5 completed, 2 recruiting, 2 unknown, 1 active not recruiting. 6 Phase 1, 6 Phase 2, 1 Early Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 6 | Completed 3, Unknown 2, Active not recruiting 1 |
| Phase 2 | 6 | Completed 3, Unknown 2, Active not recruiting 1 |
| Early Phase 1 | 1 | Suspended 1 |
Follow Achromatopsia: new trials by email Literature Review of Achromatopsia trials Research Report on Achromatopsia
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 |
|---|---|---|---|---|---|---|
| Natural History Study of Inherited Retinal Diseases Retinal Dystrophies, Color Vision Defects | Recruiting | Zhongmou Therapeutics | 200 | 2025-07-20 | 2025-12-10 | |
| PBA Use for Treatment of ATF6-/- Patients PBA - ACHROMATOPSIA 7, Achromatopsia | Early Phase 1 | Suspended | Columbia University | 2 | 2025-04-08 | 2026-09-11 |
| 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 |
| Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGA3 Achromatopsia (A Clarity Clinical Trial) AGTC-402 - Achromatopsia | Phase 1 / Phase 2 | Unknown | Beacon Therapeutics | 24 | 2017-08-03 | 2022-07-22 |
| 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 |
| Safety and Efficacy Trial of AAV Gene Therapy in Patients With CNGB3 Achromatopsia (A Clarity Clinical Trial) rAAV2tYF-PR1.7-hCNGB3 - Achromatopsia | Phase 1 / Phase 2 | Unknown | Beacon Therapeutics | 32 | 2016-04-11 | 2022-07-22 |
| Safety and Efficacy of rAAV.hCNGA3 Gene Therapy in Patients With CNGA3-linked Achromatopsia rAAV.hCNGA3 - Achromatopsia | Phase 1 / Phase 2 | Active not recruiting | STZ eyetrial | 13 | 2015-11-01 | 2024-04-18 |
| Inherited Retinal Degenerative Disease Registry Eye Diseases Hereditary, Retinal Disease | Recruiting | Foundation Fighting Blindness | 20000 | 2014-06-01 | 2026-05-19 | |
| Clinical and Genetic Characterization of Individuals With Achromatopsia Achromatopsia | Completed | Beacon Therapeutics | 56 | 2013-06-01 | 2017-10-13 | |
| CNTF Implants for CNGB3 Achromatopsia NT-501 CNTF-releasing implant - Eye Disease, Achromatopsia | Phase 1 / Phase 2 | Completed | National Eye Institute (NEI) | 5 | 2012-07-01 | 2016-11-21 |
| Natural History Study for Achromatopsia Ocular assessments - Achromatopsia | Completed | MeiraGTx UK II Ltd | 85 | 2011-09-13 | 2023-06-27 |
Color Vision Defects (12) · Achromatopsia 3 (2) · Eye Diseases (2) · Vision Disorders (2) · Abetalipoproteinemia (1) · Bardet-Biedl Syndrome (1) · Charcot-Marie-Tooth Disease (1) · Choroideremia (1) · Cone Dystrophy (1) · Cone-Rod Dystrophies (1) · Enhanced S-Cone Syndrome (1) · Eye Diseases, Hereditary (1) · Fundus Albipunctatus (1) · Gyrate Atrophy (1) · Kearns-Sayre Syndrome (1)
Pathological Conditions, Signs and Symptoms (12) · Cone Dystrophy (11) · Eye Diseases, Hereditary (11) · Nervous System Diseases (11) · Neurologic Manifestations (11) · Sensation Disorders (11) · Signs and Symptoms (11) · Eye Diseases (10) · Vision Disorders (10) · Retinal Degeneration (2) · Abnormalities, Multiple (1) · Blindness (1) · Brain Diseases (1) · Brain Diseases, Metabolic (1) · Brain Diseases, Metabolic, Inborn (1)
MeiraGTx UK II Ltd (4) · Beacon Therapeutics (3) · Columbia University (1) · Foundation Fighting Blindness (1) · National Eye Institute (NEI) (1) · STZ eyetrial (1) · Zhongmou Therapeutics (1)
United States (9) · United Kingdom (4) · China (1) · Germany (1) · Israel (1)
David Jacobs, MD, MBA (2) · Dominik Fischer, Prof. (1) · James Bainbridge (1) · James Bainbridge, Prof (1) · Matt Feinsod, MD (1) · Paul A Sieving, MD, PhD (1) · Stephen Tsang, MD (1) · Todd Durham, PhD (1) · Yin Shen (1)
PMID 25205868, PMID 27479814, PMID 30194931, PMID 32352493, PMID 34006508, PMID 37172884
2016 (2), R24EY022023 (2), 2014 (1)