Study of AAV5-hFIX in Severe or Moderately Severe Haemophilia B

Hemophilia B Clinical Trial

Official title:

A Phase I/IIb Extension Study Assessing the Long-term Safety and Efficacy of an Adeno-associated Viral Vector Containing a Codon-optimized Human Factor IX Gene (AAV5-hFIX) Previously Administered to Adult Patients With Severe or Moderately Severe Haemophilia B During the CT-AMT-060-01 Phase I/II Study

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT05360706
• Other study ID #: CSL220_1002 (CT-AMT-060-04)
• Secondary ID: 2020-000739-28
• Status: Active, not recruiting
• Start date: March 18, 2021
• Est. completion date: May 2026

Study information

• Verified date: May 2024
• Source: CSL Behring
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

This is an open-label, extension study enrolling patients who have successfully completed all assessments in Study CT-AMT-060-01 (Years 1-5). Assessment phase will begin at Visit 36 (the first clinical visit in this extension study, approximately 5.5 years after the initial dosing visit Study CT-AMT-060-01) and go to Visit 45 (10-years post-dosing in Study CT-AMT-060-01).

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 9
• Est. completion date: May 2026
• Est. primary completion date: May 2026
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

• Subjects with congenital hemophilia B who completed Study CTAMT-060-01
• Able to provide informed consent following receipt of verbal and written information about the trial.

Exclusion Criteria:

• Enrolled subjects will have already been assessed based on the exclusion criteria for Study CT-AMT-060-01.

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia B

Intervention

Genetic:
• AAV5-hFIX
AAV5 containing a codon-optimized human factor IX gene

Locations

Country	Name	City	State
Germany	Vivantes Klinikum im Friedrichshain	Berlin
Germany	Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet	Frankfurt
Netherlands	Amsterdam UMC - Locatie AMC	Amsterdam
Netherlands	Groningen UMC	Groningen
Netherlands	Erasmus MC	Rotterdam
Netherlands	Universitair Medisch Centrum Utrecht	Utrecht

Sponsors (1)

Lead Sponsor	Collaborator
CSL Behring

Countries where clinical trial is conducted

Germany,  Netherlands, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Number of Adverse events Possibly or probably related to previous AAV5-hFIX administration		6-10 years after dosing in CT-AMT-060-01 study
Primary	Neutralizing FIX antibodies (FIX inhibitors)		6-10 years after dosing in CT-AMT-060-01 study
Primary	ALT/AST levels		6-10 years after dosing in CT-AMT-060-01 study
Primary	Liver pathology score Assessed by ultrasound every 6 months		6-10 years after dosing in CT-AMT-060-01 study
Primary	Alfa fetoprotein levels		6-10 years after dosing in CT-AMT-060-01 study
Secondary	Endogenous Percent FIX activity		6-10 years after dosing in CT-AMT-060-01 study
Secondary	Total Consumption of FIX Replacement Therapy on-demand and prophylactic		6-10 years after dosing in CT-AMT-060-01 study
Secondary	Annualized bleeding rate Including all bleeds (treated and untreated), spontaneous bleeds, traumatic bleeds and joint bleeds		6-10 years after dosing in CT-AMT-060-01 study
Secondary	Number of Procedures (including major and minor surgeries)		6-10 years after dosing in CT-AMT-060-01 study
Secondary	Quality of Life questionnaire SF-36 score		6-10 years after dosing in CT-AMT-060-01 study
Secondary	Quality of Life questionnaire EQ-5D-5L score		6-10 years after dosing in CT-AMT-060-01 study
Secondary	Hemophilia Joint Health Score		6-10 years after dosing in CT-AMT-060-01 study