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

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

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).

Study Overview

• Status: Active, not recruiting
• Conditions: Hemophilia B
• Intervention / Treatment: Genetic: AAV5-hFIX

• Study Type: Observational
• Enrollment (Actual): 9

Contacts and Locations

Study Locations

• Germany
  • Berlin, Germany
    • Vivantes Klinikum im Friedrichshain
  • Frankfurt, Germany
    • Universitaetsklinikum Frankfurt - Klinikum der Johann Wolfgang Goethe Universitaet
• Netherlands
  • Amsterdam, Netherlands
    • Amsterdam UMC - Locatie AMC
  • Groningen, Netherlands
    • Groningen UMC
  • Rotterdam, Netherlands
    • Erasmus MC
  • Utrecht, Netherlands
    • Universitair Medisch Centrum Utrecht

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

• Sampling Method: Non-Probability Sample

Study Population

Male subjects with severe or moderately severe haemophilia B, who previously received an infusion of AMT-060 and completed all assessments in Study CTAMT-060-01

Description

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.

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

Group / Cohort	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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Number of Adverse events Possibly or probably related to previous AAV5-hFIX administration	6-10 years after dosing in CT-AMT-060-01 study
Neutralizing FIX antibodies (FIX inhibitors)	6-10 years after dosing in CT-AMT-060-01 study
ALT/AST levels	6-10 years after dosing in CT-AMT-060-01 study
Liver pathology score Assessed by ultrasound every 6 months	6-10 years after dosing in CT-AMT-060-01 study
Alfa fetoprotein levels	6-10 years after dosing in CT-AMT-060-01 study

Secondary Outcome Measures

Outcome Measure	Time Frame
Endogenous Percent FIX activity	6-10 years after dosing in CT-AMT-060-01 study
Total Consumption of FIX Replacement Therapy on-demand and prophylactic	6-10 years after dosing in CT-AMT-060-01 study
Number of Procedures (including major and minor surgeries)	6-10 years after dosing in CT-AMT-060-01 study
Quality of Life questionnaire SF-36 score	6-10 years after dosing in CT-AMT-060-01 study
Quality of Life questionnaire EQ-5D-5L score	6-10 years after dosing in CT-AMT-060-01 study
Hemophilia Joint Health Score	6-10 years after dosing in CT-AMT-060-01 study
Annualized bleeding rate Including all bleeds (treated and untreated), spontaneous bleeds, traumatic bleeds and joint bleeds	6-10 years after dosing in CT-AMT-060-01 study

Collaborators and Investigators

• Sponsor: CSL Behring
• Investigators: Study Director: Study Director, CSL Behring

Study record dates

Study Major Dates

• Study Start (Actual): March 18, 2021
• Primary Completion (Estimated): May 1, 2026
• Study Completion (Estimated): May 1, 2026

Study Registration Dates

• First Submitted: April 26, 2022
• First Submitted That Met QC Criteria: April 29, 2022
• First Posted (Actual): May 4, 2022

Study Record Updates

• Last Update Posted (Actual): April 3, 2024
• Last Update Submitted That Met QC Criteria: April 2, 2024
• Last Verified: March 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Blood Coagulation Disorders; Hemophilia A; Hemophilia B
• Other Study ID Numbers: CSL220_1002 (CT-AMT-060-04); 2020-000739-28 (EudraCT Number)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: YES
• IPD Plan Description: CSL will consider on a case-by-case basis requests to share Individual Patient Data (IPD) with external bona-fide, qualified scientific and medical researchers. For information on the process and requirements for submitting a voluntary data sharing request for IPD, please contact CSL at clinicaltrials@cslbehring.com.
• IPD Sharing Time Frame: Requests for IPD will generally be considered once review by major regulatory authorities (ie FDA, EMA) is complete and the primary publication is available.

IPD Sharing Access Criteria

Proposed research should seek to answer a previously unanswered important medical or scientific question.

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.

• IPD Sharing Supporting Information Type: STUDY_PROTOCOL

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No