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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

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Trial Profile

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

Status: Completed
Phase of Trial: Phase I/II

Latest Information Update: 08 May 2024

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At a glance

  • Drugs Aguracingene cadoparvovec (Primary) ; Entacingene turiparvovec (Primary)
  • Indications Colour vision defects
  • Focus Adverse reactions
  • Sponsors MeiraGTx

    • Most Recent Events

    • 02 May 2024 Status changed from active, no longer recruiting to completed.
    • 13 Jan 2022 Planned End Date changed from 7 Aug 2024 to 15 Jan 2026.
    • 13 Jan 2022 Planned primary completion date changed from 10 Jul 2024 to 15 Jan 2026.

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