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A Randomised, Open Label, Outcomes-Assessor Masked, Prospective, Parallel Controlled Group, Phase 3 Clinical Trial of Retinal Gene Therapy for Choroideremia Using an Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1)

Trial Profile

A Randomised, Open Label, Outcomes-Assessor Masked, Prospective, Parallel Controlled Group, Phase 3 Clinical Trial of Retinal Gene Therapy for Choroideremia Using an Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1)

Status: Completed
Phase of Trial: Phase III

Latest Information Update: 15 Dec 2023

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At a glance

  • Drugs Timrepigine emparvovec (Primary)
  • Indications Choroideraemia
  • Focus Registrational; Therapeutic Use
  • Acronyms STAR
  • Sponsors Biogen; Nightstar Therapeutics; NightstaRx

Most Recent Events

  • 14 Jun 2021 According to a Biogen media release, detailed results of this study will be made available at a future scientific forum.
  • 14 Jun 2021 Topline Results published in the Biogen Media Release.
  • 14 Jun 2021 Primary endpoint has not been met. (Percentage of Participants with a 15-Letter Improvement from Baseline in Best Corrected Visual Acuity (BCVA) at Month 12 as Measured by the Early Treatment of Diabetic Retinopathy Study (ETDRS) Chart), according to an Biogen media release.

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