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NCT02396342

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

Study 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.

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Interventions

AAV5-hFIXGENETIC
AAV5hFIX gene therapy

Study Locations

FacilityCityStateCountry
uniQure Investigative SiteCopenhagenDenmark
uniQure Investigative SiteBerlinGermany
uniQure Investigative SiteFrankfurtGermany
uniQure Investigative SiteAmsterdamNetherlands
uniQure Investigative SiteGroningenNetherlands
uniQure Investigative SiteRotterdamNetherlands
uniQure Investigative SiteUtrechtNetherlands

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: May 29, 2026