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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 (AAV5- hRKp.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)

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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 (AAV5- hRKp.RPGR) for Gene Therapy of Adults and Children With X-linked Retinitis Pigmentosa Owing to Defects in Retinitis Pigmentosa GTPase Regulator (RPGR)

Status: Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 26 Sep 2024

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At a glance

  • Drugs Botaretigene sparoparvovec (Primary)
  • Indications Retinitis pigmentosa
  • Focus Adverse reactions; First in man
  • Sponsors MeiraGTx

    • Most Recent Events

    • 20 Nov 2023 Planned End Date changed from 1 Nov 2026 to 18 Nov 2026.
    • 20 Nov 2023 Planned primary completion date changed from 1 Nov 2026 to 18 Nov 2026.
    • 26 Sep 2023 Planned End Date changed from 1 Jun 2023 to 1 Nov 2026.

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