Study of AAV5-hFIX in Severe or Moderately Severe Haemophilia B
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
2 other identifiers
observational
9
2 countries
6
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).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for all trials
Started Mar 2021
Longer than P75 for all trials
6 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
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Study Timeline
Key milestones and dates
Study Start
First participant enrolled
March 18, 2021
CompletedFirst Submitted
Initial submission to the registry
April 26, 2022
CompletedFirst Posted
Study publicly available on registry
May 4, 2022
CompletedPrimary Completion
Last participant's last visit for primary outcome
May 1, 2026
CompletedStudy Completion
Last participant's last visit for all outcomes
May 1, 2026
CompletedAugust 26, 2025
August 1, 2025
5.1 years
April 26, 2022
August 25, 2025
Conditions
Outcome Measures
Primary Outcomes (5)
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 Outcomes (7)
Endogenous Percent FIX activity
6-10 years after dosing in CT-AMT-060-01 study
Total Consumption of FIX Replacement Therapy on-demand and prophylactic
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
Number of Procedures (including major and minor surgeries)
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
- +2 more secondary outcomes
Study Arms (1)
CSL220
AAV5 containing a codon-optimized human factor IX gene
Interventions
Eligibility Criteria
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
You may qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- CSL Behringlead
Study Sites (6)
Vivantes Klinikum im Friedrichshain
Berlin, Germany
Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet
Frankfurt, Germany
Amsterdam UMC - Locatie AMC
Amsterdam, Netherlands
Groningen UMC
Groningen, Netherlands
Erasmus MC
Rotterdam, Netherlands
Universitair Medisch Centrum Utrecht
Utrecht, Netherlands
Biospecimen
After biological samples have been analyzed, they will remain stored for potential re-analysis at any time during the study to a maximum of up to 1 year after the study has been completed, before being destroyed.
MeSH Terms
Conditions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY DIRECTOR
Study Director
CSL Behring
Study Design
- Study Type
- observational
- Observational Model
- COHORT
- Time Perspective
- PROSPECTIVE
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
April 26, 2022
First Posted
May 4, 2022
Study Start
March 18, 2021
Primary Completion
May 1, 2026
Study Completion
May 1, 2026
Last Updated
August 26, 2025
Record last verified: 2025-08
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL
- Time Frame
- Requests for IPD will generally be considered once review by major regulatory authorities (ie FDA, EMA) is complete and the primary publication is available.
- Access Criteria
- Proposed research should seek to answer a previously unanswered important medical or scientific question. Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD. If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.
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.