A Phase 1 Safety Study in Subjects With Severe Hemophilia B (Factor IX Deficiency) Using a Single-Stranded, Adeno-Associated Pseudotype 8 Viral Vector to Deliver the Gene for Human Factor IX

Trial Profile

A Phase 1 Safety Study in Subjects With Severe Hemophilia B (Factor IX Deficiency) Using a Single-Stranded, Adeno-Associated Pseudotype 8 Viral Vector to Deliver the Gene for Human Factor IX

Discontinued
Phase of Trial: Phase I

Latest Information Update: 18 May 2017

At a glance

  • Drugs AAV8-hFIX19 (Primary)
  • Indications Haemophilia B
  • Focus Adverse reactions
  • Sponsors Spark Therapeutics
  • Most Recent Events

    • 07 Jun 2017 Biomarkers information updated
    • 16 Dec 2016 Status changed from active, no longer recruiting to discontinued.
    • 03 Dec 2016 Results published in the Media Release
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