A Phase I/IIb extension study assessing the long-term safety and efficacy of a gene therapy (AAV5-hFIX) previously administered to adult patients with severe or moderately severe haemophilia B during the CT-AMT-060-01 Phase I/II study.
Phase 1
Recruiting
- Conditions
- MedDRA version: 20.0Level: LLTClassification code: 10018939Term: Haemophilia B (Factor IX) Class: 10010331Therapeutic area: Diseases [C] - Hemic and Lymphatic Diseases [C15]Haemophilia B
- Registration Number
- CTIS2024-512603-39-00
- Lead Sponsor
- CSL Behring LLC
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Recruiting
- Sex
- Male
- Target Recruitment
- 9
Inclusion Criteria
Subjects with congenital hemophilia B who completed Study CT-AMT-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 & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To assess the long-term safety (6-10 years after dosing, inclusive) of a systemic administration of AAV5-hFIX, an AAV vector containing a codon-optimized human coagulation hFIX gene, to adult subjects with severe or moderately severe haemophilia B.;Secondary Objective: To assess the long-term efficacy of a systemic administration of AAV5-hFIX, an AAV vector containing a codon-optimized human coagulation Factor IX (hFIX) gene, to adult subjects with severe or moderately severe haemophilia B.;Primary end point(s): The primary endpoint is to demonstrate the long-term safety (6-10 years) after dosing of AAV5-hFIX., Primary safety endpoints include the following: - AEs possibly or probably related to previous AAV5-hFIX administration, Neutralizing FIX antibodies (FIX inhibitors), ALT/aspartate aminotransferase (AST) levels, Liver pathology (assessed by ultrasound), Alpha-fetoprotein (AFP)
- Secondary Outcome Measures
Name Time Method Secondary end point(s):The secondary endpoints will focus on the long-term efficacy (6-10 years) after dosing of AAV5-hFIX on FIX activity, overall FIX utilization, bleeding events, any procedures, and QoL.;Secondary end point(s):Secondary efficacy endpoints include the following: - Endogenous FIX activity;Secondary end point(s):Utilization of FIX-replacement therapy;Secondary end point(s):Annualized bleeding rate; including the following: o All bleeds (treated and untreated) o Spontaneous bleeds o Traumatic bleeds o Joint bleeds;Secondary end point(s):Procedures (including major and minor surgeries);Secondary end point(s):SF-36 and EQ-5D-5L Quality of Life (QoL) scores;Secondary end point(s):Hemophilia Joint Health Score (HJHS)