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

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ClinicalTrials.gov Identifier: NCT05962398
Recruitment Status : Enrolling by invitation
First Posted : July 27, 2023
Last Update Posted : May 1, 2024
Sponsor:
Information provided by (Responsible Party):
CSL Behring

Study Description
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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
Hemophilia B Genetic: AAV5-hFIXco-Padua

Study Design
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Study Type : Observational
Estimated 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
Actual Study Start Date : August 30, 2023
Estimated Primary Completion Date : March 2035
Estimated Study Completion Date : March 2035

Resource links provided by the National Library of Medicine


Groups and Cohorts
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Group/Cohort 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 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 Name: Etranacogene dezaparvovec


Outcome Measures
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Primary Outcome Measures :
  1. Number of Participants With Serious Adverse Events (SAEs) and AEs of Special Interest (AESIs) [ Time Frame: From Year 5 after administration of CSL222 in parent study up to Year 15 ]
  2. Percentage of Participants With SAEs and AESIs [ Time Frame: From Year 5 after administration of CSL222 in parent study up to Year 15 ]
  3. Number of SAEs and AESIs [ Time Frame: From Year 5 after administration of CSL222 in parent study up to Year 15 ]

Secondary Outcome Measures :
  1. Number of Bleeding Episodes [ Time Frame: From Year 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) [ Time Frame: From Year 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 [ Time Frame: From Year 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 [ Time Frame: From Year 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 [ Time Frame: From Year 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 [ Time Frame: From Year 5 after administration of CSL222 in parent study up to Year 15 ]
  7. Number of Participants Remaining Free of Previous Continuous Routine FIX Prophylaxis [ Time Frame: From Year 5 after administration of CSL222 in parent study up to Year 15 ]
  8. Number of Participants With New Target Joints [ Time Frame: From Year 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 [ Time Frame: From Year 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) Utility Value [ Time Frame: From Year 6 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-5D 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 responses will be converted into a single overall health state utility value. This utility value will be derived using the EQ-5D-5L Crosswalk Index Value Calculator v2.xls [Hernández Alava et al, 2023], using the United Kingdom value set.

  11. EQ-5D-5L VAS Overall Score [ Time Frame: From Year 6 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-5D VAS. The EQ-5D VAS, which measures overall health status on a vertical visual analogue scale, ranges from 0 to 100. A higher score indicates better QoL.

  12. Hem-A-QoL Overall Score [ Time Frame: From Year 6 after administration of CSL222 in parent study up to Year 15 ]
    The Hem-A-QoL (Hemophilia Quality of Life Questionnaire for Adults) consists of 46 items comprising 10 domains: physical health, feelings, treatment, work and school, dealing with hemophilia, family planning, future, partnerships and sexuality, sports and leisure, and view of yourself for which responses will be recorded from 5 options (never, seldom, sometimes, often, and always; for some items, there is also a "not applicable" option). The total score is based on the scores for each domain and ranges from 0 to 100, with lower scores reflecting a better QoL.


Eligibility Criteria
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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Gender Based Eligibility:   Yes
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Adult male participants with hemophilia B who were previously treated with CSL222 in study CSL222_2001 (NCT03489291) or CSL222_3001 (NCT03569891).
Criteria

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
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05962398


Locations
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United States, Arkansas
Arkansas Children's Hospital - Pharmacology
Little Rock, Arkansas, United States, 72202
United States, California
UC Davis Medical Center
Sacramento, California, United States, 95817
University of California, San Diego (UCSD)
San Diego, California, United States, 92121
United States, Michigan
University of Michigan Medical Center
Ann Arbor, Michigan, United States, 48109
Sponsors and Collaborators
CSL Behring
Investigators
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Study Director: Study Director CSL Behring LLC
More Information
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Responsible Party: CSL Behring
ClinicalTrials.gov Identifier: NCT05962398    
Other Study ID Numbers: CSL222_3003
First Posted: July 27, 2023    Key Record Dates
Last Update Posted: May 1, 2024
Last Verified: April 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description:

CSL will consider requests to share Individual Patient Data (IPD) from systematic review groups or bona-fide researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.

Applicable country specific privacy and other laws and regulations will be considered and may prevent sharing of IPD.

If the request is approved and the researcher has executed an appropriate data sharing agreement, IPD that has been appropriately anonymized will be available.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Time Frame: IPD requests may be submitted to CSL no earlier than 12 months after publication of the results of this study via an article made available on a public website.
Access Criteria:

Requests may only be made by systematic review groups or bona-fide researchers whose proposed use of the IPD is non-commercial in nature and has been approved by an internal review committee.

An IPD request will not be considered by CSL unless the proposed research question seeks to answer a significant and unknown medical science or patient care question as determined by CSL's internal review committee.

The requesting party must execute an appropriate data sharing agreement before IPD will be made available.

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
 Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked