Study Investigating a PEGylated Recombinant Factor VIII (BAX 855) for Hemophilia A (PROLONG-ATE Study)
A Phase 2/3, Multi-Center, Open Label Study of Efficacy, Safety, and Pharmacokinetics of PEGylated Recombinant Factor VIII (BAX 855) Administered for Prophylaxis and Treatment of Bleeding in Previously Treated Patients With Severe Hemophilia A
2 other identifiers
interventional
159
19 countries
68
Brief Summary
To assess efficacy and safety, including immunogenicity of BAX 855 administered as prophylaxis and as on-demand therapy in adult and adolescent (12-65 years) previously treated patients (PTPs) with severe hemophilia A To determine the pharmacokinetic (PK) parameters of BAX 855.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P75+ for phase_2
Started Jan 2013
Shorter than P25 for phase_2
68 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
November 21, 2012
CompletedFirst Posted
Study publicly available on registry
November 29, 2012
CompletedStudy Start
First participant enrolled
January 31, 2013
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 17, 2014
CompletedStudy Completion
Last participant's last visit for all outcomes
July 17, 2014
CompletedResults Posted
Study results publicly available
September 7, 2016
CompletedMay 20, 2021
April 1, 2021
1.5 years
November 21, 2012
March 4, 2016
April 30, 2021
Conditions
Outcome Measures
Primary Outcomes (1)
Annualized Bleeding Rate (ABR)
Comparisons between prophylactic and on-demand treatment were based on ABR estimates from a negative binomial regression model, taking into account the treatment regimen, target joints and age at screening, and duration of the observation period for efficacy.
9 months
Secondary Outcomes (30)
Rate of Success of BAX 855 for Treatment of Bleeding Episodes
At least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm and 6 months (± 2 weeks) for the on-demand arm.
Average Number of BAX 855 Infusions Needed for the Treatment of Bleeding Episodes
From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].
Number of Participants With ≤1, 2, 3, 4, 5, 6, or >6 Month Time Intervals Between Bleeding Episodes or no Bleeding Episodes
From first exposure to BAX 855 until the end of the study, [at least 50 exposure days or 6 months (±2 weeks), whichever occurs last, for the prophylaxis arm; and 6 months (± 2 weeks) for the on-demand arm].
Weight-adjusted Consumption of BAX 855 - Per Prophylactic Infusion and Pharmacokinetic (PK) Infusion
Prophylactic Infusion: ≥50 exposure days or 6 months (±2 weeks), whichever occurs last. PK Infusion: PK #1 Pre-infusion within 30 minutes; Post-infusion 10 min, and 0.5, 1, 3, 6, 24, 32, 48, 56 hours (h). PK #2 also at Post-infusion 96h
Weight-adjusted Consumption of BAX 855 - Per Treatment of Bleeding Episode (BE) and Per BE for Maintenance of Hemostasis
Treatment of Bleeding Episode (BE): Minor/Moderate BE every 12 to 24 hours until bleeding is resolved; Major BE every 8 to 12 hours until bleeding is resolved. Per BE for Maintenance of Hemostasis: within 48 hours after bleeding episode resolution.
- +25 more secondary outcomes
Study Arms (2)
Prophylaxis
EXPERIMENTALOn-demand
EXPERIMENTALInterventions
Pharmacokinetic (PK) evaluation of ADVATE
Pharmacokinetic (PK) evaluation of BAX 855
Eligibility Criteria
You may qualify if:
- Participant and/or legal representative has/have voluntarily provided signed informed consent
- Participant is 12 to 65 years old at the time of screening
- Participant is male with severe hemophilia A (Factor VIII (FVIII) clotting activity \< 1%) as confirmed by central laboratory at screening after the appropriate washout period or a documented FVIII clotting activity \<1%
- Participant has been previously treated with plasma-derived FVIII concentrates or recombinant FVIII for ≥150 documented exposure days (EDs)
- Participant is currently receiving prophylaxis or on-demand therapy with FVIII
- Participant is willing and able to comply with the requirements of the protocol
You may not qualify if:
- Participant has detectable FVIII inhibitory antibodies (≥ 0.6 Bethesda Units (BU) using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening
- Participant has 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 been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand's disease).
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (72)
University of Colorado
Aurora, Colorado, 80045, United States
University of Florida, College of Medicine
Gainesville, Florida, 32610, United States
Children's Healthcare of Atlanta
Atlanta, Georgia, 30322, United States
Bleeding and Clotting Disorders Institute
Peoria, Illinois, 61614, United States
University of Kentucky Medical Center
Lexington, Kentucky, 40504, United States
University of Louisville Hospital
Louisville, Kentucky, 40202, United States
Tulane University Medical School
New Orleans, Louisiana, 70124, United States
Children's Mercy Hospitals & Clinics
Kansas City, Missouri, 66211, United States
Weill Cornell Medical College-New York Presbyterian Hospital
New York, New York, 10065, United States
University of North Carolina Chapel Hill
Chapel Hill, North Carolina, 27599, United States
Duke University Medical Center
Durham, North Carolina, 27710, United States
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229, United States
The Cleveland Clinic Foundation
Cleveland, Ohio, 44195, United States
Penn State Milton S. Hershey Medical Center
Hershey, Pennsylvania, 17033, United States
Palmetto Health
Columbia, South Carolina, 29203, United States
University of Utah Health Sciences Center
Salt Lake City, Utah, 84132, United States
Puget Sound Blood Group
Seattle, Washington, 98104, United States
Royal Adelaide Hospital
Adelaide, South Australia, 5000, Australia
The Alfred Hospital
Clayton, Victoria, 3168, Australia
Fremantle Hospital
Fremantle, Western Australia, 6160, Australia
Hollywood Specialist Centre
Nedlands, Western Australia, 6009, Australia
Landes-Frauen-und Kinderklinik Linz
Linz, 4020, Austria
AKH - Medizinische Universität Wien
Vienna, 1090, Austria
SHAT of Oncohaematology Diseases
Sofia, 1527, Bulgaria
Fakultni nemocnice Brno
Brno, 61300, Czechia
Fakultni nemocnice Olomouc
Olomouc, 775 20, Czechia
Fakultni nemocnice v Motole
Prague, 150 06, Czechia
Gerinnungszentrum Rhein-Ruhr
Duisburg, North Rhine-Westphalia, 47051, Germany
Vivantes Klinikum im Friedrichshain - Landsberger Allee
Berlin, 10249, Germany
Universitaetsklinikum Hamburg-Eppendorf
Hamburg, 20246, Germany
Werlhof-Institut MVZ
Hanover, 30159, Germany
Rambam Health Care Campus
Haifa, 3109601, Israel
Chaim Sheba Medical Center
Tel Aviv, 64239, 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
Hyogo College of Medicine Hospital
Nishinomiya-shi, Hyōgo, 663-8501, Japan
St. Marianna University School of Medicine Hospital
Kawasaki-shi, Kanagawa, 216-8511, Japan
Nara Medical University Hospital
Kashihara-shi, Nara, 634-8522, Japan
Tokyo Medical University Hospital
Shinjuku-ku, Tokyo-To, 160-0023, Japan
Ogikubo Hospital
Suginami-ku, Tokyo-To, 167-8515, Japan
Vilnius University Hospital Santariskiu Clinics, Public Institution
Vilnius, 08661, Lithuania
Children's Hospital, Affiliate of Vilnius University Hospital Santariskiu Klinikos
Vilnius, LT-08406, Lithuania
Hospital Pulau Pinang
George Town, Pulau Pinang, 10990, Malaysia
Hospital Tengku Ampuan Rahimah
Klang, Selangor, 41200, Malaysia
Pusat Darah Negara
Kuala Lumpur, 50400, 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, 011026, Romania
Chonnam National University Hwasun Hospital
Hwasun, Jeollanam-do, 519-763, South Korea
Pusan National University Hospital
Busan, 602-739, South Korea
Eulji University Hospital
Daejeon, 302-120, 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, 07010, Spain
Complejo Hospitalario Universitario A Coruña
A Coruña, La Coruña, 15006, Spain
Hospital Regional Universitario de Malaga
Málaga, Málaga, 29010, Spain
Hospital Universitari i Politecnic La Fe
Valencia, 46026, Spain
Skånes Universitetssjukhus, Malmö
Malmo, 20502, Sweden
Karolinska Universitetssjukhuset, Solna
Stockholm, 17176, Sweden
Universitatsspital Zurich
Zurich, 8091, Switzerland
Tri-Service General Hospital
Taipei, 11490, Taiwan
SI V.K.Gusak Emergency and Reconstructive Surgery Institute of NAMSU Center of IT
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
Royal Free Hospital
London, Greater London, NW3 2QG, United Kingdom
St Thomas' Hospital
London, Greater London, SE1 7EH, United Kingdom
Manchester Royal Infirmary
Manchester, Greater Manchester, M13 9WL, United Kingdom
Royal Manchester Children's Hospital
Manchester, Greater Manchester, M13 9WL, United Kingdom
Leicester Royal Infirmary
Leicester, Leicestershire, LE1 5WW, United Kingdom
Churchill Hospital
Oxford, Oxfordshire, OX3 7LJ, United Kingdom
Related Publications (1)
Konkle BA, Stasyshyn O, Chowdary P, Bevan DH, Mant T, Shima M, Engl W, Dyck-Jones J, Fuerlinger M, Patrone L, Ewenstein B, Abbuehl B. Pegylated, full-length, recombinant factor VIII for prophylactic and on-demand treatment of severe hemophilia A. Blood. 2015 Aug 27;126(9):1078-85. doi: 10.1182/blood-2015-03-630897. Epub 2015 Jul 8.
PMID: 26157075DERIVED
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 2
- Allocation
- NON RANDOMIZED
- Masking
- NONE
- Purpose
- PREVENTION
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
November 21, 2012
First Posted
November 29, 2012
Study Start
January 31, 2013
Primary Completion
July 17, 2014
Study Completion
July 17, 2014
Last Updated
May 20, 2021
Results First Posted
September 7, 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.