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
- Conditions
- Haemophilia BMedDRA version: 20.0Level: LLTClassification code 10018939Term: Haemophilia B (Factor IX)System Organ Class: 100000004850Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2020-000739-28-DK
- Lead Sponsor
- uniQure biopharma B.V.
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 10
Inclusion Criteria
1. Patients with congenital hemophilia B who completed Study CT-AMT-060-01
2. Able to provide informed consent following receipt of verbal and written information about the trial.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 9
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 1
Exclusion Criteria
Enrolled patients 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
- Secondary Outcome Measures
Name Time Method