Clinical trial NCT02610127: Post-Marketing Non-Interventional Safety Evaluation of Obizur in the Treatment of Bleeding Episodes for Patients With Acquired Hemophilia A
Official title: Post-Marketing Non-Interventional Safety Evaluation of Obizur in the Treatment of Bleeding Episodes for Patients With Acquired Hemophilia A
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Acquired Hemophilia A
Lead sponsor: Takeda
Sponsor class: INDUSTRY
Collaborators: name: Baxalta Innovations GmbH, now part of Shire
First posted: 2015-11-20
Enrollment: 53
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER

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.

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.

Eligibility 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.

Primary outcomes:
measure: Incidence of therapy-related SAEs and level of severity
timeFrame: Throughout the study period of approximately 4 years
Secondary outcomes:
measure: Hemostatic effectiveness assessment for resolution of bleeding
timeFrame: Throughout the study period, up to approximately 4 years
measure: Time to bleeding resolution, participant study termination, or switch to another treatment
timeFrame: Throughout the study period, up to approximately 4 years
measure: Number of Obizur units/kg required for control of bleeding
timeFrame: Throughout the study period, up to approximately 4 years
measure: Number of Obizur infusions required for control of bleeding
timeFrame: Throughout the study period, up to approximately 4 years
measure: 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.
timeFrame: Throughout the study period of approximately 4 years
measure: Impact of the inhibitor on hemostatic efficacy and any associated clinical manifestations.
timeFrame: Throughout the study period of approximately 4 years
measure: Occurrence of hypersensitivity reactions
timeFrame: Throughout the study period of approximately 4 years
measure: Occurrence of any thrombogenic event
timeFrame: Throughout the study period of approximately 4 years
Other outcomes:
Locations:
city: Aurora
country: United States
facility: University of Colorado Health
state: Colorado
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Atlanta
country: United States
facility: Emory University School of Medicine
state: Georgia
city: Peoria
country: United States
facility: Bleeding and Clotting Disorders Institute
state: Illinois
city: Indianapolis
country: United States
facility: Indiana Hemophilia and Thrombosis Center
state: Indiana
city: Iowa City
country: United States
facility: University of Iowa Hospitals & Clinics
state: Iowa
city: New Orleans
country: United States
facility: Tulane University Hospital & Clinics
state: Louisiana
city: Detroit
country: United States
facility: Henry Ford Health System
state: Michigan
city: East Lansing
country: United States
facility: Michigan State University
state: Michigan
city: Saint Louis
country: United States
facility: Washington University
state: Missouri
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Winston-Salem
country: United States
facility: Comprehensive Cancer Center of Wake Forest Unversity
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Philadelphia
country: United States
facility: University of Pennsylvania Health System
state: Pennsylvania
city: Salt Lake City
country: United States
facility: University of Utah Health Sciences Center
state: Utah
city: Milwaukee
country: United States
facility: Blood Center of Southeast Wisconsin
state: Wisconsin
Study officials:
affiliation: Shire
name: Study Director
role: STUDY_DIRECTOR
References: