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AMT-061 in Severe or Moderately Severe Hemophilia B
NCT03569891Phase 3
Key facts
- Status
- Completed
- Start
- Jun 2018
- Primary completion
- Sep 2021
- Countries
- 8
- Sponsor
- CSL Limited
- Modality
- Gene Editing, Gene Silencing, Gene Addition/Replacement
- Conditions
- hemophilia B (HB)
01
Overview
Phase 3, open-label trial of etranacogene dezaparvovec in hemophilia B (HB) (2 arms), sponsored by CSL Limited. Primary endpoint: Annualized Bleeding Rate for All Bleeding Episodes. Started Jun 2018; status: completed.
02
Design
2 arms- Study design
- Non-randomizedOpen-labelSingle group
FIX replacement (Lead-in Period)
Subjects record their use of FIX replacement therapy and bleeding episodes in a dedicated e-diary during the lead-in phase.
AMT-061
Subjects receive a single infusion of AAV5-hFIXco-Padua (AMT-061) at baseline.
03
Who it enrols
Adult males with severe or moderately severe congenital hemophilia B, currently on FIX prophylaxis with >150 previous exposure days to FIX protein, without history of FIX inhibitors or active HIV/HBV/HCV infections.
04
Endpoints
Primary
Annualized Bleeding Rate for All Bleeding Episodes
Secondary
Factor IX Activity Levels
Annualized Exogenous FIX Consumption
Adjusted Annualized Infusion Rate of FIX Replacement Therapy
Percent of Participants Who Discontinued FIX Prophylaxis and Remained Free of Routine FIX Prophylaxis
Percentage of Participants With Trough FIX Activity <12% of Normal
ABR for FIX-treated Bleeding Episodes
Annualized Rate of Spontaneous Bleeding Episodes
Annualized Rate of Joint Bleeding Episodes
05
Results
Primary endpoints met
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