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A Prospective, Open-label, Phase 1b, Single-arm, Safety Study of an Intravitreal Application of a Recombinant Adeno-associated Virus Vector Expressing CNGA1 (AAV2.NN-CNGA1) in Patients With Retinitis Pigmentosa Due to CNGA1 Mutations

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Trial Profile

A Prospective, Open-label, Phase 1b, Single-arm, Safety Study of an Intravitreal Application of a Recombinant Adeno-associated Virus Vector Expressing CNGA1 (AAV2.NN-CNGA1) in Patients With Retinitis Pigmentosa Due to CNGA1 Mutations

Status: Recruiting
Phase of Trial: Phase I

Latest Information Update: 31 Mar 2025

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At a glance

  • Drugs VG 901 (Primary)
  • Indications Retinitis pigmentosa
  • Focus Adverse reactions

    • Most Recent Events

    • 28 Mar 2025 Planned End Date changed from 1 Dec 2025 to 1 Apr 2026.
    • 28 Mar 2025 Planned primary completion date changed from 1 Dec 2025 to 1 Apr 2026.
    • 08 Jan 2025 According to a ViGeneron media release, The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to VG901, and the independent Data Safety Monitoring Board (DSMB) has unanimously approved dose escalation in this ongoing Phase 1b clinical trial.

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