Clinical Trial
NCT03001310 Completed
Gene Therapy for Achromatopsia (CNGB3)
An Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/8-hCARp.hCNGB3) for Gene Therapy of Adults and Children With Achromatopsia Owing to Defects in CNGB3
Disease
Disease type
CNGA3-linked achromatopsia
Patient type
Adult
Children
Inclusion / Exclusion
Funding
private
Members involved
Others investigators
Pr Katarina Stingl
Representative
Germany
Low Vision Daily Life and Patients Groups (TWG5), Registries & Epidemiology (TWG7), Research (TWG8), Retinal Rare Eye Diseases (WG1)
Working Group Leader
ERN EYE member investigating site
HCP : Principal investigators
Centre for Ophthalmology, University Eye Hospital, Tübingen, Germany
Adress
Department für Augenheilkunde Tübingen Forschungsinstitut für Augenheilkunde Elfriede-Aulhorn-Str. 7
72076 Tübingen
Germany