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A Dose Escalation (Phase 1), and Dose Expansion (Phase 2/3) Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV8) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR)

Trial Profile

A Dose Escalation (Phase 1), and Dose Expansion (Phase 2/3) Clinical Trial of Retinal Gene Therapy for X-linked Retinitis Pigmentosa Using an Adeno-Associated Viral Vector (AAV8) Encoding Retinitis Pigmentosa GTPase Regulator (RPGR)

Recruiting
Phase of Trial: Phase I/II

Latest Information Update: 29 Dec 2018

At a glance

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

    • 30 Nov 2018 According to the European Clinical Trials Database, Dose Expansion phase 3 has been added in the study and The primary efficacy endpoint is improvement from Baseline in microperimetry at 3 has also added since trial focus TU also added.
    • 13 Nov 2018 According to a Nightstar Therapeutics media release, six-month follow-up data on all 18 patients in the dose escalation study is expected to be available in the second quarter of 2019, with one-year follow-up data expected to be available in the fourth quarter of 2019.
    • 22 Sep 2018 According to a Nightstar Therapeutics media release, enrollment of the dose escalation study in the trial was completed in August 2018, consisting of six cohorts of three patients each for a total of 18 adult patients. One-year follow-up data on all 18 patients in the dose escalation study is expected to be available in the second half of 2019.
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