Acquired Hemophilia ANCT02610127
Post-Marketing Non-Interventional Safety Evaluation of Obizur in the Treatment of Bleeding Episodes for Patients With Acquired Hemophilia A
An observational study of Acquired Hemophilia A, run by Baxalta now part of Shire at 16 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Acquired Hemophilia A
- What is being tested?
- OBIZUR
- How many people
- 53 participants
- Where
- 16 sitesUnited States
- Study dates
- 30 Dec 2015to 7 Jun 2019
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Participant is ≥18 years of age at the time of informed consent.
- Participant has AHA, and is being treated/was treated with Obizur.
- Participant or the participant's legally authorized representative is willing and able to provide informed consent, unless informed consent is not required
Who cannot join
- Participant has a known anaphylactic reaction to the active substance, to any of the excipients, or to hamster protein.
- Participant has a concomitant bleeding disorder(s) other than acquired hemophilia A (AHA).
- 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.
Trial locations and contacts
16 sites in 1 country.
16 sites in 1 country
United States16 sites
Emory University School of Medicine
Atlanta, Georgia, 30322
University of Colorado Health
Aurora, Colorado, 80045
Cleveland Clinic
Cleveland, Ohio, 44195
Henry Ford Health System
Detroit, Michigan, 48202
Duke University Medical Center
Durham, North Carolina, 27710
Michigan State University
East Lansing, Michigan, 48823
University of Florida
Gainesville, Florida, 32610
Indiana Hemophilia and Thrombosis Center
Indianapolis, Indiana, 46260
University of Iowa Hospitals & Clinics
Iowa City, Iowa, 52242
Blood Center of Southeast Wisconsin
Milwaukee, Wisconsin, 53225
Tulane University Hospital & Clinics
New Orleans, Louisiana, 70112
Bleeding and Clotting Disorders Institute
Peoria, Illinois, 61615
University of Pennsylvania Health System
Philadelphia, Pennsylvania, 19104
University of Utah Health Sciences Center
Salt Lake City, Utah, 84132
Washington University
St Louis, Missouri, 63110
Comprehensive Cancer Center of Wake Forest Unversity
Winston-Salem, North Carolina, 27157
About this study
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.
Read the 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.
- Conditions
- Acquired Hemophilia A
- Being tested
- OBIZUR
- Medical subject headings
- Factor 8 deficiency, acquired
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
OBIZURBiological
Also called rpFVIII, Recombinant pFVIII, Antihemophilic Factor (Recombinant), Porcine Sequence
Treating physician will determine treatment regimen, frequency of laboratory and clinical assessments, according to routine clinical practice.
| Study group | Role | What they receive |
|---|---|---|
| OBIZUR - Prospective Participants | Participants enrolled and treated with Obizur after the prospective study start date Receives: Biological: OBIZUR | |
| OBIZUR - Retrospective Participants | Retrospective chart review of participants treated with OBIZUR from product approval date until prior to the prospective study start date Receives: Biological: OBIZUR |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of therapy-related SAEs and level of severity
Measured: Throughout the study period of approximately 4 years
Secondary outcomes (8)
Hemostatic effectiveness assessment for resolution of bleeding
Determined as either bleeding stopped or did not stop
Measured: Throughout the study period, up to approximately 4 years
Time to bleeding resolution, participant study termination, or switch to another treatment
Measured: Throughout the study period, up to approximately 4 years
Number of Obizur units/kg required for control of bleeding
Measured: Throughout the study period, up to approximately 4 years
Number of Obizur infusions required for control of bleeding
Measured: 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.
Measured: Throughout the study period of approximately 4 years
Impact of the inhibitor on hemostatic efficacy and any associated clinical manifestations.
Measured: Throughout the study period of approximately 4 years
Occurrence of hypersensitivity reactions
Measured: Throughout the study period of approximately 4 years
Occurrence of any thrombogenic event
Measured: Throughout the study period of approximately 4 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Nov 2015 (estimated)
- Study start30 Dec 2015
- Primary completion7 Jun 2019
- Study completion7 Jun 2019
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Acquired Hemophilia AStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Other
- Enrolment
- 53 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baxalta now part of Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Baxalta Innovations GmbH, now part of Shire
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Shire
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02610127
- Sponsor's protocol ID
- 241302
- Other IDs
- EUPAS36659
- Status last verified
- Aug 2021
- First submitted
- 29 Oct 2015
- Last updated
- 5 Aug 2021
- Results posted
- No
- Data monitoring committee
- No
- Individual data sharing
- Yes: 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.
Study at a glance
From the registry record, last updated by the sponsor on 5 Aug 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Post-Marketing Non-Interventional Safety Evaluation of Obizur in the Treatment of Bleeding Episodes for Patients With Acquired Hemophilia A
- ClinicalTrials.gov ID
- NCT02610127
- Acronym / sponsor ID
- 241302
- Conditions
- Acquired Hemophilia A
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 53 participants (actual)
- Lead sponsor
- Baxalta now part of ShireIndustry (pharma, biotech, devices)
- Collaborators
- Baxalta Innovations GmbH, now part of Shire
- Study start
- 30 Dec 2015 (actual)
- Primary completion
- 7 Jun 2019 (actual)
- Study completion
- 7 Jun 2019 (actual)
- First posted
- 20 Nov 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Other
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| OBIZUR - Prospective Participants | – | Biological: OBIZURParticipants enrolled and treated with Obizur after the prospective study start date |
| OBIZUR - Retrospective Participants | – | Biological: OBIZURRetrospective chart review of participants treated with OBIZUR from product approval date until prior to the prospective study start date |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- Participant is ≥18 years of age at the time of informed consent.
- Participant has AHA, and is being treated/was treated with Obizur.
- Participant or the participant's legally authorized representative is willing and able to provide informed consent, unless informed consent is not required
Key exclusion criteria (3 listed)
- Participant has a known anaphylactic reaction to the active substance, to any of the excipients, or to hamster protein.
- Participant has a concomitant bleeding disorder(s) other than acquired hemophilia A (AHA).
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| OBIZURAlso: rpFVIII, Recombinant pFVIII, Antihemophilic Factor (Recombinant), Porcine SequenceTreating physician will determine treatment regimen, frequency of laboratory and clinical assessments, according to routine clinical practice. | Biological | OBIZUR - Prospective Participants, OBIZUR - Retrospective Participants |
Primary outcome
Incidence of therapy-related SAEs and level of severity
Time frame: Throughout the study period of approximately 4 years
8 secondary outcomes
Hemostatic effectiveness assessment for resolution of bleeding
Time frame: 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
Number of Obizur units/kg required for control of bleeding
Time frame: 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
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
Impact of the inhibitor on hemostatic efficacy and any associated clinical manifestations.
Time frame: Throughout the study period of approximately 4 years
Occurrence of hypersensitivity reactions
Time frame: Throughout the study period of approximately 4 years
Occurrence of any thrombogenic event
Time frame: Throughout the study period of approximately 4 years
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 5 Aug 2021.
- To obtain more information on the study, click here/on this linkLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. 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.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Study Director study director
Shire
16 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Acquired Hemophilia A, run by Baxalta now part of Shire at 16 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 5 Aug 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 16 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of therapy-related SAEs and level of severity (time frame: Throughout the study period of approximately 4 years). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Aug 2021.