Brief Title
Non-interventional Post-authorisation Study to Document the Immunogenicity, Safety, and Efficacy of NUWIQ
Official Title
Prospective, Multinational, Non-interventional Post-authorisation Study to Document the Long-term Immunogenicity, Safety, and Efficacy of Human-cl rhFVIII (Simoctocog Alfa) in Patients With Haemophilia A Treated in Routine Clinical Practice
Brief Summary
Prospective, multinational, non-interventional post-authorisation study to collect additional
clinical data and to ensure consistency in the long-term between the outcome from
pre-authorisation clinical studies (in 135 previously treated paediatric and adult patients)
and routine clinical practice. Besides aspects such as general product safety and efficacy,
there will be a focus on immunogenicity, particularly on inhibitor development. The diagnosis
of FVIII inhibitor will be based on clinical observations and confirmed by FVIII inhibitor
testing in the laboratory.
Study Type
Observational
Primary Outcome
Number of Patients With FVIII Inhibitors
Secondary Outcome
Annualized Rate of Breakthrough Bleeds to Assess Efficacy in Prophylactic Treatment
Condition
Hemophilia A
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Estimated Enrollment
80
Start Date
January 2015
Completion Date
August 20, 2020
Primary Completion Date
August 20, 2020
Eligibility Criteria
Inclusion Criteria:
- Haemophilia A (FVIII:C ≤ 2%) based on medical history; at least 100 patients should
have severe haemophilia A (FVIII:C < 1%)
- Male patients of any age
- Previous treatment with a FVIII concentrate for more than 150 EDs
- Availability of detailed documentation (patient diary, log book, etc.) covering either
the last 50 EDs or the last 2 years per patient to confirm treatment modality (i.e.,
prophylaxis, on-demand, recent surgery, or immune tolerance induction)
- Inhibitor negative (< 0.6 BU) at study entry as confirmed by a recovery test with
previous FVIII product and inhibitor test in a central laboratory
- Immunocompetence (CD4+ count > 200/µL), HIV-negative, or having a viral load < 200
particles/µL or < 400,000 copies/mL
- Decision to prescribe Human-cl rhFVIII before enrolment into the study
- Written informed consent by the patient or the patient's parent or legal guardian
Exclusion Criteria:
- Patients treated with any investigational medicinal product (IMP) except FVIII IMP
within 30 days prior to the Screening Visit or patients planning to undergo treatment
with any IMP other than Human-cl rhFVIII are not eligible for enrolment into the
study.
Gender
Male
Ages
N/A - N/A
Accepts Healthy Volunteers
No
Contacts
Kate Khair, PhD, ,
Location Countries
Argentina
Location Countries
Argentina
Administrative Informations
NCT ID
NCT02962765
Organization ID
GENA-99
Responsible Party
Sponsor
Study Sponsor
Octapharma
Study Sponsor
Kate Khair, PhD, Principal Investigator, Great Ormond Street Hospital
Verification Date
September 2021