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Phase I, Multicentre, Open Label, Single Dose, Dose-ranging Clinical Trial to Investigate the Safety and Tolerability of a Gene Therapy rAAV2/5-PBGD for the Treatment of Acute Intermittent Porphyria

Trial Profile

Phase I, Multicentre, Open Label, Single Dose, Dose-ranging Clinical Trial to Investigate the Safety and Tolerability of a Gene Therapy rAAV2/5-PBGD for the Treatment of Acute Intermittent Porphyria

Status: Completed
Phase of Trial: Phase I

Latest Information Update: 31 May 2016

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At a glance

  • Drugs AMT 021 (Primary)
  • Indications Acute intermittent porphyria
  • Focus Adverse reactions
  • Sponsors Digna Biotech
  • Most Recent Events

    • 31 Aug 2018 Biomarkers information updated
    • 31 May 2016 According to an uniQure media release, results from this trial were published in the Journal of Hepatology.
    • 06 Apr 2015 Top-line, 1-year follow-up data has been reported, according to a uniQure media release.
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