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Long-term Follow-up Study of Male Adults With Hemophilia B Previously Treated With Etranacogene Dezaparvovec (CSL222)

Sponsor
CSL Behring (Industry)
Overall Status
Not yet recruiting
CT.gov ID
NCT05962398
Collaborator
(none)
56
Enrollment
139
Anticipated Duration (Months)

Study Details

Study Description

Brief Summary

The primary purpose of this study is to assess the long-term safety in male adults with hemophilia B who were treated with CSL222 in studies CSL222_2001 (NCT03489291) or CSL222_3001 (NCT03569891).

Condition or Disease Intervention/Treatment Phase
  • Genetic: AAV5-hFIXco-Padua

Study Design

Study Type:
Observational
Anticipated Enrollment :
56 participants
Observational Model:
Cohort
Time Perspective:
Prospective
Official Title:
An Extension Study Assessing the Long-term Safety and Efficacy of Etranacogene Dezaparvovec (CSL222) Previously Administered to Adult Male Subjects With Hemophilia B
Anticipated Study Start Date :
Aug 1, 2023
Anticipated Primary Completion Date :
Mar 1, 2035
Anticipated Study Completion Date :
Mar 1, 2035

Arms and Interventions

Arm Intervention/Treatment
CSL222

Participants who received CSL222 and completed either the study CSL222_2001 (NCT03489291) or CSL222_3001 (NCT03569891) will be followed up from Year 5.5 after administration of CSL222 in parent study up to Year 15.

Genetic: AAV5-hFIXco-Padua
Single intravenous infusion of AAV5-hFIXco-Padua in the study CSL222_2001 (NCT03489291) or CSL222_3001 (NCT03569891).
Other Names:
  • Etranacogene dezaparvovec

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Number of Participants With Serious Adverse Events (SAEs) and AEs of Special Interest (AESIs) [From Year 5.5 after administration of CSL222 in parent study up to Year 15]

    2. Percentage of Participants With SAEs and AESIs [From Year 5.5 after administration of CSL222 in parent study up to Year 15]

    3. Number of SAEs and AESIs [From Year 5.5 after administration of CSL222 in parent study up to Year 15]

    Secondary Outcome Measures

    1. Number of Bleeding Episodes [From Year 5.5 after administration of CSL222 in parent study up to Year 15]

      The total, spontaneous, traumatic, and joint bleeds will be analyzed.

    2. Annualized Bleeding Rate (ABR) [From Year 5.5 after administration of CSL222 in parent study up to Year 15]

      The total, spontaneous, traumatic, and joint bleeds will be analyzed. ABR is calculated as the total bleeding episodes divided by the total time at risk.

    3. Number of Participants With Zero Bleeds [From Year 5.5 after administration of CSL222 in parent study up to Year 15]

    4. Correlation Analysis of ABR as a Function of Mean Factor IX (FIX) Activity [From Year 5.5 after administration of CSL222 in parent study up to Year 15]

      Correlation between ABR and mean FIX activity will be explored using a statistical modeling approach (like, generalized estimating equations [GEE] model for ABR with mean FIX activity as the independent variable).

    5. Number of Participants With FIX Activity [From Year 5.5 after administration of CSL222 in parent study up to Year 15]

      FIX activity will be assessed using the one-stage activated partial thromboplastin time assay and a chromogenic assay.

    6. Number of Participants Using FIX Replacement Therapy [From Year 5.5 after administration of CSL222 in parent study up to Year 15]

    7. Number of Participants Remaining Free of Previous Continuous Routine FIX Prophylaxis [From Year 5.5 after administration of CSL222 in parent study up to Year 15]

    8. Number of Participants With New Target Joints [From Year 5.5 after administration of CSL222 in parent study up to Year 15]

      Target joint is defined as 3 or more spontaneous bleeding episodes into a single joint within a consecutive 6-month period.

    9. Percentage of Resolution of Target Joints [From Year 5.5 after administration of CSL222 in parent study up to Year 15]

      Target joint is defined as 3 or more spontaneous bleeding episodes into a single joint within a consecutive 6-month period.

    10. EuroQol-5 Dimensions-5 Levels (EQ-5D-5L) VAS Overall Score [From Year 5.5 after administration of CSL222 in parent study up to Year 15]

      The EQ-5D-5L questionnaire consists of the EQ-5D-5L descriptive system and the EQ visual analogue scale (VAS). The EQ-5D-5L descriptive system of health-related QoL consists of 5 dimensions (mobility, self-care, usual activities, pain/discomfort, anxiety/depression) for which responses will be recorded on 5 levels of severity (no problems, slight problems, moderate problems, severe problems, and extreme problems). The EQ VAS overall score ranges from 0 to 100. A higher score indicates better QoL.

    Eligibility Criteria

    Criteria

    Ages Eligible for Study:
    18 Years and Older
    Sexes Eligible for Study:
    Male
    Accepts Healthy Volunteers:
    No
    Inclusion Criteria:

    • Treatment with CSL222 in Study CSL222_2001 or Study CSL222_3001.

    • Provided written informed consent.

    • Willing and able to adhere to all protocol requirements.

    Exclusion Criteria:

    Contacts and Locations

    Locations

    No locations specified.

    Sponsors and Collaborators

    • CSL Behring

    Investigators

    • Study Director: Study Director, CSL Behring LLC

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    CSL Behring
    ClinicalTrials.gov Identifier:
    NCT05962398
    Other Study ID Numbers:
    • CSL222_3003
    First Posted:
    Jul 27, 2023
    Last Update Posted:
    Aug 2, 2023
    Last Verified:
    Jul 1, 2023
    Individual Participant Data (IPD) Sharing Statement:
    Yes
    Plan to Share IPD:
    Yes
    Studies a U.S. FDA-regulated Drug Product:
    Yes
    Studies a U.S. FDA-regulated Device Product:
    No
    Additional relevant MeSH terms:
    Hemophilia A
    Hemophilia B

    Study Results

    No Results Posted as of Aug 2, 2023