Now Recruiting

Safety, immunogenicity and hemostatic efficacy of PEGylated recombinant FVIII (BAX 855) in previously untreated patients (PUPs) < 6 years of age with severe hemophilia A

WHAT IS THE PURPOSE OF THIS STUDY?

The purpose of this study is to investigate safety, immunogenicity and hemostatic efficacy of PEGylated recombinant FVIII (BAX 855) in previously untreated patients (PUPs) < 6 years of age with severe hemophilia A (baseline FVIII level < 1%) and < 3 EDs to ADVATE, BAX 855 or plasma transfusion.

TRIAL AT A GLANCE

Condition

Hemophilia A

Study Intervention

PEGylated Recombinant Factor VIII

Possibility of Active Treatment

Study Type

Interventional

Phase

Phase 3

IS THIS STUDY RIGHT FOR ME?

AGE

UP TO 5

Years

GENDERS

ACCEPTS HEALTHY VOLUNTEERS

No

ENTRY CRITERIA

Inclusion Criteria1. Participant is < 6 years old at the time of screening2. Participant is previously untreated with < 3 exposure days (EDs) to ADVATE, BAX 855 or plasma transfusion at any time prior to screening3. Participant has severe hemophilia A (Factor VIII (FVIII) < 1%) as determined by the central laboratory, or a historical FVIII level < 1% as determined at any local laboratory, optionally supported by an additional FVIII gene mutation consistent with severe hemophilia A4. Participant is immune competent with a CD4+ count > 200 cells/mm^3, as confirmed by the central laboratory at screening5. Parent or legally authorized representative is willing and able to comply with the requirements of the protocolAdditional inclusion criteria for Part B (immune tolerance induction (ITI))1. Parent or legal representative has/have voluntarily provided signed informed consent for ITI portion2. Participant has a confirmed positive high titer inhibitor (> 5.00 Bethesda unit (BU)) or has a positive confirmed low titer inhibitor (≥ 0.6 BU) as determined by the central laboratory based on a second repeat blood sample with a. poorly controlled bleeding despite increased BAX 855 doses, or b. requires bypassing agents to treat bleeding

Exclusion Criteria1. Participant has detectable FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of the Bethesda assay) as confirmed by central laboratory at screening2. Participant has a history of FVIII inhibitory antibodies (≥ 0.6 BU using the Nijmegen modification of the Bethesda assay or the Bethesda assay) at any time prior to screening3. Participant has been diagnosed with an inherited or acquired hemostatic defect other than hemophilia A (eg, qualitative platelet defect or von Willebrand’s disease)4. Participant has been previously treated with any type of FVIII concentrate other than ADVATE or BAX 855, or was administered ADVATE, BAX 855 or plasma transfusion for ≥ 3 EDs at any time prior to screening5. Participant receives >2 EDs of ADVATE in total during the periods prior to enrollment and during the screening period, until the baseline infusion.6. The participant’s weight is anticipated to be < 5 kg at the baseline visit7. Participant’s platelet count is < 100,000/mL8. Participant has known hypersensitivity towards mouse or hamster proteins, polyethylene glycol (PEG) or Tween 809. Participant has severe chronic hepatic dysfunction [eg, > 5 times upper limit of normal alanine aminotransferase (ALT), aspartate aminotransferase (AST), or a documented international normalized ratio (INR) > 1.5] in his medical history or at the time of screening10. Participant has severe renal impairment (serum creatinine > 1.5 times the upper limit of normal)11. 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 participation12. Participant is scheduled to receive during the course of the study a systemic immunomodulating drug (e.g. corticosteroid agents at a dose equivalent to hydrocortisone greater than 10 mg/day or α-interferon) other than anti-retroviral chemotherapy13. 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 study14. Parent or legally authorized representative has a medical, psychiatric, or cognitive illness or recreational drug/alcohol use that, in the opinion of the investigator, would affect participant safety or compliance15. Parent, legally authorized representative or participant are 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.Additional exclusion criteria for Part B (ITI)1. Spontaneous disappearance of the inhibitor prior to ITI2. FVIII inhibitor titer ≥ 0.6 BU is not confirmed by a second new blood sample and determined at the central laboratory3. Inability or unwillingness to comply with the protocol

Study Locations

We are planning* on conducting this study in 21 countries around the world.

    • Austria
    • Belgium
    • Bulgaria
    • Czech Republic
    • Denmark
    • France
    • Germany
    • Hong Kong
    • Italy
    • Korea (Republic of)
    • Malaysia
    • Netherlands
    • Norway
    • Romania
    • Singapore
    • Spain
    • Taiwan, Province of China
    • Thailand
    • Ukraine
    • United Kingdom
    • United States of America

Enter your city or postal code to find a location near you.

RESULTS

  • 1.

    Royal Manchester Children's Hospital

    Manchester, Greater Manchester
    United Kingdom

    miles

  • 2.

    Medizinische Universitat Wien

    Vienna,
    Austria

    miles

  • 3.

    SHAT Oncohaematology Diseases

    Sofia,
    Bulgaria

    miles

  • 4.

    MHAT Sv. Marina, EAD

    Varna,
    Bulgaria

    miles

  • 5.

    UMHAT Sv. Georgi, EAD

    Plovdiv,
    Bulgaria

    miles

  • 6.

    Fakultni nemocnice v Motole

    Praha 5,
    Czech Republic

    miles

  • 7.

    Hopital Necker Enfants Malades

    Paris cedex 15,
    France

    miles

  • 8.

    Hospital Pulau Pinang

    Georgetown, Pulau Pinang
    Malaysia

    miles

  • 9.

    Hospital Umum Sarawak

    Kuching, Kuching
    Malaysia

    miles

  • 10.

    Hospital Ampang

    Ampang, Kuala Lumpur
    Malaysia

    miles

  • 11.

    Hospital Kuala Lumpur

    Kuala Lumpur, Kuala Lumpur
    Malaysia

    miles

  • 12.

    Hospital HRPB

    Ipoh, Perak
    Malaysia

    miles

  • 13.

    HOSPITAL A Coruna

    A Coruna,
    Spain

    miles

  • 14.

    Hospital Universitario La Paz

    Madrid, Madrid
    Spain

    miles

  • 15.

    Taichung Veterans General

    Taichung,
    Taiwan, Province of China

    miles

  • 16.

    SI Institute of Blood Pathology and Transfusion Medicine of NAMSU

    Lviv,
    Ukraine

    miles

  • 17.

    Bristol Royal H. for Children

    Bristol,
    United Kingdom

    miles

  • 18.

    Evelina Children's Hospital - St Thomas' Hospital

    London,
    United Kingdom

    miles

  • 19.

    Univ Hospital Southampton

    Southampton, Hampshire
    United Kingdom

    miles

  • 20.

    Bleeding and Clotting Dis.Inst.

    Peoria, Illinois
    United States of America

    miles

  • 21.

    Cincinnati Children's Hospital

    Cincinnati, Ohio
    United States of America

    miles

  • 22.

    Phoenix Childrens Hospital

    Phoenix, Arizona
    United States of America

    miles

  • 23.

    Penn State MS Hershey Med Ctr

    Hershey, Pennsylvania
    United States of America

    miles

  • 24.

    The Presbyterian Hospital

    Charlotte, North Carolina
    United States of America

    miles

  • 25.

    Ulsan University Hospital

    Ulsan,
    Korea (Republic of)

    miles

  • 26.

    Severance Hospital, Yonsei

    Seoul,
    Korea (Republic of)

    miles

  • 27.

    Tri-Service General Hospital

    Taipei City,
    Taiwan, Province of China

    miles

  • 28.

    HUDERF

    Bruxelles,
    Belgium

    miles

  • 29.

    Cliniques Uni Saint-Luc

    Bruxelles,
    Belgium

    miles

  • 30.

    CHU CAEN Hopital Cote de Nacre

    Caen cedex 9, Calvados
    France

    miles

  • 31.

    Ospedale Maggiore Policlinico

    Milano,
    Italy

    miles

  • 32.

    Rigshospitalet Copenhagen

    Copenhagen,
    Denmark

    miles

  • 33.

    CHU de Rouen

    ROUEN Cedex,
    France

    miles

  • 34.

    Hopital Jeanne de Flandre - CHU Lille

    Lille Cedex,
    France

    miles

  • 35.

    Presidio Ospedaliero F. Alessi

    Catania, Catania
    Italy

    miles

  • 36.

    Umberto I Pol. di Roma-Università di Roma La Sapienza

    Rome,
    Italy

    miles

  • 37.

    Eulji University Hospital

    Daejeon,
    Korea (Republic of)

    miles

  • 38.

    Spitalul Clinic

    Targu Mures,
    Romania

    miles

  • 39.

    KKH

    Singapore,
    Singapore

    miles

  • 40.

    China Medical University Hospital

    Taichung,
    Taiwan, Province of China

    miles

  • 41.

    Kaohsiung Chung- Ho Memorial Hosp

    Kaohsiung,
    Taiwan, Province of China

    miles

  • 42.

    Rainbow Babies/Childrens Htl

    Cleveland, Ohio
    United States of America

    miles

  • 43.

    Univ Florida College Medicine

    Gainesville, Florida
    United States of America

    miles

  • 44.

    Primary Children's Medical Center

    Salt Lake City, Utah
    United States of America

    miles

  • 45.

    Ann & Robert H. Lurie Children's H

    Chicago, Illinois
    United States of America

    miles

  • 46.

    Emory University

    Atlanta, Georgia
    United States of America

    miles

  • 47.

    Texas Children's Hospital

    Houston, Texas
    United States of America

    miles

  • 48.

    Weill Cornell Medical College

    New York, New York
    United States of America

    miles

  • 49.

    Hospital Sultanah Nur Zahirah

    Terengganu,
    Malaysia

    miles

  • 50.

    Chinese University Of Hong Kong

    Shatin,
    Hong Kong

    miles

  • 51.

    The University of Hong Kong Queen Mary Hospital

    Hong Kong,
    Hong Kong

    miles

  • 52.

    Srinagarind Hospital

    Muang, Khon Kaen
    Thailand

    miles

  • 53.

    Ramathibodi Hospital

    Ratchathewi, Bangkok
    Thailand

    miles

  • 54.

    Siriraj Hospital

    Bangkoknoi, Bangkok
    Thailand

    miles

  • 55.

    King Chulalongkorn Memorial

    Patumwan, Bangkok
    Thailand

    miles

  • 56.

    Maharaj Nakorn Chiang Mai

    Muang, Chiang Mai
    Thailand

    miles

  • 57.

    University of California

    Sacramento, California
    United States of America

    miles

  • 58.

    Werlhof-Institut GmbH

    Hannover, Niedersachsen
    Germany

    miles

  • 59.

    Azienda Ospedaliera Universitaria Careggi

    Firenze, Firenze
    Italy

    miles

  • 60.

    Universitair Medisch Centrum Groningen (UMCG)

    Groningen,
    Netherlands

    miles

  • 61.

    Oslo Universitetssykehus - Rikshospitalet

    Oslo,
    Norway

    miles

*Planning is an intent to conduct this study in the specified countries and is subject to change.