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
Latest Information Update: 11 Mar 2023
At a glance
- Drugs Entacingene turiparvovec (Primary)
- Indications Colour vision defects
- Focus Adverse reactions; First in man
- Sponsors MeiraGTx
- 05 Dec 2019 Status changed from active, no longer recruiting to completed.
- 12 Sep 2019 Planned End Date changed from 1 Feb 2019 to 8 Dec 2019.
- 14 May 2019 According to a MeiraGTx media release, dosing in the study has been completed, a total of 23 patients were treated: 11 adults in dose escalation cohorts and 12 children in a pediatric expansion cohort.