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Post-Marketing Non-Interventional Safety Evaluation of Obizur in the Treatment of Bleeding Episodes for Patients With Acquired Hemophilia A

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT02610127
Recruitment Status : Active, not recruiting
First Posted : November 20, 2015
Last Update Posted : May 2, 2019
Sponsor:
Collaborator:
Baxalta Innovations GmbH, now part of Shire
Information provided by (Responsible Party):
Shire ( Baxalta now part of Shire )

Brief Summary:
The overall objective is to enroll patients with acquired hemophilia A (AHA) who are prescribed and treated with Obizur, to assess safety, and to describe factors related to safety, utilization and effectiveness in a real-world setting.

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

Detailed Description:

This study is a multi-center, uncontrolled, open-label, non-interventional post-marketing safety surveillance study to describe the use of Obizur in patients with acquired hemophilia A (AHA), and secondarily, where data are available, to describe the hemostatic effectiveness and immunogenicity of Obizur.

Patients should be enrolled at the earliest possible time point after initiating Obizur.

In an attempt to collect safety and utilization data on patients treated with Obizur since Food and Drug Administration (FDA) approval in October 2014, Baxalta will make an effort to identify all persons treated with Obizur and to collect data for as many patients as possible.


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Study Type : Observational
Estimated Enrollment : 60 participants
Observational Model: Cohort
Time Perspective: Other
Official Title: Post-Marketing Non-Interventional Safety Evaluation of Obizur in the Treatment of Bleeding Episodes for Patients With Acquired Hemophilia A
Actual Study Start Date : December 30, 2015
Estimated Primary Completion Date : June 7, 2019
Estimated Study Completion Date : June 7, 2019


Group/Cohort Intervention/treatment
OBIZUR - Prospective Participants
Participants enrolled and treated with Obizur after the prospective study start date
Biological: OBIZUR
Treating physician will determine treatment regimen, frequency of laboratory and clinical assessments, according to routine clinical practice.
Other Names:
  • rpFVIII
  • Recombinant pFVIII
  • Antihemophilic Factor (Recombinant)
  • Porcine Sequence

OBIZUR - Retrospective Participants
Retrospective chart review of participants treated with OBIZUR from product approval date until prior to the prospective study start date
Biological: OBIZUR
Treating physician will determine treatment regimen, frequency of laboratory and clinical assessments, according to routine clinical practice.
Other Names:
  • rpFVIII
  • Recombinant pFVIII
  • Antihemophilic Factor (Recombinant)
  • Porcine Sequence




Primary Outcome Measures :
  1. Incidence of therapy-related SAEs and level of severity [ Time Frame: Throughout the study period of approximately 4 years ]

Secondary Outcome Measures :
  1. Hemostatic effectiveness assessment for resolution of bleeding [ Time Frame: Throughout the study period, up to approximately 4 years ]
    Determined as either bleeding stopped or did not stop

  2. Time to bleeding resolution, participant study termination, or switch to another treatment [ Time Frame: Throughout the study period, up to approximately 4 years ]
  3. Number of Obizur units/kg required for control of bleeding [ Time Frame: Throughout the study period, up to approximately 4 years ]
  4. Number of Obizur infusions required for control of bleeding [ Time Frame: Throughout the study period, up to approximately 4 years ]
  5. Titer of newly recognized anti-porcine Factor VIII (anti-pFVIII) neutralizing antibodies (inhibitors) or increase in titer of anti-pFVIII inhibitors from baseline and changes over time. [ Time Frame: Throughout the study period of approximately 4 years ]
  6. Impact of the inhibitor on hemostatic efficacy and any associated clinical manifestations. [ Time Frame: Throughout the study period of approximately 4 years ]
  7. Occurrence of hypersensitivity reactions [ Time Frame: Throughout the study period of approximately 4 years ]
  8. Occurrence of any thrombogenic event [ Time Frame: Throughout the study period of approximately 4 years ]


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Participant is ≥18 years of age at the time of informed consent.
  2. Participant has AHA, and is being treated/was treated with Obizur.
  3. Participant or the participant's legally authorized representative is willing and able to provide informed consent, unless informed consent is not required

Exclusion Criteria:

  1. Participant has a known anaphylactic reaction to the active substance, to any of the excipients, or to hamster protein.
  2. Participant has a concomitant bleeding disorder(s) other than acquired hemophilia A (AHA).
  3. Participant has participated in another 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 during the course of the study.

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): NCT02610127


Locations
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United States, Colorado
University of Colorado Health
Aurora, Colorado, United States, 80045
United States, Florida
University of Florida
Gainesville, Florida, United States, 32610
United States, Georgia
Emory University School of Medicine
Atlanta, Georgia, United States, 30322
United States, Illinois
Bleeding and Clotting Disorders Institute
Peoria, Illinois, United States, 61615
United States, Indiana
Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana, United States, 46260
United States, Iowa
University of Iowa Hospitals & Clinics
Iowa City, Iowa, United States, 52242
United States, Louisiana
Tulane University Hospital & Clinics
New Orleans, Louisiana, United States, 70112
United States, Michigan
Henry Ford Health System
Detroit, Michigan, United States, 48202
Michigan State University
East Lansing, Michigan, United States, 48823
United States, Missouri
Washington University
Saint Louis, Missouri, United States, 63110
United States, North Carolina
Duke University Medical Center
Durham, North Carolina, United States, 27710
Comprehensive Cancer Center of Wake Forest Unversity
Winston-Salem, North Carolina, United States, 27157
United States, Ohio
Cleveland Clinic
Cleveland, Ohio, United States, 44195
United States, Pennsylvania
University of Pennsylvania Health System
Philadelphia, Pennsylvania, United States, 19104
United States, Utah
University of Utah Health Sciences Center
Salt Lake City, Utah, United States, 84132
United States, Wisconsin
Blood Center of Southeast Wisconsin
Milwaukee, Wisconsin, United States, 53225
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: NCT02610127     History of Changes
Other Study ID Numbers: 241302
First Posted: November 20, 2015    Key Record Dates
Last Update Posted: May 2, 2019
Last Verified: April 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: Shire provides access to the de-identified individual participant data for eligible studies to aid qualified researchers in addressing legitimate scientific objectives. These IPDs will be provided following approval of a data sharing request, and under the terms of a data sharing agreement.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
Access Criteria: IPD from eligible studies will be shared with qualified researchers according to the criteria and process described in the Data Sharing section of the www.shiretrials.com website. 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.
URL: https://www.shiretrials.com/en/our-commitment-to-transparency/data-sharing-with-researchers

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