Non-interventional Post-authorisation Study to Document the Immunogenicity, Safety, and Efficacy of NUWIQ
- 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
- 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.
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)
CHU Hôtel Dieu
🇫🇷Nantes, France
Hopital Pontchaillou
🇫🇷Rennes, France
Centro de Tratamiento de la Hemofilia Cordoba
🇦🇷Córdoba, Argentina
Fundación de Hemofilia de Salta
🇦🇷Salta, Argentina
University of Florida
🇺🇸Gainesville, Florida, United States
Belarusian Research Center for Pediatric Oncology, Hematology and Immunology
🇧🇾Borovlyany, Belarus
Nicklaus Children's Hospital
🇺🇸Miami, Florida, United States
Gulf States Hemophilia and Thrombophilia
🇺🇸Houston, Texas, United States
CTH Centro de Tratamiento de Hematologia y Hemoterapia Córdoba S.A.
🇦🇷Córdoba, Argentina
Blood Centre, University Hospital
🇨🇿Ostrava, Czechia
Centro Mayo
🇦🇷Santiago del Estero, Argentina
Hospital de Especialidades Teodoro Maldonado Carbo
🇪🇨Guayaquil, Ecuador
Fakultní nemocnice Brno
🇨🇿Brno, Czechia
CHRU Hôpital Nord
🇫🇷Saint-Priest-en-Jarez, France
Pedias Inc. Centro Hospitalario La Paz
🇬🇹Guatemala, Guatemala
CRTH, Hopital Purpan
🇫🇷Toulouse, France
Azienda Ospedaliera di Padova
🇮🇹Padova, Italy
L'Azienda Ospedaliero Universitaria Consorziale Policlinico, U.O. di Medicina Trasfusionale, Centro Emofilia e Trombosi
🇮🇹Bari, Italy
A.O. Città della Salute e della Scienza di Torino - Ospedale Regina Margherita
🇮🇹Torino, Italy
U.O.C. Ematologia, Ospedale San Giacomo Apostolo
🇮🇹Castelfranco Veneto, Italy
UOC Malattie emorragiche e della coagulazione, Azienda Ospedaliera Universitaria Careggi
🇮🇹Firenze, Italy
S.C. Ematologia U, A.O.U. Città della Salute e della Scienza di Torino
🇮🇹Torino, Italy
Fondazione IRCCS Ca Granda
🇮🇹Milan, Italy
AOU Federico II - Dipartimento di Medicina Clinica e Chirurgica
🇮🇹Naples, Italy
AOU Policlinico di Palermo
🇮🇹Palermo, Italy
Ospedale ARNAS Civico
🇮🇹Palermo, Italy
Azienda Sanitaria Locale Napoli 1 Centro
🇮🇹Naples, Italy
Dipartimento Di Medicina dell'Universita degli Studi di Perugia
🇮🇹Perugia, Italy
Oslo University Hospital
🇳🇴Oslo, Norway
Dipartimento di Biotecnologie Cellulari ed Ematologia -"Sapienza" Università di Roma
🇮🇹Rome, Italy
Centro Hospitalar Cova da Beira
🇵🇹Covilhã, Portugal
Vilnius University Hospital, Santariskiu Klinikos-Children's Hospital
🇱🇹Vilnius, Lithuania
National Haemophilia Center
🇸🇰Bratislava, Slovakia
Great Ormond Street Hospital (GOSH)
🇬🇧London, United Kingdom
The Royal London Hospital
🇬🇧London, United Kingdom
Nottingham University Hospitals NHS Trust
🇬🇧Nottingham, United Kingdom
St. Thomas' Hospital
🇬🇧London, United Kingdom
Tulane University
🇺🇸New Orleans, Louisiana, United States
Hemophilia Treatment Center of Nevada
🇺🇸Las Vegas, Nevada, United States