Multicentre, Non-controlled, Prospective, Post-Marketing Safety Study Following Long-Term Prophylactic Optivate® Treatment in Subjects with Severe Haemophilia A.
- Conditions
- Severe Haemophilia ATherapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
- Registration Number
- EUCTR2012-004606-10-DE
- Lead Sponsor
- BIO PRODUCTS LABORATORY LIMITED (BPL)
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- ot Recruiting
- Sex
- Male
- Target Recruitment
- 13
Written informed consent or, if less than 18 years of age written assent (where possible) and their parent/guardian’s has given written informed consent.
Severe haemophilia A (< 1% FVIII:C). Subjects suffering from severe haemophilia A (< 2%) may be enrolled, but only after approval by BPL. Subjects with a Factor VIII of < 2% may not constitute more than 50% of the total patient population. A separate statistical evaluation will be conducted for the < 1% and < 2% populations. Basal FVIII level taken from subject’s lowest level recorded, or the level measured at screening, whichever is lower.
Previously Treated Patients (PTPs) with > 150 exposure days on prior Factor VIII therapy (of which at least the last 50 EDs or 2 years treatment can be confirmed by way of subject records).
Immunocompetent with CD4 count > 200 /µL.
HIV negative or a viral load < 200 particles /µL.
Are the trial subjects under 18? yes
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
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
History of inhibitor development to FVIII or a positive result on the Nijmegen-Bethesda at screening (quantitative result of > 0.6 Bethesda Units [BU]) prior to the administration of Optivate®.
Known or suspected hypersensitivity to the Investigational Medicinal Product (IMP) or its excipients.
Clinically significant liver disease (serum Alanine Aminotransferase [ALT] levels greater than three times the upper limit of the normal range), renal disease (serum creatinine > 200mol/L), or coagulopathy other than haemophilia A. History of unreliability or non-cooperation (including not being able to complete the study diary).
Participating in, or have taken part in another trial within the last 30 days.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To assess post-marketing immunogenicity of Optivate® by monitoring plasma inhibitor levels for at least 100 Exposure Days (EDs) for each subject.;Secondary Objective: To assess efficacy and tolerability by monitoring factor VIII (FVIII) recovery and Adverse Events (AEs).;Primary end point(s): Immunogenicity of Optivate® by monitoring plasma inhibitor level for at least 100 EDs for each subject.;Timepoint(s) of evaluation of this end point: after 60 weeks
- Secondary Outcome Measures
Name Time Method