Hemophilia BNCT05360706
Study of AAV5-hFIX in Severe or Moderately Severe Haemophilia B
An observational study of Hemophilia B, run by CSL Behring at 6 sites in 2 countries.
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… Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Hemophilia B
- What is being tested?
- AAV5-hFIX
- How many people
- 9 participants
- Where
- 6 sitesin 2 countries
- Study dates
- 18 Mar 2021to 26 Mar 2026
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Study population: Male subjects with severe or moderately severe haemophilia B, who previously received an infusion of AMT-060 and completed all assessments in Study CTAMT-060-01
Who may be able to join
- - 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.
Who cannot join
- - Enrolled subjects will have already been assessed based on the exclusion
- criteria for Study CT-AMT-060-01.
Trial locations and contacts
6 sites in 2 countries.
6 sites in 2 countries
Netherlands4 sites
Amsterdam UMC - Locatie AMC
Amsterdam
Groningen UMC
Groningen
Erasmus MC
Rotterdam
Universitair Medisch Centrum Utrecht
Utrecht
Germany2 sites
Vivantes Klinikum im Friedrichshain
Berlin
Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitae
Frankfurt
About this study
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
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).
- Conditions
- Hemophilia B
- Being tested
- AAV5-hFIX
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
AAV5-hFIXGenetic
Also called CSL220
AAV5 containing a codon-optimized human factor IX gene
| Study group | Role | What they receive |
|---|---|---|
| CSL220 | AAV5 containing a codon-optimized human factor IX gene Receives: Genetic: AAV5-hFIX |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Adverse events Possibly or probably related to previous AAV5-hFIX administration
Measured: 6-10 years after dosing in CT-AMT-060-01 study
Neutralizing FIX antibodies (FIX inhibitors)
Measured: 6-10 years after dosing in CT-AMT-060-01 study
ALT/AST levels
Measured: 6-10 years after dosing in CT-AMT-060-01 study
Liver pathology score Assessed by ultrasound every 6 months
Measured: 6-10 years after dosing in CT-AMT-060-01 study
Alfa fetoprotein levels
Measured: 6-10 years after dosing in CT-AMT-060-01 study
Secondary outcomes (7)
Endogenous Percent FIX activity
Measured: 6-10 years after dosing in CT-AMT-060-01 study
Total Consumption of FIX Replacement Therapy on-demand and prophylactic
Measured: 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
Measured: 6-10 years after dosing in CT-AMT-060-01 study
Number of Procedures (including major and minor surgeries)
Measured: 6-10 years after dosing in CT-AMT-060-01 study
Quality of Life questionnaire SF-36 score
Measured: 6-10 years after dosing in CT-AMT-060-01 study
Quality of Life questionnaire EQ-5D-5L score
Measured: 6-10 years after dosing in CT-AMT-060-01 study
Hemophilia Joint Health Score
Measured: 6-10 years after dosing in CT-AMT-060-01 study
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 May 2022
- Study start18 Mar 2021
- Primary completion26 Mar 2026
- Study completion26 Mar 2026
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia BStudies of the same condition, open and under way.
- More trials run by CSL BehringThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 9 participants (actual)
Sponsor and investigators
- Lead sponsor
- CSL Behring
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, CSL Behring
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05360706
- Sponsor's protocol ID
- CSL220_1002 (CT-AMT-060-04)
- Other IDs
- 2020-000739-28
- Status last verified
- May 2026
- First submitted
- 26 Apr 2022
- Last updated
- 26 May 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Yes: CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.
Study at a glance
From the registry record, last updated by the sponsor on 26 May 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05360706
- Acronym / sponsor ID
- CSL220_1002 (CT-AMT-060-04)
- Conditions
- Hemophilia B
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 9 participants (actual)
- Lead sponsor
- CSL BehringIndustry (pharma, biotech, devices)
- Study start
- 18 Mar 2021 (actual)
- Primary completion
- 26 Mar 2026 (actual)
- Study completion
- 26 Mar 2026 (actual)
- First posted
- 4 May 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CSL220 | – | Genetic: AAV5-hFIXAAV5 containing a codon-optimized human factor IX gene |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Male subjects with severe or moderately severe haemophilia B, who previously received an infusion of AMT-060 and completed all assessments in Study CTAMT-060-01
Key inclusion criteria (3 listed)
- - 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.
Key exclusion criteria (2 listed)
- - Enrolled subjects will have already been assessed based on the exclusion
- criteria for Study CT-AMT-060-01.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| AAV5-hFIXAlso: CSL220AAV5 containing a codon-optimized human factor IX gene | Genetic | CSL220 |
Primary outcome
Number of Adverse events Possibly or probably related to previous AAV5-hFIX administration
Time frame: 6-10 years after dosing in CT-AMT-060-01 study
Neutralizing FIX antibodies (FIX inhibitors)
Time frame: 6-10 years after dosing in CT-AMT-060-01 study
ALT/AST levels
Time frame: 6-10 years after dosing in CT-AMT-060-01 study
Liver pathology score Assessed by ultrasound every 6 months
Time frame: 6-10 years after dosing in CT-AMT-060-01 study
Alfa fetoprotein levels
Time frame: 6-10 years after dosing in CT-AMT-060-01 study
7 secondary outcomes
Endogenous Percent FIX activity
Time frame: 6-10 years after dosing in CT-AMT-060-01 study
Total Consumption of FIX Replacement Therapy on-demand and prophylactic
Time frame: 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
Time frame: 6-10 years after dosing in CT-AMT-060-01 study
Number of Procedures (including major and minor surgeries)
Time frame: 6-10 years after dosing in CT-AMT-060-01 study
Quality of Life questionnaire SF-36 score
Time frame: 6-10 years after dosing in CT-AMT-060-01 study
Quality of Life questionnaire EQ-5D-5L score
Time frame: 6-10 years after dosing in CT-AMT-060-01 study
Hemophilia Joint Health Score
Time frame: 6-10 years after dosing in CT-AMT-060-01 study
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 May 2026.
- Individual participant dataSharing planned: Yes. CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
CSL Behring
6 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia B, run by CSL Behring at 6 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 26 May 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 6 sites in Netherlands, Germany. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Adverse events Possibly or probably related to previous AAV5-hFIX administration (time frame: 6-10 years after dosing in CT-AMT-060-01 study). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 May 2026.