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NCT Number: NCT06008938

An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B

This observational, post-authorization, long-term follow-up study aims to investigate the short and long-term effectiveness and safety of HEMGENIX in patients with hemophilia B. The study will also include a cohort of patients with hemophilia B treated with FIX prophylaxis to enable interpretation of relevant efficacy and safety findings of HEMGENIX.

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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

All sexes

Study type

Observational

Primary location

Medical University Vienna, Vienna, Austria

Loading trial locations.

Who can participate

Healthy volunteers accepted: No

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

Inclusion criteria

  • HEMGENIX Cohort:
  • - Treatment with commercial HEMGENIX.
  • - Have provided signed written informed consent within 3 months before or within 6 months after HEMGENIX treatment, or within 6 months of when the study is initiated at the participating site.
  • FIX Prophylaxis Cohort:
  • - Adult patients (≥ 18 years) with hemophilia B who have consented and enrolled in ATHN Transcends Hemophilia Cohort (or a similar registry) and are receiving FIX prophylaxis therapy.

Exclusion criteria

  • HEMGENIX Cohort:
  • - The patient population that will be observed in this study must not have been treated with etranacogene dezaparvovec in a clinical trial.

Treatment and study plan

HEMGENIX

Genetic

HEMGENIX is a gene therapy medicinal product that aims to deliver a factor IX (FIX) gene expression cassette to the liver of patients with hemophilia B.

Other names: Etranacogene dezaparvovec

Factor IX (FIX)

Biological

FIX prophylaxis therapy

Primary outcomes

  1. Bleeding Rate (all bleeds) - HEMGENIX Cohort

    Time frame: During the 52 weeks following stable FIX expression (6 to 18 months) in the follow-up period and at Months 6 to: 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 after HEMGENIX treatment.

    Annualised Bleeding Rate (ABR) for all bleeds

Secondary outcomes

  1. Bleeding Rate (specified bleed types) - HEMGENIX Cohort

    Time frame: During follow-up period (52 weeks following stable FIX expression [6 to 18 months]), and at Months 6 to: 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 after HEMGENIX treatment.

    ABR for spontaneous, FIX-treated, traumatic, and joint bleeds

  2. Zero bleeds - HEMGENIX Cohort

    Time frame: During follow-up after HEMGENIX treatment, up to 180 months

    Number of patients with zero bleeds

  3. Correlation analysis - HEMGENIX Cohort

    Time frame: Over the 6 to: 18, 24, 36, 48, 60, 72, 96, 108, 120, 132, 144, 156, 168, and 180 months after HEMGENIX treatment.

    Correlation analysis of ABR as a function of mean FIX activity.

  4. Bleeding Rate - FIX Prophylaxis Cohort

    Time frame: In the period Months 0 to: 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 Months.

    ABR (total, spontaneous, FIX-treated, traumatic, and joint bleeds)

  5. FIX activity - HEMGENIX Cohort

    Time frame: Day 1, Week 6, and Months 6, 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 after HEMGENIX treatment.

    Mean uncontaminated endogenous FIX activity (%)

  6. Annualized consumption of FIX replacement therapy - HEMGENIX Cohort

    Time frame: During the follow-up periods (52 weeks following stable FIX expression [6 to 18 months], & at Months 0 to: 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180), & at yearly intervals (ie, at 0 to 1 yr, 1 to 2 yrs, etc, up to 14 to 15 yrs).

    Mean annualized consumption (IU/kg/Yr) of FIX replacement therapy (including FIX replacement for various clinical scenarios eg, prophylaxis, spontaneous bleeding events, medical procedures, surgery, and trauma).

  7. Annualized consumption of FIX replacement therapy - FIX Prophylaxis Cohort

    Time frame: At Months 0 to: 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180.

    Mean annualized consumption (IU/kg/Yr) of FIX replacement therapy (including FIX replacement for various clinical scenarios eg, prophylaxis, spontaneous bleeding events, medical procedures, surgery, and trauma).

  8. Number of patients remaining free of previous continuous routine prophylaxis - HEMGENIX Cohort

    Time frame: At Months 6 to: 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 after HEMGENIX treatment.

    Number and proportion of patients remaining free of previous continuous routine prophylaxis (FIX replacement therapy)

  9. Target joints occurrence - HEMGENIX Cohort

    Time frame: After HEMGENIX treatment, during the follow-up periods (52 weeks following stable FIX expression [6 to 18 months], and at Months 6 to: 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180).

    The annualized occurrence of target joints (defined as 3 or more spontaneous bleeding events into a single joint within a consecutive 6-month period).

  10. Target joints resolution - HEMGENIX Cohort

    Time frame: During the follow-up period, up to 180 months

    The percentage of resolution of target joints (target resolution is defined as a recorded target joint with 2 or fewer spontaneous bleeding events within a consecutive 12-month period).

  11. Target joints - FIX Prophylaxis Cohort

    Time frame: From Months 0 to 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180.

    The annualized occurrence of target joints

  12. Related Serious Adverse Events (SAEs) - number

    Time frame: During 15 years of follow-up

    Incidence (number) of related SAEs

  13. Related SAEs - percent

    Time frame: During 15 years of follow-up

    Incidence (percent) of related SAEs

  14. Adverse Events of Special Interest (AESIs) - number

    Time frame: During 15 years of follow-up

    Incidence (number) of AESIs

  15. AESIs - percent

    Time frame: During 15 years of follow-up

    Incidence (percent) of AESIs

Study contacts

Contact information is provided by the study sponsor or research team.

Trial Registration Coordinator

CONTACT

[email protected]

+1 610-878-4697

Sponsors and collaborators

Lead sponsor

CSL Behring

Industry

Registry information

Official study title

An Observational Post-authorization Long-term Follow-up Study to Characterize the Effectiveness and Safety of HEMGENIX® (Etranacogene Dezaparvovec) in Patients With Hemophilia B

Acronym: IX-TEND 4001

Important dates

Study start
2023
Primary completion
2043
Study completion
2043
First posted
Aug 24, 2023
Registry last updated
Feb 19, 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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