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

August 4, 2021 updated by: Baxalta now part of Shire
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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
53

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Colorado
      • Aurora, Colorado, United States, 80045
        • University of Colorado Health
    • Florida
      • Gainesville, Florida, United States, 32610
        • University of Florida
    • Georgia
      • Atlanta, Georgia, United States, 30322
        • Emory University School of Medicine
    • Illinois
      • Peoria, Illinois, United States, 61615
        • Bleeding and Clotting Disorders Institute
    • Indiana
      • Indianapolis, Indiana, United States, 46260
        • Indiana Hemophilia and Thrombosis Center
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • University of Iowa Hospitals & Clinics
    • Louisiana
      • New Orleans, Louisiana, United States, 70112
        • Tulane University Hospital & Clinics
    • Michigan
      • Detroit, Michigan, United States, 48202
        • Henry Ford Health System
      • East Lansing, Michigan, United States, 48823
        • Michigan State University
    • Missouri
      • Saint Louis, Missouri, United States, 63110
        • Washington University
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Duke University Medical Center
      • Winston-Salem, North Carolina, United States, 27157
        • Comprehensive Cancer Center of Wake Forest Unversity
    • Ohio
      • Cleveland, Ohio, United States, 44195
        • Cleveland Clinic
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • University of Pennsylvania Health System
    • Utah
      • Salt Lake City, Utah, United States, 84132
        • University of Utah Health Sciences Center
    • Wisconsin
      • Milwaukee, Wisconsin, United States, 53225
        • Blood Center of Southeast Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Other

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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:
  • Antihemophilic Factor (Recombinant)
  • rpFVIII
  • Recombinant pFVIII
  • 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:
  • Antihemophilic Factor (Recombinant)
  • rpFVIII
  • Recombinant pFVIII
  • Porcine Sequence

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Incidence of therapy-related SAEs and level of severity
Time Frame: Throughout the study period of approximately 4 years
Throughout the study period of approximately 4 years

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
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
Throughout the study period, up to approximately 4 years
Time to bleeding resolution, participant study termination, or switch to another treatment
Time Frame: Throughout the study period, up to approximately 4 years
Throughout the study period, up to approximately 4 years
Number of Obizur units/kg required for control of bleeding
Time Frame: Throughout the study period, up to approximately 4 years
Throughout the study period, up to approximately 4 years
Number of Obizur infusions required for control of bleeding
Time Frame: Throughout the study period, up to approximately 4 years
Throughout the study period, up to approximately 4 years
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
Throughout the study period of approximately 4 years
Impact of the inhibitor on hemostatic efficacy and any associated clinical manifestations.
Time Frame: Throughout the study period of approximately 4 years
Throughout the study period of approximately 4 years
Occurrence of hypersensitivity reactions
Time Frame: Throughout the study period of approximately 4 years
Throughout the study period of approximately 4 years
Occurrence of any thrombogenic event
Time Frame: Throughout the study period of approximately 4 years
Throughout the study period of approximately 4 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Baxalta now part of Shire

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Baxalta Innovations GmbH, now part of Shire

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
December 30, 2015

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
June 7, 2019

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
June 7, 2019

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
October 29, 2015

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
November 18, 2015

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
November 20, 2015

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
August 5, 2021

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 4, 2021

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 241302
  • EUPAS36659 (Registry Identifier: EUPAS)

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)

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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