ADVATE 2 mL Post-Authorization Safety Surveillance (PASS)

Study Overview
=================
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.

Official Title
-----------------
ADVATE 2 mL (Reconstituted in 2 mL SWFI) POST-AUTHORIZATION SAFETY SURVEILLANCE STUDY

Conditions
-----------------
Hemophilia A, Congenital Factor VIII (FVIII) Deficiency

Intervention / Treatment
-----------------
* Biological: Octocog alfa (recombinant human coagulation factor VIII) [ADVATE]


Participation Criteria
=================
Eligibility Criteria
-----------------
Inclusion Criteria: 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 Exclusion Criteria: 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

Ages Eligible for Study
-----------------
Maximum Age: 12 Years

Sexes Eligible for Study
-----------------
All

Accepts Healthy Volunteers
-----------------
No

Study Plan
=================
How is the study designed?
-----------------

Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| ADVATE - 2mL<br> | Biological: Octocog alfa (recombinant human coagulation factor VIII) [ADVATE]<br>* The investigators shall determine all treatment regimens according to product labeling information and standard practice.<br>* Other names: ADVATE;|

What is the study measuring?
-----------------
Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| 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) | Causally related = possibly or probably related | 6 months | 
| Number of Factor VIII (FVIII) inhibitors in all participants |  | 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 | 
| Subjective hemostatic effectiveness rating of excellent, good, fair, or none for each bleeding episode treated |  | 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 | 
| Overall effectiveness of prophylaxis in participants who are on a prophylactic regimen |  | 6 months | 
| Global assessment rating of hemostatic effectiveness of ADVATE reconstituted in 2 mL sterile water for injection (SWFI) in surgical or dental procedures | Global assessment rating = excellent, good, fair, or none | 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 | 
| 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 |