| NCT ID | Gene | Sponsor | Vector / promoter | Route | Phase | Age eligibility | Enrollment | Status | Primary endpoint(s) | Key secondary endpoints | Reported outcomes / development notes |
|---|---|---|---|---|---|---|---:|---|---|---|---|
| NCT02610582 | CNGA3 | STZ eyetrial | rAAV.hCNGA3; AAV8.hCNGA3 reported in review; promoter not specified in ClinicalTrials.gov chunk | Subretinal injection after pars plana vitrectomy | Phase I/II | 6–12 years and >=18 years; pediatric cohort C n=6 | 13 | Active, not recruiting | Safety at 12 months; adverse events/abnormal labs related to treatment (pqac-00000023, pqac-00000027) | Contrast sensitivity (Pelli Robson) at 6 months; BCVA (ETDRS), microperimetry (MAIA), chromatic pupil campimetry, VFQ25/CVFQ, A3-PRO; broader efficacy measures of improved visual function (pqac-00000023, pqac-00000027) | Review reports 9 CNGA3-ACHM patients treated; well tolerated with no serious adverse events; increases in visual acuity and contrast sensitivity persisted for at least 3 years; phase IIb follow-up planned for second eye and children 6–12 years (pqac-00000024) |
| NCT02935517 | CNGA3 | Beacon Therapeutics | AGTC-402 / rAAV2tYF-PR1.7-hCNGA3 | Subretinal, one eye | Phase I/II | Adults >=18 years in groups 1–5; 6–17 years in group 3a; 4–8 years in groups 4a and 6 | 24 | Active, not recruiting | Safety: proportion with grade 3 or greater adverse events over 1 year (pqac-00000026) | Change in visual acuity, light discomfort/light aversion, and color vision vs pretreatment over 1 year (pqac-00000026) | Gong 2024 describes this as an open-label dose-escalation subretinal AAV2-variant trial using engineered cone opsin promoter; participants assigned to 4 dose groups in review summary; outcomes for CNGA3 arm described as less encouraging than CNGB3 in available review commentary (pqac-00000024, pqac-00000026) |
| NCT02599922 | CNGB3 | Beacon Therapeutics | rAAV2tYF-PR1.7-hCNGB3 | Subretinal | Phase I/II | Not stated in retrieved ClinicalTrials.gov chunks; review describes adults and children across achromatopsia programs | 32 | Active, not recruiting | Not fully detailed in retrieved ClinicalTrials.gov chunks; review characterizes trial as phase I/II open-label dose-escalation (pqac-00000024) | Not fully detailed in retrieved ClinicalTrials.gov chunks; review notes visual-function secondary outcomes (pqac-00000024) | Gong 2024 reports sequential assignment to 4 dose groups and that rAAV2tYF-PR1.7-hCNGB3 improved photosensitivity in some patients (pqac-00000024) |
| NCT03758404 | CNGA3 | MeiraGTx UK II Ltd | AAV2/8-hG1.7p.coCNGA3 (review) | Not stated in retrieved ClinicalTrials.gov chunk; review groups these with similar subretinal phase I/II trials | Phase I/II | Adults and children (review) | 11 | Completed | Incidence of treatment-related adverse events at 6 months (review) (pqac-00000024) | Improvements in visual function (review) (pqac-00000024) | Gong 2024 describes this as similar to NCT03001310, evaluating AAV2/8-hG1.7p.coCNGA3 in adults and children (pqac-00000024) |
| NCT03001310 | CNGB3 | MeiraGTx UK II Ltd | AAV2/8-hCARp.hCNGB3 | Subretinal, single administration; low/intermediate/high dose escalation | Phase I/II | >=3 years | 23 | Completed | Composite safety over 6 weeks post administration, including serious ocular/non-ocular events possibly related to ATIMP (pqac-00000025) | Week-24 change in BCVA (ETDRS), mean retinal sensitivity by static perimetry, and QoL (EQ-VAS) for children/adults (pqac-00000025) | Gong 2024 also describes a similar phase I/II open-label dose-escalation trial in adults and children; primary outcome framed as treatment-related adverse events at 6 months and secondary outcomes as visual-function improvements (pqac-00000024, pqac-00000025) |


*Table: This table summarizes the key human CNGA3- and CNGB3-targeted gene therapy trials for achromatopsia using only retrieved ClinicalTrials.gov records and the 2024 Gong review. It is useful for comparing sponsors, vectors, eligibility, endpoints, and the current state of clinical development.*