Non-interventional Post-authorisation Study to Document the Immunogenicity, Safety, and Efficacy of NUWIQ

Completed

Conditions: Hemophilia A

Registration Number: NCT02962765

Lead Sponsor: Octapharma

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.

Detailed Description: Not available

Recruitment & Eligibility

Status: COMPLETED

Sex: Male

Target Recruitment: 80

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.

Study & Design

Study Type: OBSERVATIONAL

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Number of Patients With FVIII Inhibitors	Screening through to study completion (minimum 1.7 months; maximum 31.6 months)	FVIII inhibitors will be determined based on clinical observations and confirmed by FVIII inhibitor testing in the laboratory.
Number of Patients With Adverse Drug Reactions	Recorded from screening through to study completion (minimum 1.7 months; maximum 31.6 months)	Adverse drug reactions (ADRs) including hypersensitivity reactions will be recorded by patients in treatment diaries which will be reviewed at each Follow-up Visit.

Secondary Outcome Measures

Name	Time	Method
Annualized Rate of Breakthrough Bleeds to Assess Efficacy in Prophylactic Treatment	Monitored throughout the study from screening through to study completion (minimum 3.7 months; maximum 21.2 months)	Total number of bleeding episodes under prophylaxis treatment divided by the duration of prophylactic phase (in years)
Assessment of the Efficacy of On-demand Treatment of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale	Monitored throughout the study from screening through to study completion (minimum 1.7 months; maximum 31.6 months)	At the end of a BE, treatment efficacy was to be assessed 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 scale including the four items 'excellent,' 'good,' moderate,' and 'none.' Excellent result was defined as abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single injection. Good was definite pain relief and/or improvement in signs of bleeding within approximately 8-12 hours after an injection requiring up to 2 injections for complete resolution. Moderate was probable or slight beneficial effect within approximately 12 hours after the first injection requiring more than two injections for complete resolution. None was no improvement after 12 hours, or worsening of symptoms, requiring more than 2 injections for complete resolution.
Overall Assessment of the Effectiveness of Surgical Prophylaxis by the Treating Physicians	From start of surgery until end of post-operative period	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 hematologist. Based on this assessment, efficacy ratings assessed as either 'excellent' or 'good' will be considered 'successfully treated'.
Assessment of the Efficacy of Treatment of Bleeding Episodes (BEs) Based on a 4-point Efficacy Scale	Recorded from screening through to study completion (minimum 1.7 months; maximum 31.6 months)	At the end of a BE, treatment efficacy was to be assessed 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 scale including the four items 'excellent', 'good', 'moderate', and 'none.' Excellent result was defined as abrupt pain relief and/or unequivocal improvement in objective signs of bleeding within approximately 8 hours after a single injection. Good was definite pain relief and/or improvement in signs of bleeding within approximately 8-12 hours after an injection requiring up to 2 injections for complete resolution. Moderate was probable or slight beneficial effect within approximately 12 hours after the first injection requiring more than two injections for complete resolution. None was no improvement after 12 hours, or worsening of symptoms, requiring more than 2 injections for complete resolution.

Trial Locations

Locations (39): University of Florida
🇺🇸
Gainesville, Florida, United States
Nicklaus Children's Hospital
🇺🇸
Miami, Florida, United States
Tulane University
🇺🇸
New Orleans, Louisiana, United States
Hemophilia Treatment Center of Nevada
🇺🇸
Las Vegas, Nevada, United States
Gulf States Hemophilia and Thrombophilia
🇺🇸
Houston, Texas, United States
Centro de Tratamiento de la Hemofilia Cordoba
🇦🇷
Córdoba, Argentina
CTH Centro de Tratamiento de Hematologia y Hemoterapia Córdoba S.A.
🇦🇷
Córdoba, Argentina
Fundación de Hemofilia de Salta
🇦🇷
Salta, Argentina
Centro Mayo
🇦🇷
Santiago del Estero, Argentina
Belarusian Research Center for Pediatric Oncology, Hematology and Immunology
🇧🇾
Borovlyany, Belarus
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University of Florida
🇺🇸Gainesville, Florida, United States