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A Phase II/III Expansion Study to Evaluate the Safety and Efficacy of NSR-RPGR in Patients with a Diagnosis of X-linked Retinitis Pigmentosa (XLRP) due to RPGR mutations

Trial Profile

A Phase II/III Expansion Study to Evaluate the Safety and Efficacy of NSR-RPGR in Patients with a Diagnosis of X-linked Retinitis Pigmentosa (XLRP) due to RPGR mutations

Planning
Phase of Trial: Phase II/III

Latest Information Update: 28 Nov 2018

At a glance

  • Drugs AAV RPGR Gene Therapy NightstaRx (Primary)
  • Indications Retinitis pigmentosa
  • Focus Registrational; Therapeutic Use
  • Sponsors Nightstar Therapeutics
  • Most Recent Events

    • 28 Nov 2018 New trial record
    • 13 Nov 2018 According to a Nightstar Therapeutics media release, this modified phase 2/3 expansion study is designed to be consistent with recommendations in FDAs draft guidance on the development of gene therapy products for retinal disorders.
    • 13 Nov 2018 According to a Nightstar Therapeutics media release, preliminary efficacy data is expected to be available in mid-2019, which would serve as the basis for discussions with regulatory agencies on potential phase 3 requirements.One-year follow-up data from the expansion study is expected to be available in 2020.
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