A Phase I/II, Open-label, Uncontrolled, Single-dose, Dose-ascending, Multi-centre Trial Investigating an Adeno-associated Viral Vector Containing a Codon-optimized Human Factor IX Gene (AAV5-hFIX) Administered to Adult Patients With Severe or Moderately Severe Hemophilia B

Trial Profile

A Phase I/II, Open-label, Uncontrolled, Single-dose, Dose-ascending, Multi-centre Trial Investigating an Adeno-associated Viral Vector Containing a Codon-optimized Human Factor IX Gene (AAV5-hFIX) Administered to Adult Patients With Severe or Moderately Severe Hemophilia B

Active, no longer recruiting
Phase of Trial: Phase I/II

Latest Information Update: 01 Nov 2017

At a glance

  • Drugs AMT 060 (Primary)
  • Indications Haemophilia B
  • Focus Adverse reactions; First in man
  • Sponsors uniQure
  • Most Recent Events

    • 01 Nov 2017 Long-term clinical data (upto 24 months of follow-up) will be presented at the 59th American Society of Hematology (ASH) Annual Meeting 2017, according to an uniQure media release.
    • 11 Jul 2017 Results published in an uniQure media release.
    • 10 Jul 2017 Results up to 18 months of follow-up from the low-dose cohort and up to one year of follow-up from the second dose cohort have been presented at the 26th Biennial Congress of the International Society on Thrombosis and Haemostasis (ISTH), according to a uniQure media release.
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