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Prospective, Non-interventional Study to Evaluate the Safety and Effectiveness of Obizur in Real-life Practice

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ClinicalTrials.gov Identifier: NCT03199794
Recruitment Status : Recruiting
First Posted : June 27, 2017
Last Update Posted : April 11, 2019
Sponsor:
Collaborator:
Baxalta Innovations GmbH, now part of Shire
Information provided by (Responsible Party):
Shire ( Baxalta now part of Shire )

Brief Summary:
The study addresses the safety, utilisation and effectiveness of Obizur in the treatment of bleeding episodes in real-life clinical practice in Europe.

Condition or disease Intervention/treatment
Acquired Hemophilia A Biological: OBIZUR

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Study Type : Observational
Estimated Enrollment : 50 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Prospective, Non-interventional Study to Evaluate the Safety and Effectiveness of Obizur in Real-life Practice
Actual Study Start Date : December 14, 2016
Estimated Primary Completion Date : May 31, 2021
Estimated Study Completion Date : May 31, 2021


Group/Cohort Intervention/treatment
OBIZUR participants
Participants previously treated with OBIZUR and continue to be treated with OBIZUR during the study.
Biological: OBIZUR
Treating physician will determine treatment regimen and frequency of laboratory and clinical assessments according to routine clinical practice.
Other Names:
  • Recombinant pFVIII
  • Porcine Sequence
  • rpFVIII
  • Antihemophilic Factor (Recombinant)




Primary Outcome Measures :
  1. Number of AEs and SAEs including seriousness, severity and outcome [ Time Frame: From first administration of Obizur up to 180 days after the last administration of Obizur. ]
    AE - adverse event, SAE - serious adverse event.

  2. Number of AESIs including seriousness, severity, relationship to therapy, outcome, and treatment discontinuation [ Time Frame: From first administration of Obizur up to 180 days after the last administration of Obizur. ]
    Adverse Events of Special Interest (AESI) are as follows: hypersensitivity reactions, thromboembolic events and dose dispensing medication errors.

  3. Number of thromboembolic events [ Time Frame: From first administration of Obizur up to 180 days after the last administration of Obizur. ]
    Thromboembolic events include disseminated intravascular coagulation (DIC), venous thrombosis, pulmonary embolism, myocardial infarction and stroke.

  4. Number of dose dispensing medication errors [ Time Frame: From first administration of Obizur up to 180 days after the last administration of Obizur. ]
    Dose dispensing medication erros include miscalculation of dose while prescribing (calculation of correct dose based on the participant's weight) or administration of the incorrect dose.


Secondary Outcome Measures :
  1. Immunogenicity; newly recognized anti-pFVIII inhibitor or increase in titre of anti-pFVIII inhibitors and evolution of titre over time [ Time Frame: From first administration of Obizur up to 180 days after the last administration of Obizur. ]
  2. Obizur treatment regimen, as available [ Time Frame: From first administration of Obizur up to 180 days after the last administration of Obizur. ]
    This may include details of the Obizur treatment regimen utilized, as available

  3. Other medication administered for haemostatic control, as available [ Time Frame: From first administration of Obizur up to 180 days after the last administration of Obizur. ]
    This may include additional medications, treatments and procedures (other than Obizur) undertaken to control a bleeding episode.

  4. Overall effectiveness assessment for resolution of bleeding [ Time Frame: From first administration of Obizur up to 180 days after the last administration of Obizur. ]
    Resolution of bleeding determined as either bleeding stopped or did not stop. If bleeding did not stop, a reason should be provided.

  5. Dose per infusion administered to achieve bleeding control, death or change in haemostatic treatment other than Obizur [ Time Frame: From first administration of Obizur up to 180 days after the last administration of Obizur. ]
    Bleeding control defined as all bleeding stopped.

  6. Number of infusions administered to achieve bleeding control, death or change in haemostatic treatment other than Obizur [ Time Frame: From first administration of Obizur up to 180 days after the last administration of Obizur. ]
    Bleeding control defined as all bleeding stopped.

  7. Time to achieve bleeding control, death or change in haemostatic treatment other than Obizur [ Time Frame: From first administration of Obizur up to 180 days after the last administration of Obizur. ]
    Bleeding control defined as all bleeding stopped.



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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
A participant with acquired hemophilia (AH) must be prescribed Obizur for the treatment of a bleeding episode by a physician, independent of and prior to the decision to enrol the participant in the study.
Criteria

Inclusion Criteria:

  • Adult participant (or legal representative) is willing to provide informed consent
  • Participant is being treated or was treated (treatment initiation within 30 days) with Obizur in routine clinical practice

Exclusion Criteria:

  • Participant has known anaphylactic reactions to the active substance, hamster protein or to any of the following excipients: Polysorbate 80; sodium chloride; calcium chloride dihydrate; sucrose; Tris Base; Tris HCl; Tri-sodium citrate dihydrate; sterilized water for injections
  • Participant has participated in a clinical study involving a medicinal product or device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving a medicinal product or device at study entry

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03199794


Contacts
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Contact: Shire Contact +1 866 842 5335 ClinicalTransparency@shire.com

Locations
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Austria
AKH - Medizinische Universität Wien Recruiting
Vienna, Austria, 1090
Germany
Vivantes Klinikum im Friedrichshain Recruiting
Berlin, Germany, 10249
Universitaetsklinikum Bonn Recruiting
Bonn, Germany, 53127
Klinikum der Johann Wolfgang Goethe-Universitaet Recruiting
Frankfurt, Germany, 60590
Medizinische Hochschule Hannover Recruiting
Hannover, Germany, 30625
Italy
Azienda Ospedaliera Nazionale Santi Antonio e Biagio e Cesare Arrigo Recruiting
Alessandria, Italy, 15100
Principal Investigator: Roberto Santi, MD         
Azienda Ospedaliera Pugliese Ciaccio Recruiting
Catanzaro, Italy, 88100
Azienda Ospedaliero Universitaria Policlinico Paolo Giaccone Recruiting
Palermo, Italy, 90127
Fondazione IRCCS Policlinico San Matteo Recruiting
Pavia, Italy, 27100
Contact: Site Contact    +390382502174    c.ambaglio@smatteo.pv.it   
Principal Investigator: Chiara Ambaglio, MD         
Policlinico Universitario Agostino Gemelli Recruiting
Roma, Italy, 00168
Umberto I Pol. di Roma-Università di Roma La Sapienza Recruiting
Rome, Italy, 00161
Principal Investigator: Antonio Chistolini, MD         
Istituto Clinico Humanitas Recruiting
Rozzano, Italy, 20089
Netherlands
Radboud University Medical Centre Recruiting
Nijmegen, Netherlands, 6525 GA
Principal Investigator: Saskia Schols, MD         
Sponsors and Collaborators
Baxalta now part of Shire
Baxalta Innovations GmbH, now part of Shire
Investigators
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Study Director: Study Director Shire

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Responsible Party: Baxalta now part of Shire
ClinicalTrials.gov Identifier: NCT03199794     History of Changes
Other Study ID Numbers: 241501
First Posted: June 27, 2017    Key Record Dates
Last Update Posted: April 11, 2019
Last Verified: April 2019

Additional relevant MeSH terms:
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Hemophilia A
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Factor VIII
Coagulants