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Completed

NCT Number: NCT05360706

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

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).

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Key information

Conditions

Hemophilia B Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Genetic Diseases, X-Linked Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

18 year and older

Sex eligibility

Male

Study type

Observational

Primary location

Vivantes Klinikum im Friedrichshain, Berlin, Germany

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Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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.

Treatment and study plan

AAV5-hFIX

Genetic

AAV5 containing a codon-optimized human factor IX gene

Other names: CSL220

Primary outcomes

  1. 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

  2. Neutralizing FIX antibodies (FIX inhibitors)

    Time frame: 6-10 years after dosing in CT-AMT-060-01 study

  3. ALT/AST levels

    Time frame: 6-10 years after dosing in CT-AMT-060-01 study

  4. Liver pathology score Assessed by ultrasound every 6 months

    Time frame: 6-10 years after dosing in CT-AMT-060-01 study

  5. Alfa fetoprotein levels

    Time frame: 6-10 years after dosing in CT-AMT-060-01 study

Secondary outcomes

  1. Endogenous Percent FIX activity

    Time frame: 6-10 years after dosing in CT-AMT-060-01 study

  2. Total Consumption of FIX Replacement Therapy on-demand and prophylactic

    Time frame: 6-10 years after dosing in CT-AMT-060-01 study

  3. 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

  4. Number of Procedures (including major and minor surgeries)

    Time frame: 6-10 years after dosing in CT-AMT-060-01 study

  5. Quality of Life questionnaire SF-36 score

    Time frame: 6-10 years after dosing in CT-AMT-060-01 study

  6. Quality of Life questionnaire EQ-5D-5L score

    Time frame: 6-10 years after dosing in CT-AMT-060-01 study

  7. Hemophilia Joint Health Score

    Time frame: 6-10 years after dosing in CT-AMT-060-01 study

Sponsors and collaborators

Lead sponsor

CSL Behring

Industry

Registry information

Official study 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

Important dates

Study start
2021
Primary completion
2026
Study completion
2026
First posted
May 4, 2022
Registry last updated
May 26, 2026

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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