Mutations in the CEP290 Gene. | UNKNOWN | PHASE2, PHASE3 | 2021-03-23 | 2023-12 | 2023-12 |
| NCT04123626 | A Study to Evaluate the Safety and Tolerability of QR-1123 in Subjects With Autosomal Dominant Retinitis Pigmentosa Due to the P23H Mutation in the RHO Gene | ACTIVE_NOT_RECRUITING | PHASE1, PHASE2 | 2019-10-07 | 2022-06-07 | 2022-06-07 |
| NCT03913143 | A Study to Evaluate Efficacy, Safety, Tolerability and Exposure After a Repeat-dose of Sepofarsen (QR-110) in LCA10 (ILLUMINATE) | ACTIVE_NOT_RECRUITING | PHASE2, PHASE3 | 2019-04-04 | 2023-03 | 2022-01-31 |
| NCT03780257 | Study to Evaluate Safety and Tolerability of QR-421a in Subjects With RP Due to Mutations in Exon 13 of the USH2A Gene | COMPLETED | PHASE1, PHASE2 | 2019-03-06 | 2021-10-14 | 2021-10-14 |
| NCT02564354 | Exploratory Study to Evaluate QR-010 in Subjects With Cystic Fibrosis ΔF508 CFTR Mutation | COMPLETED | PHASE1 | 2015-09 | 2016-09 | 2016-09 |
| NCT02532764 | Dose Escalation Study of QR-010 in Homozygous ΔF508 Cystic Fibrosis Patients | COMPLETED | PHASE1, PHASE2 | 2015-06 | 2017-09-14 | 2017-09-14 |