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

Active, not recruiting

• Conditions: Hemophilia B
• Interventions: Genetic: AAV5-hFIX

• Registration Number: NCT05360706
• Lead Sponsor: CSL Behring

Brief 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).

Detailed Description

Not available

Study Timeline

• Study Start: 2021-03-18
• Study First Submitted: 2022-04-26
• Study First Submitted QC: 2022-04-29
• Study First Posted: 2022-05-04
• Status Verified: 2025-01
• Last Update Submitted: 2025-01-06
• Last Update Posted: 2025-01-07
• Primary Completion: 2026-05
• Study Completion: 2026-05

Recruitment & Eligibility

• Status: ACTIVE_NOT_RECRUITING
• Sex: Male
• Target Recruitment: 9

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.

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Exclusion Criteria

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

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Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Arm && Interventions

Group	Intervention	Description
CSL220	AAV5-hFIX	AAV5 containing a codon-optimized human factor IX gene

Primary Outcome Measures

Name	Time	Method
Number of Adverse events Possibly or probably related to previous AAV5-hFIX administration	6-10 years after dosing in CT-AMT-060-01 study
Neutralizing FIX antibodies (FIX inhibitors)	6-10 years after dosing in CT-AMT-060-01 study
ALT/AST levels	6-10 years after dosing in CT-AMT-060-01 study
Liver pathology score Assessed by ultrasound every 6 months	6-10 years after dosing in CT-AMT-060-01 study
Alfa fetoprotein levels	6-10 years after dosing in CT-AMT-060-01 study

Secondary Outcome Measures

Name	Time	Method
Total Consumption of FIX Replacement Therapy on-demand and prophylactic	6-10 years after dosing in CT-AMT-060-01 study
Number of Procedures (including major and minor surgeries)	6-10 years after dosing in CT-AMT-060-01 study
Endogenous Percent FIX activity	6-10 years after dosing in CT-AMT-060-01 study
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
Quality of Life questionnaire EQ-5D-5L score	6-10 years after dosing in CT-AMT-060-01 study
Hemophilia Joint Health Score	6-10 years after dosing in CT-AMT-060-01 study
Quality of Life questionnaire SF-36 score	6-10 years after dosing in CT-AMT-060-01 study

Trial Locations

Locations (6)

Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet; 🇩🇪 Frankfurt, Germany

Erasmus MC; 🇳🇱 Rotterdam, Netherlands

Universitair Medisch Centrum Utrecht; 🇳🇱 Utrecht, Netherlands

Vivantes Klinikum im Friedrichshain; 🇩🇪 Berlin, Germany

Amsterdam UMC - Locatie AMC; 🇳🇱 Amsterdam, Netherlands

Groningen UMC; 🇳🇱 Groningen, Netherlands