Clinical trial

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

Name

CSL222_4001

Description

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.

Trial arms

Trial start

2023-06-15

Estimated PCD

2043-08-01

Trial end

2043-08-01

Status

Recruiting

Treatment

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.

Arms:

HEMGENIX

Other names:

Etranacogene dezaparvovec

Factor IX (FIX)

FIX prophylaxis therapy

Arms:

FIX Prophylaxis

Size

500

Primary endpoint

Bleeding Rate (all bleeds) - HEMGENIX Cohort

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.

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.

Protocol

{'studyType': 'OBSERVATIONAL', 'patientRegistry': False, 'designInfo': {'observationalModel': 'COHORT', 'timePerspective': 'PROSPECTIVE'}, 'enrollmentInfo': {'count': 500, 'type': 'ESTIMATED'}}

Updated at

2024-04-19

1 organization

2 products

1 indication

Organization

Product

Indication

Product