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

Hemophilia B Clinical Trial

— IX-TEND 4001  

Official 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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06008938
• Other study ID #: CSL222_4001
• Secondary ID: EUPAS106066
• Status: Recruiting
• Start date: June 15, 2023
• Est. completion date: August 2043

Study information

• Verified date: April 2024
• Source: CSL Behring
• Contact: Trial Registration Coordinator
• Phone: 1-610-878-4000
• Email: clinicaltrials[@]cslbehring.com
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 500
• Est. completion date: August 2043
• Est. primary completion date: August 2043
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia B

Intervention

Genetic:
• HEMGENIX
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.

Biological:
• Factor IX (FIX)
FIX prophylaxis therapy

Locations

Country	Name	City	State
United States	American Thrombosis and Hemostasis Network	Rochester	New York

Sponsors (1)

Lead Sponsor	Collaborator
CSL Behring

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Bleeding Rate (all bleeds) - HEMGENIX Cohort	Annualised Bleeding Rate (ABR) for all bleeds	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.
Secondary	Bleeding Rate (specified bleed types) - HEMGENIX Cohort	ABR for spontaneous, FIX-treated, traumatic, and joint bleeds	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.
Secondary	Zero bleeds - HEMGENIX Cohort	Number of patients with zero bleeds	During follow-up after HEMGENIX treatment, up to 180 months
Secondary	Correlation analysis - HEMGENIX Cohort	Correlation analysis of ABR as a function of mean FIX activity.	Over the 6 to: 18, 24, 36, 48, 60, 72, 96, 108, 120, 132, 144, 156, 168, and 180 months after HEMGENIX treatment.
Secondary	Bleeding Rate - FIX Prophylaxis Cohort	ABR (total, spontaneous, FIX-treated, traumatic, and joint bleeds)	In the period Months 0 to: 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 Months.
Secondary	FIX activity - HEMGENIX Cohort	Mean uncontaminated endogenous FIX activity (%)	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.
Secondary	Annualized consumption of FIX replacement therapy - HEMGENIX Cohort	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).	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).
Secondary	Annualized consumption of FIX replacement therapy - FIX Prophylaxis Cohort	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).	At Months 0 to: 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180.
Secondary	Number of patients remaining free of previous continuous routine prophylaxis - HEMGENIX Cohort	Number and proportion of patients remaining free of previous continuous routine prophylaxis (FIX replacement therapy)	At Months 6 to: 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 after HEMGENIX treatment.
Secondary	Target joints occurrence - HEMGENIX Cohort	The annualized occurrence of target joints (defined as 3 or more spontaneous bleeding events into a single joint within a consecutive 6-month period).	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).
Secondary	Target joints resolution - HEMGENIX Cohort	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).	During the follow-up period, up to 180 months
Secondary	Target joints - FIX Prophylaxis Cohort	The annualized occurrence of target joints	From Months 0 to 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180.
Secondary	Related Serious Adverse Events (SAEs) - number	Incidence (number) of related SAEs	During 15 years of follow-up
Secondary	Related SAEs - percent	Incidence (percent) of related SAEs	During 15 years of follow-up
Secondary	Adverse Events of Special Interest (AESIs) - number	Incidence (number) of AESIs	During 15 years of follow-up
Secondary	AESIs - percent	Incidence (percent) of AESIs	During 15 years of follow-up