Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3
Long-term Follow-up Study of Participants Following an Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/8-hCARp.hCNGB3 and AAV2/8-hG1.7p.coCNGA3) for Gene Therapy of Adults and Children With Achromatopsia Owing to Defects in CNGB3 or CNGA3
This study is a longer-term follow-up study for patients who participated in one of the clinical trials: AAV - CNGB3 retinal gene therapy for patients with achromatopsia, or AAV - CNGA3 retinal gene therapy for patients with achromatopsia.
The follow up study is designed to collect data on longer-term safety and efficacy.
Subretinal administration of a single low dose of either AAV - CNGB3 or AAV - CNGA3
Subretinal administration of a single medium dose of either AAV - CNGB3 or AAV - CNGA3
Subretinal administration of a single high dose of either AAV - CNGB3 or AAV - CNGA3
Inclusion in the study will be limited to individuals who: Are able to give informed consent or assent, with or without the guidance of their parent(s)/guardian(s) where appropriate Received AAV2/8-hCARp.hCNGB3 or AAV2/8-hG1.7p.coCNGA3 by intraocular administration in the prior open-label, Phase I/II, dose escalation study (EudraCT 2016-002290-35 or EudraCT 2018-003431-29) Are willing to adhere to the protocol and long-term follow-up Individuals will be excluded who: Are unwilling or unable to meet with the requirements of the study