STUDY PROTOCOL - Routine Practice Data Collection and Evaluation of Etranacogene dezaparvovec and Prophylactic Factor IX (FIX) Replacement

Sep 30, 2026

Uploaded by nayy☆

Study Protocol for a prospective, non-interventional study (CSL222_5002) evaluating etranacogene dezaparvovec (Hemgenix®) routine-practice effectiveness and tolerability versus prophylactic Factor IX (FIX) replacement with recombinant or plasma-derived FIX in adults with severe or moderately severe haemophilia B without a history of FIX inhibitors. Primary endpoint is annualized bleeding rate (ABR); secondary endpoints include survival, bleeding subcategories, pain (BPI-SF), joint status (HJHS), health-related quality of life (Haemo-QoL-A), and safety/tolerability assessments including AEs, AESI, and exploratory FIX utilization and infusion metrics. Enrollment starts May 2024 and ends 1 January 2026 with registry reporting through 31 December 2028.

LanguageEnglish

CategoryHealthcare

FormatPDF

Pages210

Reading Time8 h 49 min

AccessFree to download

Study Protocol

Observational Study Information

Research question and objectives

Endpoints and tolerability

Exploratory endpoints

Inclusion and Exclusion Criteria

Study design

STUDY PROTOCOL Routine Practice Data Collection and Evaluation of etranacogene dezaparvovec (Hemgenix® ) and prophylactic Factor IX (FIX) replacement in severe and moderately severe haemophilia B without a history of FIX inhibitors: a prospective, non-interventional study mandated by G-BA Study Number: CSL222_5002 Medicinal Product Etranacogene dezaparvovec (Hemgenix® ) Marketing CSL Behring GmbH (CSL) Authorization Holder: Emil-von-Behring-Strasse 76 35041 Marburg Germany Protocol Version: original v1.0 Protocol Date: 9 October 2023 Compliance: This study will be conducted in accordance with standards of pharmacovigilance practices. Good Clinical Practice ICH guideline should serve as guidance document. Local (eg, country specific) and regional (eg, European Union directives) regulations may apply and must be followed. This protocol may include information and data that contain trade secrets and privileged or confidential information that is the property of the marketing authorization holder (“CSL”) . This information must not be made public without written permission from CSL. These restrictions on disclosure will apply equally to all future information supplied to you. This material may be disclosed to and used by your staff and associates as may be necessary to conduct the study. Observational Study Information | Title | Routine Practice Data Collection and Evaluation of etranacogene dezaparvovec (Hemgenix® ) and prophylactic Factor IX (FIX) replacement in severe and moderately severe haemophilia B without a history of FIX inhibitors: a prospective, non-interventional study mandated by G-BA | | --- | --- | | Protocol version identifier | original v1.0 | | Date of last version of protocol | 9 October 2023 | | Marketing Authorization
Holder | CSL Behring GmbH (CSL) Emil-von-Behring-Strasse 76
35041 Marburg Germany
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xxxxxxxxxxxxxxxxxxxxxxxxxxxxxxx | | Medicinal Product | Etranacogene dezaparvovec (Hemgenix® ): gene therapy medicinal product, single-dose infusion of 2 x 1013 gene copies/ kg body weight
Prophylactic FIX replacement: plasma-derived and recombinant coagulation FIX products, prophylactic dosing and route of administration according to corresponding Summary of Product Characteristics (SmPC) | | Research question and objectives | This non-interventional study aims to evaluate the overall effectiveness and tolerability of gene therapy etranacogene dezaparvovec (Hemgenix® ) compared to a prophylaxis with recombinant or plasma-derived FIX products in patients with severe or moderately severe haemophilia B. The study represents a non-randomized Routine Practice Data Collection using data documented by haemophilia sites that are routinely captured for reporting to the German Haemophilia Registry (DHR) .
Effectiveness and tolerability will be assessed based on patient-relevant endpoints resulting from G-BA resolution mandating this study:
Primary endpoint:
Annualized bleeding rate (ABR): All treated bleeding
Secondary endpoints:
• Survival: Overall survival
• Morbidity:
• Bleeding: ABR for
▪ Severe bleeding
▪ Life-threatening bleeding
▪ Joint bleeding
• Pain: Brief Pain Inventory – Short Form (BPI-SF)
• Joint status: Hemophilia Joint Health Score (HJHS)
• Health-related quality of life:
Haemophilia-specific Health-related Quality of Life Questionnaire for Adults (Haemo-QoL-A)
• Tolerability: | | --- | --- | | | • Adverse events (AE)
• Serious AE (AE leading to death or hospitalization)
• Adverse events of special interest (AESI) and serious AESI
▪ Thromboembolic events
▪ Development of FIX inhibitors
▪ Symptomatic liver damage
▪ Malignant neoplasms Exploratory endpoints:
• FIX utilization
• Annualized infusion rate of prophylactic FIX concentrates (number of infusions)
• Annualized infusion rate of on-demand FIX concentrates (number of infusions)
• Re

FAQs

What is the purpose of this study protocol?

To evaluate the overall effectiveness and tolerability of gene therapy etranacogene dezaparvovec (Hemgenix®) compared with prophylactic FIX replacement in adults with severe or moderately severe haemophilia B without a history of FIX inhibitors.

What are the primary and key secondary endpoints?

The primary endpoint is annualized bleeding rate (ABR). Secondary endpoints include overall survival, bleeding subcategories (including severe and life-threatening and joint bleeding), pain (BPI-SF), joint status (HJHS), and health-related quality of life (Haemo-QoL-A), along with tolerability assessments based on adverse events.

Who is eligible and when will data be collected?

Eligible participants are adults with severe or moderately severe haemophilia B (≤ 5% endogenous FIX activity) who received recombinant or plasma-derived FIX concentrates and can provide signed informed consent; key exclusions include prior FIX inhibitors, advanced hepatic fibrosis/cirrhosis, active infection, limited life expectancy, or known intolerance to FIX concentrates or etranacogene dezaparvovec. Enrollment is expected to start May 2024, end 1 January 2026, and data collection continues until 31 December 2028.

Sep 30, 2026

Uploaded by nayy☆