Clinical trial NCT06008938: An Observational Cohort Study to Characterize the Effectiveness and Safety of HEMGENIX® in Patients With Hemophilia B
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
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Hemophilia B
Keywords: Hemophilia B
HEMGENIX
Lead sponsor: CSL Behring
Sponsor class: INDUSTRY
First posted: 2023-08-24
Enrollment: 500
Sex: ALL
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: Patients with hemophilia B who have been treated with HEMGENIX and are enrolled in either the sponsor's study or the ATHN Transcends study NCT04398628 (Hemophilia Cohort, Gene Therapy Outcomes Arm), and patients who receive routine prophylaxis treatment with FIX replacement therapy that are enrolled in the ATHN Transcends study (Hemophilia Cohort, Natural History Arm), or a similar registry. All patients will provide signed informed consent required for participation.

Brief 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.


Eligibility criteria: 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.

Primary outcomes:
measure: Bleeding Rate (all bleeds) - HEMGENIX Cohort
timeFrame: 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 outcomes:
measure: Bleeding Rate (specified bleed types) - HEMGENIX Cohort
timeFrame: 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.
measure: Zero bleeds - HEMGENIX Cohort
timeFrame: During follow-up after HEMGENIX treatment, up to 180 months
measure: Correlation analysis - HEMGENIX Cohort
timeFrame: Over the 6 to: 18, 24, 36, 48, 60, 72, 96, 108, 120, 132, 144, 156, 168, and 180 months after HEMGENIX treatment.
measure: Bleeding Rate - FIX Prophylaxis Cohort
timeFrame: In the period Months 0 to: 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 Months.
measure: FIX activity - HEMGENIX Cohort
timeFrame: 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.
measure: Annualized consumption of FIX replacement therapy - HEMGENIX Cohort
timeFrame: 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).
measure: Annualized consumption of FIX replacement therapy - FIX Prophylaxis Cohort
timeFrame: At Months 0 to: 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180.
measure: Number of patients remaining free of previous continuous routine prophylaxis - HEMGENIX Cohort
timeFrame: At Months 6 to: 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180 after HEMGENIX treatment.
measure: Target joints occurrence - HEMGENIX Cohort
timeFrame: 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).
measure: Target joints resolution - HEMGENIX Cohort
timeFrame: During the follow-up period, up to 180 months
measure: Target joints - FIX Prophylaxis Cohort
timeFrame: From Months 0 to 12, 18, 24, 36, 48, 60, 72, 84, 96, 108, 120, 132, 144, 156, 168, and 180.
measure: Related Serious Adverse Events (SAEs) - number
timeFrame: During 15 years of follow-up
measure: Related SAEs - percent
timeFrame: During 15 years of follow-up
measure: Adverse Events of Special Interest (AESIs) - number
timeFrame: During 15 years of follow-up
measure: AESIs - percent
timeFrame: During 15 years of follow-up
Other outcomes:
Locations:
city: Rochester
country: United States
facility: American Thrombosis and Hemostasis Network
state: New York
Study officials:
affiliation: CSL Behring
name: Global Program Director
role: STUDY_DIRECTOR
References: