9 completed, 1 terminated. 5 Phase 1, 4 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 5 | Completed 5 |
| Phase 2 | 4 | Completed 4 |
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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 |
|---|---|---|---|---|---|---|
| Gene Therapy for Achromatopsia (CNGA3) adeno-associated virus vector AAV- CNGA3 - Achromatopsia | Phase 1 / Phase 2 | Completed | MeiraGTx UK II Ltd | 11 | 2019-08-12 | 2022-12-01 |
| A Phase 1 Open-Label, Dose Escalation Study to Determine the Optimal Dose, Safety, and Activity of AAV2hAQP1 in Subjects With Radiation-Induced Parotid Gland Hypofunction and Xerostomia AAV2hAQP1: 1 x 10^11 vg/gland (single gland), AAV2hAQP1: 3 x 10^10 vg/gland (both glands), AAV2hAQP1: 3 x 10^11 vg/gland (single gland) - Radiation-Induced Parotid Gland Hypofunction, Xerostomia Due to Radiotherapy | Phase 1 | Completed | MeiraGTx UK II Ltd | 24 | 2019-06-30 | 2023-04-24 |
| Natural History Study of Patients With X-linked Retinal Dystrophy Associated With Mutations in Retinitis Pigmentosa GTPase Regulator (RPGR) Retinitis Pigmentosa | Completed | MeiraGTx UK II Ltd | 140 | 2017-12-19 | 2024-06-20 | |
| Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) - Retinitis Pigmentosa GTPase Regulator (RPGR) AAV5-RPGR - X-Linked Retinitis Pigmentosa | Phase 1 / Phase 2 | Completed | MeiraGTx UK II Ltd | 49 | 2017-07-31 | 2025-01-08 |
| Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3 Prior exposure to AAV-CNGA3 or AAV-CNGB3 - Achromatopsia | Terminated | MeiraGTx UK II Ltd | 34 | 2017-06-29 | 2025-06-11 | |
| Gene Therapy for Achromatopsia (CNGB3) AAV - CNGB3 - Achromatopsia | Phase 1 / Phase 2 | Completed | MeiraGTx UK II Ltd | 23 | 2017-01-16 | 2023-03-08 |
| Long-Term Follow-Up Gene Therapy Study for Leber Congenital Amaurosis OPTIRPE65 (Retinal Dystrophy Associated With Defects in RPE65) AAV OPTIRPE65 - Leber Congenital Amaurosis (LCA), Eye Diseases | Completed | MeiraGTx UK II Ltd | 15 | 2016-11-01 | 2024-04-11 | |
| Natural History Study of Patients With Leber Congenital Amaurosis Associated With Mutations in RPE65 Leber Congenital Amaurosis | Completed | MeiraGTx UK II Ltd | 37 | 2016-04-01 | 2026-09-21 | |
| Clinical Trial of Gene Therapy for the Treatment of Leber Congenital Amaurosis (LCA) AAV RPE65 - Leber Congenital Amaurosis | Phase 1 / Phase 2 | Completed | MeiraGTx UK II Ltd | 15 | 2016-04-01 | 2021-07-12 |
| Natural History Study for Achromatopsia Ocular assessments - Achromatopsia | Completed | MeiraGTx UK II Ltd | 85 | 2011-09-13 | 2023-06-27 |
Color Vision Defects (4) · Leber Congenital Amaurosis (3) · Eye Diseases (1) · Eye Diseases, Hereditary (1) · Head and Neck Neoplasms (1) · Retinal Diseases (1) · Retinitis Pigmentosa (1)
United Kingdom (9) · United States (9) · Canada (2)
James Bainbridge, Prof (2) · James Bainbridge (1) · Michel Michaelides, Prof (1) · Michel Michealides, Prof (1)
PMID 30025081, PMID 32347917, PMID 37172884, PMID 38871269
2016 (3)