BAX 855 Pediatric Study
A Phase 3 Prospective, Uncontrolled, Multicenter Study Evaluating Pharmacokinetics, Efficacy, Safety, and Immunogenicity of BAX 855 (PEGylated Full-length Recombinant FVIII) in Previously Treated Pediatric Patients With Severe Hemophilia A
2 other identifiers
interventional
75
11 countries
39
Brief Summary
The study purpose is:
- 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.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at below P25 for phase_3
Started Oct 2014
Shorter than P25 for phase_3
39 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 25, 2014
CompletedFirst Posted
Study publicly available on registry
August 6, 2014
CompletedStudy Start
First participant enrolled
October 31, 2014
CompletedPrimary Completion
Last participant's last visit for primary outcome
October 23, 2015
CompletedStudy Completion
Last participant's last visit for all outcomes
October 23, 2015
CompletedResults Posted
Study results publicly available
December 5, 2016
CompletedMay 24, 2021
April 1, 2021
12 months
July 25, 2014
December 2, 2016
April 30, 2021
Conditions
Outcome Measures
Primary Outcomes (1)
Number of Participants With Inhibitory Antibodies to Factor VIII (FVIII)
Inhibitory antibodies to FVIII were measured using the Nijmegen modification of the Bethesda assay. Incidence of an FVIII inhibitory antibody was defined as an inhibitor level ≥0.6 Bethesda units \[BU\].
After first exposure to BAX 855 until completion of study - approx. 6 months per participant.
Secondary Outcomes (22)
Annualized Bleeding Rate (ABR)
During prophylaxis period of 6 months or ≥ 50 EDs, whichever occurs last
Consumption of BAX 855: Number of Prophylactic Infusions Per Month Per Participant
During prophylaxis period of 6 months or ≥ 50 EDs, whichever occurs last
Consumption of BAX 855: Number of Prophylactic Infusions Per Year (Annualized) Per Participant
During prophylaxis period of 6 months or ≥ 50 EDs, whichever occurs last
Consumption of BAX 855: Weight-adjusted Dose of Prophylactic Infusions Per Month Per Participant
During prophylaxis period of 6 months or ≥ 50 EDs, whichever occurs last
Consumption of BAX 855: Weight-adjusted Dose of Prophylactic Infusions Per Year (Annualized) Per Participant
During prophylaxis period of 6 months or ≥ 50 EDs, whichever occurs last
- +17 more secondary outcomes
Study Arms (2)
<6 years old
EXPERIMENTAL≥6 to <12 years
EXPERIMENTALInterventions
Pharmacokinetic (PK) analysis of ADVATE
Pharmacokinetic (PK) analysis of BAX 855
Eligibility Criteria
You may qualify if:
- Severe hemophilia A (Factor VIII (FVIII) \<1%) determined by central laboratory.
- \<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.
You may not qualify if:
- Participant has detectable FVIII inhibitory antibodies (≥0.4 Bethesda Units (BU) using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening.
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (39)
Phoenix Children's Hospital
Phoenix, Arizona, 85016, United States
University of Colorado
Denver, Colorado, 80220, United States
University of Florida College of Medicine
Gainesville, Florida, 32610, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611, United States
University of Louisville
Louisville, Kentucky, 40202, United States
Cornell University
New Hyde Park, New York, 11040, United States
New York Presbyterian Hospital-Weill
New York, New York, 10065, United States
Cincinnati Childrens Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Nationwide Childrens Hospital
Columbus, Ohio, 43205, United States
University of Oklahoma Health Sciences Center
Oklahoma City, Oklahoma, 73104, United States
University of Utah
Salt Lake City, Utah, 84108, United States
Virginia Commonwealth University
Richmond, Virginia, 23298, United States
UMHAP Sveti Georgi EAD
Plovdiv, 4000, Bulgaria
Specialized Hospital for Active Treatment of Oncohematological Diseases in Children
Sofia, 1527, Bulgaria
Multiprofile Hospital for Active Treatment "Sveta Marina"
Varna, 9010, Bulgaria
The Chinese University of Hong Kong
Shatin, New Territories, Hong Kong
Hospital Pulau Pinang
George Town, Pulau Pinang, 10450, Malaysia
Hospital Umum Sarawak
Kuching, Sarawak, 93586, Malaysia
Hospital Sibu
Sibu, Sarawak, 96000, Malaysia
Ampang Hospital
Ampang, Selangor, 68000, Malaysia
Tengku Ampian Rahimah (TAR) Hospital
Klang, Selangor, 41200, Malaysia
Academic Medical Centre
Amsterdam, 1105 NZ, Netherlands
Eulji University Hosptial
Seo-gu, Daejeon, 302-799, South Korea
Ulsan University Hosptial
Dong-gu, Ulsan, 682-714, South Korea
Severance Hospital
Seoul, 134-727, South Korea
Hospital Universitario La Paz
Madrid, 28046, Spain
Hospital Universitario La Fe
Valencia, 460026, Spain
Taichung Veterans General Hospital
Taichung, Xitun District, 40705, Taiwan
China Medical University Hospital
Taichung, 40447, Taiwan
Istanbul University Faculty of Medicine, Department of Pediatric Immunology
Cerrahpaşa, Istanbul, 34098, Turkey (Türkiye)
Ankara University Medical Faculty
Ankara, 06560, Turkey (Türkiye)
Akdeniz Univesity Medical Faculty
Antalya, 07059, Turkey (Türkiye)
Yuzuncu Yil University Medical Faculty
Van, 65000, Turkey (Türkiye)
Institute of Blood Pathology and Transfusion Medicine of Academy of Medical Sciences of Ukraine
Lviv, 79044, Ukraine
Birmingham Childrens Hospital NHS Trust
Birmingham, B4 6NH, United Kingdom
St. Thomas's Hospital
London, SE1 7EH, United Kingdom
Great Ormond Street Hospital For Children
London, WC1N 3JH, United Kingdom
Royal Manchester Children's Hospital
Manchester, M13 9WL, United Kingdom
Southampton General Hospital
Southampton, SO16 6YD, United Kingdom
Related Publications (1)
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.
PMID: 27891721RESULT
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Study Director
- Organization
- Shire
Study Officials
- STUDY DIRECTOR
Study Director
Takeda
Publication Agreements
- PI is Sponsor Employee
- No
- Restriction Type
- OTHER
- Restrictive Agreement
- Yes
Study Design
- Study Type
- interventional
- Phase
- phase 3
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
July 25, 2014
First Posted
August 6, 2014
Study Start
October 31, 2014
Primary Completion
October 23, 2015
Study Completion
October 23, 2015
Last Updated
May 24, 2021
Results First Posted
December 5, 2016
Record last verified: 2021-04
Data Sharing
- IPD Sharing
- Will share
- Shared Documents
- STUDY PROTOCOL, SAP, ICF, CSR
- Access Criteria
- IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.