Study Results
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View full resultsBasic Information
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COMPLETED
PHASE3
75 participants
INTERVENTIONAL
2014-10-31
2015-10-23
Brief Summary
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* To assess the incidence of FVIII inhibitory antibodies during 6 months of twice weekly prophylactic treatment with BAX 855 or 50 exposure days (EDs), whichever occurs last.
* To compare pharmacokinetic (PK) parameters to ADVATE.
* To assess hemostatic efficacy in prophylaxis and the treatment of bleeding episodes.
* To evaluate safety and immunogenicity.
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Detailed Description
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Conditions
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Study Design
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NON_RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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<6 years old
Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method
Pharmacokinetic (PK) analysis of ADVATE
PEGylated Recombinant Factor VIII
Pharmacokinetic (PK) analysis of BAX 855
PEGylated Recombinant Factor VIII
Prophylaxis treatment
≥6 to <12 years
Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method
Pharmacokinetic (PK) analysis of ADVATE
PEGylated Recombinant Factor VIII
Pharmacokinetic (PK) analysis of BAX 855
PEGylated Recombinant Factor VIII
Prophylaxis treatment
Interventions
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Antihemophilic Factor (Recombinant) - Plasma/Albumin Free Method
Pharmacokinetic (PK) analysis of ADVATE
PEGylated Recombinant Factor VIII
Pharmacokinetic (PK) analysis of BAX 855
PEGylated Recombinant Factor VIII
Prophylaxis treatment
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* \<12 years old at the time of screening.
* Participants aged ≥6 to \<12 years of age have been previously treated with plasma-derived and/or recombinant Factor VIII (rFVIII) concentrate(s) for a minimum of 150 exposure days (EDs) (based on the participant's medical records).
* Participants \<6 years of age have been previously treated with plasma-derived and/or rFVIII concentrate(s) for at least 50 EDs (based on the participant's medical records).
* Participant is human immunodeficiency virus (HIV) negative; or HIV positive with stable disease and CD4+ count of ≥200 cells/mm\^3, as confirmed by central laboratory.
* Participant and/or legal representative accepts prophylactic treatment over a period of 6 months.
* Participant and/or the legal representative is willing and able to comply with the requirements of the protocol.
Exclusion Criteria
* Participant has a history of FVIII inhibitory antibodies (≥0.4 BU using the Nijmegen modification of the Bethesda assay or ≥0.6 BU using the Bethesda assay) at any time prior to screening.
* Participant has known hypersensitivity towards mouse or hamster proteins, polyethylene glycol (PEG), or Tween 80.
* Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease).
* Participant's platelet count is \<100,000/μL.
* Participant has severe chronic hepatic dysfunction (eg, ≥5 times upper limit of normal (ULN) alanine aminotransferase (ALT), as confirmed by central laboratory at screening, or a documented international normalized ratio (INR) \>1.5).
* Participant has severe renal impairment (serum creatinine \>1.5 times ULN).
* Participant is scheduled to receive during the course of the study, an immunomodulating drug (eg, corticosteroid agents at a dose equivalent to hydrocortisone \>10 mg/day, or α-interferon) other than anti-retroviral chemotherapy.
* Participant has current or recent (\<30 days) use of other PEGylated drugs prior to study participation or is scheduled to use such drugs during study participation.
* 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 during the course of this study.
* Participant has a medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the Investigator, would affect participant safety or compliance.
* Participant's legal representative is a member of the team conducting this study or is in a dependent relationship with one of the study team members. Dependent relationships include close relatives (ie, children, partner/spouse, siblings, parents) as well as employees of the investigator or site personnel conducting the study.
11 Years
ALL
No
Sponsors
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Baxalta now part of Shire
INDUSTRY
Responsible Party
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Principal Investigators
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Study Director
Role: STUDY_DIRECTOR
Takeda
Locations
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Phoenix Children's Hospital
Phoenix, Arizona, United States
University of Colorado
Denver, Colorado, United States
University of Florida College of Medicine
Gainesville, Florida, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States
University of Louisville
Louisville, Kentucky, United States
Cornell University
New Hyde Park, New York, United States
New York Presbyterian Hospital-Weill
New York, New York, United States
Cincinnati Childrens Hospital Medical Center
Cincinnati, Ohio, United States
Nationwide Childrens Hospital
Columbus, Ohio, United States
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, United States
University of Utah
Salt Lake City, Utah, United States
Virginia Commonwealth University
Richmond, Virginia, United States
UMHAP Sveti Georgi EAD
Plovdiv, , Bulgaria
Specialized Hospital for Active Treatment of Oncohematological Diseases in Children
Sofia, , Bulgaria
Multiprofile Hospital for Active Treatment "Sveta Marina"
Varna, , Bulgaria
The Chinese University of Hong Kong
Shatin, New Territories, Hong Kong
Hospital Pulau Pinang
George Town, Pulau Pinang, Malaysia
Hospital Umum Sarawak
Kuching, Sarawak, Malaysia
Hospital Sibu
Sibu, Sarawak, Malaysia
Ampang Hospital
Ampang, Selangor, Malaysia
Tengku Ampian Rahimah (TAR) Hospital
Klang, Selangor, Malaysia
Academic Medical Centre
Amsterdam, , Netherlands
Eulji University Hosptial
Seo-gu, Daejeon, South Korea
Ulsan University Hosptial
Dong-gu, Ulsan, South Korea
Severance Hospital
Seoul, , South Korea
Hospital Universitario La Paz
Madrid, , Spain
Hospital Universitario La Fe
Valencia, , Spain
Taichung Veterans General Hospital
Taichung, Xitun District, Taiwan
China Medical University Hospital
Taichung, , Taiwan
Istanbul University Faculty of Medicine, Department of Pediatric Immunology
Cerrahpaşa, Istanbul, Turkey (Türkiye)
Ankara University Medical Faculty
Ankara, , Turkey (Türkiye)
Akdeniz Univesity Medical Faculty
Antalya, , Turkey (Türkiye)
Yuzuncu Yil University Medical Faculty
Van, , Turkey (Türkiye)
Institute of Blood Pathology and Transfusion Medicine of Academy of Medical Sciences of Ukraine
Lviv, , Ukraine
Birmingham Childrens Hospital NHS Trust
Birmingham, , United Kingdom
St. Thomas's Hospital
London, , United Kingdom
Great Ormond Street Hospital For Children
London, , United Kingdom
Royal Manchester Children's Hospital
Manchester, , United Kingdom
Southampton General Hospital
Southampton, , United Kingdom
Countries
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References
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Mullins ES, Stasyshyn O, Alvarez-Roman MT, Osman D, Liesner R, Engl W, Sharkhawy M, Abbuehl BE. Extended half-life pegylated, full-length recombinant factor VIII for prophylaxis in children with severe haemophilia A. Haemophilia. 2017 Mar;23(2):238-246. doi: 10.1111/hae.13119. Epub 2016 Nov 27.
Other Identifiers
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2014-000742-30
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
261202
Identifier Type: -
Identifier Source: org_study_id
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