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Study of AAV5-hFIX in Severe or Moderately Severe Haemophilia B

Sponsor
CSL Behring (Industry)
Overall Status
Active, not recruiting
CT.gov ID
NCT05360706
Collaborator
(none)
9
Enrollment
6
Locations
61.4
Anticipated Duration (Months)
1.5
Patients Per Site
0
Patients Per Site Per Month

Study Details

Study Description

Brief Summary

This is an open-label, extension study enrolling patients who have successfully completed all assessments in Study CT-AMT-060-01 (Years 1-5). Assessment phase will begin at Visit 36 (the first clinical visit in this extension study, approximately 5.5 years after the initial dosing visit Study CT-AMT-060-01) and go to Visit 45 (10-years post-dosing in Study CT-AMT-060-01).

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

Study Design

Study Type:
Observational
Actual Enrollment :
9 participants
Observational Model:
Cohort
Time Perspective:
Prospective
Official Title:
A Phase I/IIb Extension Study Assessing the Long-term Safety and Efficacy of an Adeno-associated Viral Vector Containing a Codon-optimized Human Factor IX Gene (AAV5-hFIX) Previously Administered to Adult Patients With Severe or Moderately Severe Haemophilia B During the CT-AMT-060-01 Phase I/II Study
Actual Study Start Date :
Mar 18, 2021
Anticipated Primary Completion Date :
May 1, 2026
Anticipated Study Completion Date :
May 1, 2026

Arms and Interventions

Arm Intervention/Treatment
CSL220

AAV5 containing a codon-optimized human factor IX gene

Genetic: AAV5-hFIX
AAV5 containing a codon-optimized human factor IX gene
Other Names:
  • CSL220

    		•

    Outcome Measures

    Primary Outcome Measures

    1. Number of Adverse events Possibly or probably related to previous AAV5-hFIX administration [6-10 years after dosing in CT-AMT-060-01 study]

    2. Neutralizing FIX antibodies (FIX inhibitors) [6-10 years after dosing in CT-AMT-060-01 study]

    3. ALT/AST levels [6-10 years after dosing in CT-AMT-060-01 study]

    4. Liver pathology score Assessed by ultrasound every 6 months [6-10 years after dosing in CT-AMT-060-01 study]

    5. Alfa fetoprotein levels [6-10 years after dosing in CT-AMT-060-01 study]

    Secondary Outcome Measures

    1. Endogenous Percent FIX activity [6-10 years after dosing in CT-AMT-060-01 study]

    2. Total Consumption of FIX Replacement Therapy on-demand and prophylactic [6-10 years after dosing in CT-AMT-060-01 study]

    3. Annualized bleeding rate (FIX-requiring) Including all bleeds, spontaneous bleeds, traumatic bleeds and joint bleeds [6-10 years after dosing in CT-AMT-060-01 study]

    4. Number of Procedures (including major and minor surgeries) [6-10 years after dosing in CT-AMT-060-01 study]

    5. Quality of Life questionnaire SF-36 score [6-10 years after dosing in CT-AMT-060-01 study]

    6. Quality of Life questionnaire EQ-5D-5L score [6-10 years after dosing in CT-AMT-060-01 study]

    7. Hemophilia Joint Health Score [6-10 years after dosing in CT-AMT-060-01 study]

    Eligibility Criteria

    Criteria

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

    • Subjects with congenital hemophilia B who completed Study CTAMT-060-01

    • Able to provide informed consent following receipt of verbal and written information about the trial.

    Exclusion Criteria:
    • Enrolled subjects will have already been assessed based on the exclusion criteria for Study CT-AMT-060-01.

    Contacts and Locations

    Locations

    Site City State Country Postal Code
    1 Vivantes Klinikum im Friedrichshain Berlin Germany
    2 uniQure Investigative Site Frankfurt Germany
    3 Amsterdam UMC - Locatie AMC Amsterdam Netherlands
    4 Universitair Medisch Centrum Groningen Groningen Netherlands
    5 Erasmus MC Rotterdam Netherlands
    6 UMC Utrecht, Van Creveldkliniek Utrecht Netherlands

    Sponsors and Collaborators

    • CSL Behring

    Investigators

    • Study Director: Study Director, CSL Behring

    Study Documents (Full-Text)

    None provided.

    More Information

    Publications

    None provided.
    Responsible Party:
    CSL Behring
    ClinicalTrials.gov Identifier:
    NCT05360706
    Other Study ID Numbers:
    • CSL220_1002 (CT-AMT-060-04)
    • 2020-000739-28
    First Posted:
    May 4, 2022
    Last Update Posted:
    May 4, 2022
    Last Verified:
    Apr 1, 2022
    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 May 4, 2022