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

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

Study Overview

• Status: Completed
• Conditions: Acquired Hemophilia A
• Intervention / Treatment: Biological: OBIZUR

• Study Type: Observational
• Enrollment (Actual): 50

Contacts and Locations

Study Locations

• Austria
  • Vienna, Austria, 1090
    • AKH - Medizinische Universitat Wien
• France
  • Rouen, France, 76000
    • CHU de Rouen - Hôpital Charles Nicolle
• Germany
  • Berlin, Germany, 10249
    • Vivantes Klinikum im Friedrichshain
  • Bonn, Germany, 53127
    • Universitaetsklinikum Bonn
  • Dresden, Germany, 01304
    • Universitaetsklinikum Carl Gustav Carus TU Dresden
  • Frankfurt, Germany, 60590
    • Klinikum der Johann Wolfgang Goethe-Universitaet
  • Hannover, Germany, 30625
    • Medizinische Hochschule Hannover
• Italy
  • Alessandria, Italy, 15100
    • Azienda Ospedaliera Nazionale Santi Antonio e Biagio e Cesare Arrigo
  • Catanzaro, Italy, 88100
    • Azienda Ospedaliera Pugliese Ciaccio
  • Palermo, Italy, 90127
    • Azienda Ospedaliero Universitaria Policlinico Paolo Giaccone
  • Pavia, Italy, 27100
    • Fondazione IRCCS Policlinico San Matteo
  • Roma, Italy, 00168
    • Policlinico Universitario Agostino Gemelli
  • Rome, Italy, 00161
    • Umberto I Pol. di Roma-Università di Roma La Sapienza
  • Rozzano, Italy, 20089
    • Istituto Clinico Humanitas
• Netherlands
  • Nijmegen, Netherlands, 6525 GA
    • Radboud University Medical Centre
• United Kingdom
  • Birmingham, United Kingdom, B15 2TH
    • University Hospital Birmingham
  • Leeds, United Kingdom, LS9 7TF
    • St James's University Hospital
  • London, United Kingdom, NW3 2QG
    • Royal Free Hospital
  • Oxford, United Kingdom, OX3 7LE
    • Churchill Hospital
  • Southampton, United Kingdom, SO16 5YA
    • Southampton General Hospital
• United States
  • Florida
    • Gainesville, Florida, United States, 32608
      • University of Florida - Shands
  • Michigan
    • Detroit, Michigan, United States, 48202
      • Henry Ford Hospital
  • North Carolina
    • Durham, North Carolina, United States, 27710
      • Duke University Medical Center
  • Ohio
    • Cleveland, Ohio, United States, 44106
      • University Hospitals Cleveland Medical Center
    • Cleveland, Ohio, United States, 44103
      • Cleveland Clinic
  • Texas
    • Houston, Texas, United States, 77030
      • The University of Texas Health Science Center at Houston

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

• 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.

Description

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

Study Plan

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Prospective

Number of groups / cohorts

1

Cohorts and Interventions

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: Antihemophilic Factor (Recombinant); rpFVIII; Recombinant pFVIII; Porcine Sequence

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of AEs and SAEs including seriousness, severity and outcome	AE - adverse event, SAE - serious adverse event.	From first administration of Obizur up to 180 days after the last administration of Obizur.
Number of AESIs including seriousness, severity, relationship to therapy, outcome, and treatment discontinuation	Adverse Events of Special Interest (AESI) are as follows: hypersensitivity reactions, thromboembolic events and dose dispensing medication errors.	From first administration of Obizur up to 180 days after the last administration of Obizur.
Number of thromboembolic events	Thromboembolic events include disseminated intravascular coagulation (DIC), venous thrombosis, pulmonary embolism, myocardial infarction and stroke.	From first administration of Obizur up to 180 days after the last administration of Obizur.
Number of dose dispensing medication errors	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.	From first administration of Obizur up to 180 days after the last administration of Obizur.

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Immunogenicity; newly recognized anti-pFVIII inhibitor or increase in titre of anti-pFVIII inhibitors and evolution of titre over time		From first administration of Obizur up to 180 days after the last administration of Obizur.
Obizur treatment regimen, as available	This may include details of the Obizur treatment regimen utilized, as available	From first administration of Obizur up to 180 days after the last administration of Obizur.
Other medication administered for haemostatic control, as available	This may include additional medications, treatments and procedures (other than Obizur) undertaken to control a bleeding episode.	From first administration of Obizur up to 180 days after the last administration of Obizur.
Overall effectiveness assessment for resolution of bleeding	Resolution of bleeding determined as either bleeding stopped or did not stop. If bleeding did not stop, a reason should be provided.	From first administration of Obizur up to 180 days after the last administration of Obizur.
Dose per infusion administered to achieve bleeding control, death or change in haemostatic treatment other than Obizur	Bleeding control defined as all bleeding stopped.	From first administration of Obizur up to 180 days after the last administration of Obizur.
Number of infusions administered to achieve bleeding control, death or change in haemostatic treatment other than Obizur	Bleeding control defined as all bleeding stopped.	From first administration of Obizur up to 180 days after the last administration of Obizur.
Time to achieve bleeding control, death or change in haemostatic treatment other than Obizur	Bleeding control defined as all bleeding stopped.	From first administration of Obizur up to 180 days after the last administration of Obizur.

Collaborators and Investigators

• Sponsor: Baxalta now part of Shire
• Collaborators: Baxalta Innovations GmbH, now part of Shire

Publications and helpful links

Helpful Links

• To obtain more information on the study, click here/on this link

Study record dates

Study Major Dates

• Study Start (Actual): December 14, 2016
• Primary Completion (Actual): July 30, 2021
• Study Completion (Actual): July 30, 2021

Study Registration Dates

• First Submitted: June 23, 2017
• First Submitted That Met QC Criteria: June 23, 2017
• First Posted (Actual): June 27, 2017

Study Record Updates

• Last Update Posted (Actual): July 21, 2022
• Last Update Submitted That Met QC Criteria: July 18, 2022
• Last Verified: July 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Hematologic Diseases; Blood Coagulation Disorders, Inherited; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Blood Coagulation Disorders; Hemophilia A; Coagulants; Factor VIII
• Other Study ID Numbers: 241501; EUPAS16055 (Registry Identifier: ENCePP)

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: Yes
• IPD Plan Description: 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.
• IPD Sharing 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.
• IPD Sharing Supporting Information Type: Study Protocol; Statistical Analysis Plan (SAP); Informed Consent Form (ICF); Clinical Study Report (CSR)