BAX 855 Continuation
A Phase 3b Continuation Study of the Safety and Efficacy of PEGylated Recombinant Factor VIII (PEG-rFVIII; BAX 855) in Prophylaxis of Bleeding in Previously Treated Patients With Severe Hemophilia A
2 other identifiers
interventional
218
22 countries
86
Brief Summary
To continue the evaluation of the safety and efficacy of BAX 855 for prophylaxis and treatment of bleeding episodes in adult and pediatric previously treated patients (PTPs) aged ≤ 75 years of age with severe hemophilia A.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_3
Started Oct 2013
Typical duration for phase_3
86 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
September 16, 2013
CompletedFirst Posted
Study publicly available on registry
September 18, 2013
CompletedStudy Start
First participant enrolled
October 15, 2013
CompletedPrimary Completion
Last participant's last visit for primary outcome
March 2, 2018
CompletedStudy Completion
Last participant's last visit for all outcomes
March 2, 2018
CompletedResults Posted
Study results publicly available
May 1, 2019
CompletedMay 24, 2021
April 1, 2021
4.4 years
September 16, 2013
February 27, 2019
April 30, 2021
Conditions
Outcome Measures
Primary Outcomes (2)
Number of Participants With Inhibitory Antibodies to Factor VIII (FVIII)
Inhibitory antibodies to Factor VIII were measured by the Nijmegen modification of the Bethesda assay. Inhibitors had to be confirmed by 2 separate assessments within a 2 to 4 week period from the central laboratory.
Baseline through end of study (53 months)
Annualized Bleed Rate (ABR) - Spontaneous Bleeds
The ABR was assessed based upon each individual bleeding episode. A bleeding episode was defined as subjective (pain consistent with a joint bleed) or objective evidence of bleeding which may or may not require treatment with FVIII. The ABR of spontaneous bleeds was reported separately for twice weekly, PK-t R, each of the every 5 days and every 7 days treatment regimens at the time of bleed.
Baseline through end of study (53 months)
Secondary Outcomes (19)
Total Annualized Bleed Rate (ABR)
Baseline through end of study (53 months)
Overall Hemostatic Efficacy Rating of BAX 855 for Treatment of Breakthrough Bleeding Episodes
Baseline through end of study (53 months)
BAX 855 Infusions Needed to Treat Bleeding Episodes
Baseline through end of study (53 months)
Total Time Intervals Between Bleeding Episodes
Baseline through end of study (53 months)
Average Dose of BAX 855 Per Prophylactic Infusion
Baseline through end of study (53 months)
- +14 more secondary outcomes
Study Arms (2)
Fixed BAX855 prophylaxis
EXPERIMENTAL45-80 IU/kg twice weekly to once per week.
Pharmacokinetic (PK)-tailored BAX 855 prophylaxis
EXPERIMENTALPK-tailored prophylactic BAX855 regimen based on participant's individual PK profile to maintain a Factor VIII (FVIII) trough level
Interventions
Antihemophilic Factor (Recombinant), PEGylated
Eligibility Criteria
You may qualify if:
- Participants Transitioning from Other BAX 855 Studies:
- Participants transitioning from other BAX 855 studies can be provided with the continuation study informed consent form (ICF) prior to the end of study visit to review and consider participation in this continuation study. These participants will complete any additional screening assessments within 2 weeks of the previous study's end of study visit and will return to the study site within 6 (± 1) weeks of the previous study end of study visit to confirm eligibility for this continuation study.
- Participants transitioning from other BAX 855 studies who meet ALL of the following criteria are eligible for this study:
- Participant has completed a previous BAX 855 study and is willing to immediately transition into this continuation study.
- Participant is ≤75 years of age at screening of the previous BAX 855 study.
- Participant continues to have a Karnofsky (for participants aged ≥ 16 years) or Lansky (for participants aged \< 16 years) performance score of ≥ 60.
- Participant is human immunodeficiency virus negative (HIV-); or HIV+ with stable disease and CD4+ count ≥ 200 cells/mm\^3, as confirmed by central laboratory at screening.
- Participant is hepatitis C virus negative (HCV-) by antibody or polymerase chain reaction (PCR) testing (if positive, antibody titer will be confirmed by PCR), as confirmed by central laboratory at screening; or HCV+ with chronic stable hepatitis.
- If female of childbearing potential, participant presents with a negative urine pregnancy test and agrees to employ adequate birth control measures for the duration of the study.
- Participant and/or legally authorized representative is willing and able to comply with the requirements of the protocol.
- BAX 855 Naïve Participants:
- BAX 855 naïve participants who are ≥ 12 years of age can only be enrolled in this continuation study after enrollment in the phase 2/3 pivotal study is closed. BAX 855 naïve participants who are \< 12 years of age can only be enrolled in this continuation study after enrollment in the pediatric previously treated patient (PTP) study is closed.
- \- Enrolment of BAX 855 naïve participants will only start once the sponsor has notified the study sites accordingly.
- BAX 855 naïve participants who meet ALL of the following criteria are eligible for this study:
- Participant is ≤75 years of age at screening.
- +35 more criteria
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (89)
Phoenix Childrens Hospital
Phoenix, Arizona, 85016-7710, United States
University of Colorado
Aurora, Colorado, 80045, United States
University of Florida College of Medicine
Gainesville, Florida, 32610-0278, United States
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611-2605, United States
Bleeding and Clotting Disorders Institute
Peoria, Illinois, 61614, United States
University of Kentucky Medical Center
Lexington, Kentucky, 40536-0284, United States
University of Louisville
Louisville, Kentucky, 40202, United States
Tulane University School of Medicine
New Orleans, Louisiana, 70112, United States
Children's Mercy Hospitals & Clinics
Kansas City, Missouri, 64108, United States
North Shore-Long Island Jewish Health System
New Hyde Park, New York, 11040, United States
New York - Presbyterian/Weill Cornell Medical Center
New York, New York, 10065, United States
University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27599-7016, United States
Duke University Medical Center
Durham, North Carolina, 27705, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
Cleveland Clinic Foundation
Cleveland, Ohio, 44195, United States
Nationwide Children's Hospital
Columbus, Ohio, 43205, United States
University of Oklahoma
Oklahoma City, Oklahoma, 73104, United States
Penn State Hershey Cancer Center
Hershey, Pennsylvania, 17033, United States
Palmetto Health Richland
Columbia, South Carolina, 29203-6863, United States
University of Utah
Salt Lake City, Utah, 84108, United States
Puget Sound Blood Group
Seattle, Washington, 98104, United States
Royal Adelaide Hospital
Adelaide, South Australia, 5000, Australia
The Alfred Hospital
Melbourne, Victoria, 3004, Australia
Fremantle Hospital
Fremantle, Western Australia, 6160, Australia
Landes-Frauen-und Kinderklinik Linz
Linz, 4020, Austria
Universitatsklinik fur Innere Medizin I
Vienna, 1090, Austria
UMHAT "Sv. Georgi", EAD
Plovdiv, 4002, Bulgaria
SHAT of Oncohaematology Diseases
Sofia, 1527, Bulgaria
MHAT 'Sv. Marina', EAD
Varna, 9010, Bulgaria
Fakultni nemocnice Brno
Brno, 625 00, Czechia
Fakultni nemocnice Olomouc
Olomouc, 775 20, Czechia
Fakultni nemocnice v Motole
Prague, 150 06, Czechia
Werlhof-Institut GmbH
Hanover, Lower Saxony, 30159, Germany
Gerinnungszentrum Rhein-Ruhr
Duisburg, North Rhine-Westphalia, 47051, Germany
Vivantes Klinikum im Friedrichshain - Landsberger Allee
Berlin, 10249, Germany
Universitaetsklinikum Hamburg-Eppendorf
Hamburg, 20246, Germany
Prince of Wales Hospital
Shatin, 00000, Hong Kong
Rambam Health Care Campus
Haifa, 3109601, Israel
Chaim Sheba Medical Center
Ramat Gan, 52621, Israel
Nagoya University Hospital
Nagoya, Aichi-ken, 466-8560, Japan
University of Occupational and Environmental Health Hospital
Kitakyushu-shi, Fukuoka, 807-8556, Japan
Hiroshima University Hospital
Hiroshima, Hiroshima, 734-8551, Japan
St. Marianna University School of Medicine Hospital
Kawasaki-shi, Kanagawa, 216-8511, Japan
Nara Medical University Hospital
Kashihara-shi, Nara, 634-8522, Japan
Ogikubo Hospital
Suginami City, Tokyo, 167-8515, Japan
Tokyo Medical University Hospital
Shinjuku-ku, Tokyo-To, 160-0023, Japan
Vilnius University Hospital Santariskiu Clinics, Public Institution
Vilnius, 8661, Lithuania
Children's Hospital, Affiliate of Vilnius University Hospital Santariskiu Klinikos
Vilnius, LT-08406, Lithuania
Penang General Hospital
George Town, Pulau Pinang, 10990, Malaysia
Hospital Umum Sarawak
Kuching, Sarawak, 93586, Malaysia
Hospital Sibu
Sibu, Sarawak, 96000, Malaysia
Hospital Ampang
Ampang, Selangor, 68000, Malaysia
Pusat Darah Negara
Kuala Lumpur, 50400, Malaysia
Hospital Pulau Pinang
Pulau Pinang, 10450, Malaysia
Academisch Medisch Centrum
Amsterdam, 1105 AZ, Netherlands
Uniwersyteckie Centrum Kliniczne
Gdansk, 80-952, Poland
Wojewodzki Szpital Specjalistyczny im.M.Kopernika w Lodzi
Lodz, 93-510, Poland
Sanador SRL
Bucharest, 11038, Romania
LLC "Alba Dent"
Kirov, 610000, Russia
Regional Clinical Hospital
Krasnoyarsk, 660022, Russia
Chonnam National University Hwasun Hospital
Hwasun-gun, Jeollanam-do, 519-763, South Korea
Pusan National University Hospital
Busan, 602-739, South Korea
Eulji University Hospital
Daejeon, 302-799, South Korea
Kyung Hee University Hospital at Gangdong
Seoul, 134-727, South Korea
Ulsan University Hospital
Ulsan, 682-714, South Korea
Hospital Universitari Son Espases
Palma de Mallorca, Balearic Islands, 7010, Spain
Complejo Hospitalario Universitario A Coruña
A Coruña, La Coruña, 15006, Spain
Hospital Regional Universitario de Malaga
Málaga, Málaga, 29010, Spain
Hospital Universitario La Paz
Madrid, 28046, Spain
Hospital Universitari i Politecnic La Fe
Valencia, 46026, Spain
Sjukhusapoteket Malmo
Malmo, 21428, Sweden
Karolinska
Stockholm, 17164, Sweden
Universitaetsspital Zuerich
Zurich, 8091, Switzerland
Taichung Veterans General Hospital
Taichung, 40705, Taiwan
Tri-Service General Hospital
Taipei, 11490, Taiwan
Ankara University Medical Faculty
Ankara, 063100, Turkey (Türkiye)
Akdeniz University Faculty of Medicine
Antalya, 07059, Turkey (Türkiye)
Istanbul University Cerrahpasa Medical Faculty
Istanbul, 34098, Turkey (Türkiye)
SI V.K.Gusak Emergency and Reconstructive Surgery Institute of NAMSU
Donetsk, 83045, Ukraine
SI Institute of Blood Pathology and Transfusion Medicine of NAMSU
Lviv, 79044, Ukraine
Bristol Royal Hospital for Children
Bristol, Avon, BS2 8BJ, United Kingdom
St Thomas' Hospital
London, Greater London, SE1 7EH, United Kingdom
Royal Free Hospital
London, Greater London, WC1E 6AG, United Kingdom
Great Ormond Street Hospital for Children
London, Greater London, WC1N 3JH, United Kingdom
Royal Manchester Children's Hospital
Manchester, Greater Manchester, M13 9WL, United Kingdom
Southampton General Hospital
Southampton, Hampshire, SO16 6YD, United Kingdom
Leicester Royal Infirmary
Leicester, Leicestershire, LE1 5WW, United Kingdom
Birmingham Children's Hospital
Birmingham, West Midlands, B4 6NH, United Kingdom
The Churchill Hospital
Oxford, OX3 7LE, United Kingdom
MeSH Terms
Conditions
Interventions
Condition 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
- PREVENTION
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
September 16, 2013
First Posted
September 18, 2013
Study Start
October 15, 2013
Primary Completion
March 2, 2018
Study Completion
March 2, 2018
Last Updated
May 24, 2021
Results First Posted
May 1, 2019
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.