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

Plain-language summary

This is an open-label extension study that continues to follow patients who successfully completed all assessments in study CT-AMT-060-01. The assessment phase begins at Visit 36, the first clinical visit roughly 5.5 years after the initial AAV5-hFIX (CSL220) dosing visit, and continues to Visit 45, which is 10 years post-dosing. Only people with congenital hemophilia B who completed CT-AMT-060-01 and could provide informed consent after verbal and written information about the trial were enrolled.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Completed

Sponsor

CSL Behring

Intervention

AAV5-hFIX (CSL220)

Start date

3/18/2021

Estimated completion

3/26/2026

Countries

Germany, Netherlands

Related genes

Locations

• Berlin, Germany
• Frankfurt, Germany
• Amsterdam, Netherlands
• Groningen, Netherlands
• Rotterdam, Netherlands
• Utrecht, Netherlands

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine 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.