A Dose Escalation, Phase 1/2 Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR)

Trial Profile

A Dose Escalation, Phase 1/2 Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR)

Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 03 Apr 2018

At a glance

  • Drugs AAV RPGR Gene Therapy NightstaRx (Primary)
  • Indications Retinitis pigmentosa
  • Focus Adverse reactions; First in man
  • Acronyms XIRIUS
  • Sponsors Nightstar Therapeutics; NightstaRx
  • Most Recent Events

    • 03 Apr 2018 According to a Nightstar Therapeutics media release, the company expects the initial data on safety and tolerability from the dose escalation cohorts of this trial to be available towards the end of 2018; this data will determine the dose for study in an expansion cohort anticipated to include up to 30 patients.
    • 22 Mar 2017 New trial record
    • 19 Mar 2017 According to a NightstaRx media release, the company has commenced enrolling and dosing subjects in this trial.
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