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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT02093741
Other study ID # 061101
Secondary ID
Status Completed
Phase
First received
Last updated
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
Health authority
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

Study Design


Related Conditions & MeSH terms


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
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