Clinical trial NCT05360706: Study of AAV5-hFIX in Severe or Moderately Severe Haemophilia B
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
Study type: OBSERVATIONAL
Overall status: ACTIVE_NOT_RECRUITING
Conditions: Hemophilia B
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2022-05-04
Enrollment: 9
Sex: MALE
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
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

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).


Eligibility criteria: 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.

Primary outcomes:
measure: Number of Adverse events Possibly or probably related to previous AAV5-hFIX administration
timeFrame: 6-10 years after dosing in CT-AMT-060-01 study
measure: Neutralizing FIX antibodies (FIX inhibitors)
timeFrame: 6-10 years after dosing in CT-AMT-060-01 study
measure: ALT/AST levels
timeFrame: 6-10 years after dosing in CT-AMT-060-01 study
measure: Liver pathology score Assessed by ultrasound every 6 months
timeFrame: 6-10 years after dosing in CT-AMT-060-01 study
measure: Alfa fetoprotein levels
timeFrame: 6-10 years after dosing in CT-AMT-060-01 study
Secondary outcomes:
measure: Endogenous Percent FIX activity
timeFrame: 6-10 years after dosing in CT-AMT-060-01 study
measure: Total Consumption of FIX Replacement Therapy on-demand and prophylactic
timeFrame: 6-10 years after dosing in CT-AMT-060-01 study
measure: Annualized bleeding rate Including all bleeds (treated and untreated), spontaneous bleeds, traumatic bleeds and joint bleeds
timeFrame: 6-10 years after dosing in CT-AMT-060-01 study
measure: Number of Procedures (including major and minor surgeries)
timeFrame: 6-10 years after dosing in CT-AMT-060-01 study
measure: Quality of Life questionnaire SF-36 score
timeFrame: 6-10 years after dosing in CT-AMT-060-01 study
measure: Quality of Life questionnaire EQ-5D-5L score
timeFrame: 6-10 years after dosing in CT-AMT-060-01 study
measure: Hemophilia Joint Health Score
timeFrame: 6-10 years after dosing in CT-AMT-060-01 study
Other outcomes:
Locations:
city: Berlin
country: Germany
facility: Vivantes Klinikum im Friedrichshain
city: Frankfurt
country: Germany
facility: Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet
city: Amsterdam
country: Netherlands
facility: Amsterdam UMC - Locatie AMC
city: Groningen
country: Netherlands
facility: Groningen UMC
city: Rotterdam
country: Netherlands
facility: Erasmus MC
city: Utrecht
country: Netherlands
facility: Universitair Medisch Centrum Utrecht
Study officials:
affiliation: CSL Behring
name: Study Director
role: STUDY_DIRECTOR
References: