CNGA3

Safety and efficacy of a single subretinal injection of rAAV.hCNGA3 in patients with CNGA3-linked achromatopsia investigated in an exploratory, dose-escalation trial.

Type: Interventional

Status of the trial: Active, Recruiting

Orphan Drug Recognition: Yes

Inclusion

Opening Date: 2014-09-12
Closing Date: 2027-04-01

Criteria

Inclusion: clinical diagnosis of achromatopsia, age ≥ 18 years, confirmed mutation in CNGA3, BCVA ≥ 20/400; first cohort: BCVA ≤ 20/160, area of residual outer nuclear layer thickness in OCT scans of posterior pole, ability to understand and willingness to consent t

More information

Children: No

Adults: Yes

Funder Type: other

HCP: Principal investigators