Clinical Trial: Trial of AAV5-hFIX in Severe or Moderately Severe Hemophilia B
Study Status: Active, not recruiting
Recruit Status: Active, not recruiting
Study Type: Interventional
Official Title: 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 I
Brief Summary: This study evaluates how safe gene therapy treatment with AAV5-hFIX is in adult patients with severe or moderately severe hemophilia B and severe bleeding type.
Detailed Summary:
Sponsor: UniQure Biopharma B.V.
Current Primary Outcome: Adverse Events [ Time Frame: Five years ]
Original Primary Outcome: Same as current
Current Secondary Outcome:
- FIX-replacement-therapy-free FIX activity [ Time Frame: Five years ]
- Bleeding rate [ Time Frame: Five years ]
- Total consumption of FIX replacement therapy [ Time Frame: Five years ]
- Short Form-36 Quality of Life scores [ Time Frame: Five years ]
- Vector DNA in semen, blood, saliva, nasal secretions, urine and faeces [ Time Frame: Up to five years but maximally until the date that 3 consecutive samples are negative ]
- Neutralizing antibodies to AAV5 [ Time Frame: Five years ]
- Total (IgM and IgG) antibodies to AAV5 [ Time Frame: Five years ]
- AAV5 capsid-specific T cells [ Time Frame: 26 weeks ]
- Antibodies to FIX [ Time Frame: Five years ]
- FIX inhibitors [ Time Frame: Five years ]
- Inflammatory markers: Interleukin(IL)-1β, IL-2, IL-6, Interferon γ, Monocyte Chemotactic Protein-1 [ Time Frame: 18 weeks ]
Original Secondary Outcome: Same as current
Information By: UniQure Biopharma B.V.
Dates:
Date Received: March 4, 2015
Date Started: May 2015
Date Completion: May 2021
Last Updated: February 2, 2017
Last Verified: February 2017
