ADVATE 2 mL Post-Authorization Safety Surveillance (PASS)

Hemophilia A Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02093741
• Other study ID #: 061101
• Status: Completed
• Start date: September 20, 2013
• Est. completion date: January 20, 2016

Study information

• Verified date: March 2021
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 65
• Est. completion date: January 20, 2016
• Est. primary completion date: January 20, 2016
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 12 Years

Eligibility

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

Related Conditions & MeSH terms

• Congenital Factor VIII (FVIII) Deficiency
• Hemophilia A

Intervention

Biological:
• Octocog alfa (recombinant human coagulation factor VIII) [ADVATE]
The investigators shall determine all treatment regimens according to product labeling information and standard practice.

Locations

Country	Name	City	State
France	CHRU Pellegrin, Hématologie - CRTH	BORDEAUX Cedex
France	Centre Hospitalier Générale, CTH	Chambery Cedex
France	CRTH Laboratoire d'Hématologie CHRU - Hôpital du Bocage	Dijon Cedex
France	American Memorial Hospital, Service de Pédiatrie	REIMS Cedex
France	CHU de Rennes Hôpital Pontchaillou	Rennes Cedex 09
France	Hôpital Nord, Pédiatrie	Saint Priest En Jarez
France	CHRU Purpan, CRTH - Pavillon Sénac	TOULOUSE Cedex 9
France	CHU de Nancy - Hôpital de Brabois- CRTH - Laboratoire Hémato-Hémostase	VANDOEUVRE-LES-NANCY Cedex
Germany	Universitätskliniken des Saarlandes, Klinik für pädiatrische Onkologie und Hämatologie	Homburg	Saarland
Germany	Klinikum Stuttgart, Olgahospital, Pädiatrie 5	Stuttgart
Hungary	Heim Pál Children's Hospital, Department of Oncology	Budapest
Hungary	Borsod-Abaúj-Zemplén County Hospital, Pediatric Dep	Miskolc
Hungary	Mohacsi Korhaz, Department of Pediatrics	Mohacs
Hungary	Josa Andras Egyetemi Oktatokorhaz, Department of Pediatrics	Nyiregyhaza
United Kingdom	Birmingham Children's Hospital NHS Trust	Birmingham
United Kingdom	Leeds General Infirmary	Leeds	West Yorkshire
United Kingdom	Great Ormond Street Hospital for Children NHS Trust	London

Sponsors (1)

Lead Sponsor	Collaborator
Baxalta now part of Shire

Countries where clinical trial is conducted

France,  Germany,  Hungary,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of all local and general, hypersensitivity and infusion-related reactions, irrespective of product-related causality for the adverse events (AEs).		6 months
Secondary	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
Secondary	Number of Factor VIII (FVIII) inhibitors in all participants		6 months
Secondary	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
Secondary	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
Secondary	Subjective hemostatic effectiveness rating of excellent, good, fair, or none for each bleeding episode treated		6 months
Secondary	Number of bleeding episodes treated with 1, 2, 3, = 4 infusions of ADVATE reconstituted in 2 mL sterile water for injection (SWFI)		6 months
Secondary	Total units of ADVATE reconstituted in 2 mL sterile water for injection (SWFI) administered to treat each bleeding episode		6 months
Secondary	Overall effectiveness of prophylaxis in participants who are on a prophylactic regimen		6 months
Secondary	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
Secondary	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
Secondary	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

External Links

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