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: 18 Jul 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

    • 06 Jun 2018 According to a Nightstar Therapeutics media release, enrolment in the expansion cohort is expected to begin in fourth quarter of 2018 and will include a low-dose control group of approximately 15 patients. Preliminary data from expansion cohort is expected in mid-2019. One year follow-up data from expansion study is expected in 2020.
    • 06 Jun 2018 According to a Nightstar Therapeutics media release, the company expects to announce preliminary dose escalation data from this trial in the third quarter of 2018 and is expected to be presented at an upcoming medical meeting. One year follow-up data from dose escalation part is expected in 2H 2019.
    • 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.
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