Acquired Hemophilia A
Conditions
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.
Interventions
BIOLOGICALOBIZUR
Treating physician will determine treatment regimen, frequency of laboratory and clinical assessments, according to routine clinical practice.
Sponsors
Baxalta Innovations GmbH, now part of Shire
Baxalta now part of Shire
Study design
Observational model
COHORT
Time perspective
OTHER
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence of therapy-related SAEs and level of severity | Throughout the study period of approximately 4 years |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| 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 | — |
| Hemostatic effectiveness assessment for resolution of bleeding | Throughout the study period, up to approximately 4 years | Determined as either bleeding stopped or did not stop |
| 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 | — |
| 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 | — |
Countries
United States
Outcome results
None listed