Clinical trial NCT02093741: ADVATE 2 mL Post-Authorization Safety Surveillance (PASS)
Official title: ADVATE 2 mL (Reconstituted in 2 mL SWFI) POST-AUTHORIZATION SAFETY SURVEILLANCE STUDY
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Hemophilia A
Congenital Factor VIII (FVIII) Deficiency
Lead sponsor: Takeda
Sponsor class: INDUSTRY
First posted: 2014-03-21
Enrollment: 65
Sex: ALL
Maximum age (years): 12
Standard ages: CHILD
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: The study population will include 60 evaluable participants aged = 12 years with severe or moderately severe hemophilia A (Factor VIII (FVIII) = 2%) with documented prior exposure to FVIII concentrates.

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.


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

Primary outcomes:
measure: Incidence of all local and general, hypersensitivity and infusion-related reactions, irrespective of product-related causality for the adverse events (AEs).
timeFrame: 6 months
Secondary outcomes:
measure: 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)
timeFrame: 6 months
measure: Number of Factor VIII (FVIII) inhibitors in all participants
timeFrame: 6 months
measure: 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
timeFrame: 6 months
measure: 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
timeFrame: 6 months
measure: Subjective hemostatic effectiveness rating of excellent, good, fair, or none for each bleeding episode treated
timeFrame: 6 months
measure: Number of bleeding episodes treated with 1, 2, 3, ≥ 4 infusions of ADVATE reconstituted in 2 mL sterile water for injection (SWFI)
timeFrame: 6 months
measure: Total units of ADVATE reconstituted in 2 mL sterile water for injection (SWFI) administered to treat each bleeding episode
timeFrame: 6 months
measure: Overall effectiveness of prophylaxis in participants who are on a prophylactic regimen
timeFrame: 6 months
measure: Global assessment rating of hemostatic effectiveness of ADVATE reconstituted in 2 mL sterile water for injection (SWFI) in surgical or dental procedures
timeFrame: 6 months
measure: 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)
timeFrame: 6 months
measure: 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)
timeFrame: 6 months
Other outcomes:
Locations:
city: BORDEAUX Cedex
country: France
facility: CHRU Pellegrin, Hématologie - CRTH
city: Chambery Cedex
country: France
facility: Centre Hospitalier Générale, CTH
city: Dijon Cedex
country: France
facility: CRTH Laboratoire d'Hématologie CHRU - Hôpital du Bocage
city: REIMS Cedex
country: France
facility: American Memorial Hospital, Service de Pédiatrie
city: Rennes Cedex 09
country: France
facility: CHU de Rennes Hôpital Pontchaillou
city: Saint Priest En Jarez
country: France
facility: Hôpital Nord, Pédiatrie
city: TOULOUSE Cedex 9
country: France
facility: CHRU Purpan, CRTH - Pavillon Sénac
city: VANDOEUVRE-LES-NANCY Cedex
country: France
facility: CHU de Nancy - Hôpital de Brabois- CRTH - Laboratoire Hémato-Hémostase
city: Homburg
country: Germany
facility: Universitätskliniken des Saarlandes, Klinik für pädiatrische Onkologie und Hämatologie
state: Saarland
city: Stuttgart
country: Germany
facility: Klinikum Stuttgart, Olgahospital, Pädiatrie 5
city: Budapest
country: Hungary
facility: Heim Pál Children's Hospital, Department of Oncology
city: Miskolc
country: Hungary
facility: Borsod-Abaúj-Zemplén County Hospital, Pediatric Dep
city: Mohacs
country: Hungary
facility: Mohacsi Korhaz, Department of Pediatrics
city: Nyiregyhaza
country: Hungary
facility: Josa Andras Egyetemi Oktatokorhaz, Department of Pediatrics
city: Leeds
country: United Kingdom
facility: Leeds General Infirmary
state: West Yorkshire
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital NHS Trust
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children NHS Trust
Study officials:
affiliation: Takeda
name: Study Director
role: STUDY_DIRECTOR
References: