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

CSL Behring

Status: Active, not recruiting

Conditions: Hemophilia B

Treatments: Genetic: AAV5-hFIX

Study type: Observational

Funder types: Industry

Identifiers

NCT05360706

2020-000739-28 (EudraCT Number)

CSL220_1002 (CT-AMT-060-04)

Details and patient eligibility

About

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).

Enrollment: 9 patients

Sex: Male

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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.

Trial design

9 participants in 1 patient group

CSL220

Description:

AAV5 containing a codon-optimized human factor IX gene

Treatment:

Genetic: AAV5-hFIX