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

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: Completed
• Conditions: Acquired Hemophilia A
• Intervention / Treatment: 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.

• Study Type: Observational
• Enrollment (Actual): 53

Contacts and Locations

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

Eligibility Criteria

• 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

How is the study designed?

Design Details

• Observational Models: Cohort
• Time Perspectives: Other

Number of groups / cohorts

2

Cohorts and Interventions

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: 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

Outcome Measure	Time Frame
Incidence of therapy-related SAEs and level of severity	Throughout the study period of approximately 4 years

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Hemostatic effectiveness assessment for resolution of bleeding	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		Throughout the study period, up to approximately 4 years
Number of Obizur units/kg required for control of bleeding		Throughout the study period, up to approximately 4 years
Number of Obizur infusions required for control of bleeding		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.		Throughout the study period of approximately 4 years
Impact of the inhibitor on hemostatic efficacy and any associated clinical manifestations.		Throughout the study period of approximately 4 years
Occurrence of hypersensitivity reactions		Throughout the study period of approximately 4 years
Occurrence of any thrombogenic event		Throughout the study period of approximately 4 years

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 30, 2015
• Primary Completion (Actual): June 7, 2019
• Study Completion (Actual): June 7, 2019

Study Registration Dates

• First Submitted: October 29, 2015
• First Submitted That Met QC Criteria: November 18, 2015
• First Posted (Estimate): November 20, 2015

Study Record Updates

• Last Update Posted (Actual): August 5, 2021
• Last Update Submitted That Met QC Criteria: August 4, 2021
• Last Verified: August 1, 2021

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: 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)