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

Status

Completed

Phases

Unknown

Study type

Observational

Source

ClinicalTrials.gov

Registry ID

NCT05360706

Enrollment

Registered

2022-05-04

Start date

2021-03-18

Completion date

2026-03-26

Last updated

2026-05-26

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hemophilia B

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).

Interventions

GENETICAAV5-hFIX

AAV5 containing a codon-optimized human factor IX gene

Study design

Observational model

COHORT

Time perspective

PROSPECTIVE

Eligibility

Sex/Gender

MALE

Age

18 Years to No maximum

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.

Design outcomes

Primary

Measure	Time frame
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

Measure	Time frame
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
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

Countries

Germany, Netherlands

Contacts

STUDY_DIRECTORStudy Director

CSL Behring

Outcome results

None listed

Source: ClinicalTrials.gov · Data processed: May 27, 2026