An Open Label Dose Escalation Phase 1 Clinical Trial of Retinal Gene Therapy for Choroideraemia Using an Adeno-associated Viral Vector (AAV2) Encoding Rab-escort Protein 1 (REP1)

Trial Profile

An Open Label Dose Escalation Phase 1 Clinical Trial of Retinal Gene Therapy for Choroideraemia Using an Adeno-associated Viral Vector (AAV2) Encoding Rab-escort Protein 1 (REP1)

Completed
Phase of Trial: Phase I/II

Latest Information Update: 03 Apr 2018

At a glance

  • Drugs AAV-REP1 (Primary)
  • Indications Choroideraemia
  • Focus Therapeutic Use
  • Most Recent Events

    • 03 Apr 2018 According to a Nightstar Therapeutics media release, two years of follow-up data from this trial will be presented at the The Association for Research in Vision and Ophthalmology (ARVO) meeting (2018).
    • 03 Apr 2018 Results of two years of follow-up of patients in this trial presented in the Nightstar Therapeutics media release.
    • 16 Nov 2017 Status changed from active, no longer recruiting to completed, according to ClinicalTrials.gov record.
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