Non-interventional Post-authorisation Study to Document the Immunogenicity, Safety, and Efficacy of NUWIQ
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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.
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
Study Start Date :
Estimated Primary Completion Date :
Estimated Study Completion Date :
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Incidence of FVIII inhibitors [ Time Frame: 100 exposure days ]
FVIII inhibitors will be determined based on clinical observations and confirmed by FVIII inhibitor testing in the laboratory.
Incidence of adverse drug reactions [ Time Frame: 100 exposure days ]
Adverse drug reactions (ADRs) including hypersensitivity reactions will be recorded in by patients in treatment diaries, which will be reviewed at each Follow-up Visit.
Secondary Outcome Measures :
Annualized rate of breakthrough bleeds to assess efficacy in prophylactic treatment [ Time Frame: 100 exposure days ]
The occurrence of bleeding events during the study will documented in the treatment diary.
Assessment of the efficacy of on-demand treatment [ Time Frame: 100 exposure days ]
The treatment of bleeding episodes (BE) will be assessed by either by the patient (or the patient's parent or legal guardian) or by the treating physician in case of on-site treatment using a 4 point efficacy scale: 'excellent', 'good', 'moderate', 'none'. Based on this assessment, efficacy ratings assessed as either 'excellent' or 'good' will be considered 'successfully treated'.
Overall assessment of the effectiveness of surgical prophylaxis by the treating physicians [ Time Frame: 100 exposure days ]
At the end of the postoperative period, an overall assessment of the efficacy of treatment in the pre-, peri-, and postoperative periods using the 'excellent,' 'good,' moderate,' and 'none' scale will be done jointly by the surgeon and the haematologist. Based on this assessment, efficacy ratings assessed as either 'excellent' or 'good' will be considered 'successfully treated'.
Biospecimen Retention: Samples Without DNA
Citrate Plasma for measuring FVIII inhibitors is recommended throughout the study.
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Ages Eligible for Study:
Child, Adult, Older Adult
Sexes Eligible for Study:
Accepts Healthy Volunteers:
The goal is to collect data on 200 previously treated male patients of any age with haemophilia (FVIII:C ≤ 2%). Patients from pre-authorisation studies can be followed up to at least 100 EDs. Newly enrolled patients have to be treated and followed for at least 100 EDs.
Of the 200 enrolled patients, at least 100 patients should have severe haemophilia A (FVIII:C < 1%).
Of the 200 enrolled patients, approx. 60 patients should be < 12 years of age. At least 10 patients should be aged between 14-18 years.
Patients with severe haemophilia A after successful immune tolerance induction (ITI) can also be included; the proportion of these ITI patients should not exceed 25% of the entire cohort.
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
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.