ADVATE 2 mL Post-Authorization Safety Surveillance (PASS)
- Conditions
- Hemophilia ACongenital Factor VIII (FVIII) Deficiency
- Interventions
- Biological: Octocog alfa (recombinant human coagulation factor VIII) [ADVATE]
- Registration Number
- NCT02093741
- Lead Sponsor
- Baxalta now part of Shire
- Brief Summary
This is a Post-Authorization Safety Surveillance (PASS) study designed to collect data on the safety and effectiveness of ADVATE reconstituted in 2 mL Sterile water for injection (SWFI) during routine clinical practice in children until 12 years of age. This surveillance study is a post-licensure commitment for ADVATE reconstituted in 2 mL SWFI.
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- COMPLETED
- Sex
- All
- Target Recruitment
- 65
- Severe or moderately severe hemophilia A (baseline Factor VIII (FVIII) ≤ 2%)
- ≤12 years of age
- Participant's legally authorized representative(s) has provided written informed consent
- Participant is prescribed ADVATE and will only receive ADVATE reconstituted in 2 mL sterile water for injection (SWFI)
- Documented history of prior exposure to ADVATE
- Documented evidence of negative inhibitor test result during ≤10 EDs prior to study entry
- Known hypersensitivity to the active substance or to any of the excipients
- Known allergic reaction to mouse or hamster proteins
- Participant has a requirement for a major surgical procedure at the time of enrollment
- Participant has no prior exposure to a FVIII concentrate
- Participant currently being treated with an immune tolerance induction (ITI) regimen
- Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand disease)
- Participant has participated in another clinical study involving an investigational product (IP) or investigational device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving an IP or investigational device or PASS registry during the course of this study
Study & Design
- Study Type
- OBSERVATIONAL
- Study Design
- Not specified
- Arm && Interventions
Group Intervention Description ADVATE - 2mL Octocog alfa (recombinant human coagulation factor VIII) [ADVATE] -
- Primary Outcome Measures
Name Time Method Incidence of all local and general, hypersensitivity and infusion-related reactions, irrespective of product-related causality for the adverse events (AEs). 6 months
- Secondary Outcome Measures
Name Time Method Number of Factor VIII (FVIII) inhibitors in all participants 6 months Subjective hemostatic effectiveness rating of excellent, good, fair, or none for each bleeding episode treated 6 months Change in Factor VIII (FVIII) infusion volume and time to mix and infuse FVIII treatment between ADVATE reconstituted in 5 mL and 2 mL sterile water for injection (SWFI) 6 months Number of Factor VIII (FVIII) inhibitors in Previously Treated Patients (PTPs) (> 50 Exposure Days (EDs)) with baseline Factor VIII (FVIII) < 1% and no history of FVIII inhibitors prior to study entry 6 months Number of Factor VIII (FVIII) inhibitors in Previously Treated Patients (PTPs) (> 50 Exposure Days (EDs)) with baseline Factor VIII (FVIII) ≤ 2% and no history of FVIII inhibitors prior to study entry 6 months Number of bleeding episodes treated with 1, 2, 3, ≥ 4 infusions of ADVATE reconstituted in 2 mL sterile water for injection (SWFI) 6 months Total units of ADVATE reconstituted in 2 mL sterile water for injection (SWFI) administered to treat each bleeding episode 6 months Global assessment rating of hemostatic effectiveness of ADVATE reconstituted in 2 mL sterile water for injection (SWFI) in surgical or dental procedures 6 months Global assessment rating = excellent, good, fair, or none
Overall effectiveness of prophylaxis in participants who are on a prophylactic regimen 6 months Change in Factor VIII (FVIII) treatment satisfaction and preference ratings from caregiver between ADVATE reconstituted in 5 mL and 2 mL sterile water for injection (SWFI) 6 months Number and type of adverse events (or adverse experiences) (AEs) considered by the investigator to be causally related to ADVATE reconstituted in 2 mL sterile water for injection (SWFI) 6 months Causally related = possibly or probably related
Trial Locations
- Locations (17)
Mohacsi Korhaz, Department of Pediatrics
🇭🇺Mohacs, Hungary
Heim Pál Children's Hospital, Department of Oncology
🇭🇺Budapest, Hungary
CHRU Pellegrin, Hématologie - CRTH
🇫🇷BORDEAUX Cedex, France
CHRU Purpan, CRTH - Pavillon Sénac
🇫🇷TOULOUSE Cedex 9, France
CHU de Nancy - Hôpital de Brabois- CRTH - Laboratoire Hémato-Hémostase
🇫🇷VANDOEUVRE-LES-NANCY Cedex, France
Great Ormond Street Hospital for Children NHS Trust
🇬🇧London, United Kingdom
Leeds General Infirmary
🇬🇧Leeds, West Yorkshire, United Kingdom
Centre Hospitalier Générale, CTH
🇫🇷Chambery Cedex, France
CHU de Rennes Hôpital Pontchaillou
🇫🇷Rennes Cedex 09, France
Hôpital Nord, Pédiatrie
🇫🇷Saint Priest En Jarez, France
Birmingham Children's Hospital NHS Trust
🇬🇧Birmingham, United Kingdom
Borsod-Abaúj-Zemplén County Hospital, Pediatric Dep
🇭🇺Miskolc, Hungary
Klinikum Stuttgart, Olgahospital, Pädiatrie 5
🇩🇪Stuttgart, Germany
CRTH Laboratoire d'Hématologie CHRU - Hôpital du Bocage
🇫🇷Dijon Cedex, France
American Memorial Hospital, Service de Pédiatrie
🇫🇷REIMS Cedex, France
Universitätskliniken des Saarlandes, Klinik für pädiatrische Onkologie und Hämatologie
🇩🇪Homburg, Saarland, Germany
Josa Andras Egyetemi Oktatokorhaz, Department of Pediatrics
🇭🇺Nyiregyhaza, Hungary