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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

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Trial Profile

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

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 11 Mar 2023

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At a glance

  • Drugs Entacingene turiparvovec (Primary)
  • Indications Colour vision defects
  • Focus Adverse reactions; First in man
  • Sponsors MeiraGTx

    • Most Recent Events

    • 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.

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