Acquired Hemophilia ANCT03199794
Prospective and Retrospective, Non-interventional Study to Evaluate the Safety and Effectiveness of Obizur in Real-life Practice
An observational study of Acquired Hemophilia A, run by Baxalta now part of Shire at 26 sites in 7 countries.
Trial summary
The study addresses the safety, utilisation and effectiveness of Obizur in the treatment of bleeding episodes in real-life clinical practice in Europe and the United States. 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
- 50 participants
- Where
- 26 sitesin 7 countries
- Study dates
- 14 Dec 2016to 30 Jul 2021
- 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
Study population: A participant with acquired hemophilia (AH) must be prescribed Obizur for the treatment of a bleeding episode by a physician, independent of and prior to the decision to enrol the participant in the study.
Who may be able to join
- Adult participant (or legal representative) is willing to provide informed consent
- Participant is being treated or was treated (treatment initiation within 30 days) with Obizur in routine clinical practice
Who cannot join
- Participant has known anaphylactic reactions to the active substance, hamster protein or to any of the following excipients: Polysorbate 80; sodium chloride; calcium chloride dihydrate; sucrose; Tris Base; Tris HCl; Tri-sodium citrate dihydrate; sterilized water for injections
- Participant has participated in a 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 at study entry
Trial locations and contacts
26 sites in 7 countries.
26 sites in 7 countries
Italy7 sites
Azienda Ospedaliera Nazionale Santi Antonio e Biagio e Cesare Arrigo
Alessandria, 15100
Azienda Ospedaliera Pugliese Ciaccio
Catanzaro, 88100
Azienda Ospedaliero Universitaria Policlinico Paolo Giaccone
Palermo, 90127
Fondazione IRCCS Policlinico San Matteo
Pavia, 27100
Policlinico Universitario Agostino Gemelli
Roma, 00168
Umberto I Pol. di Roma-Università di Roma La Sapienza
Rome, 00161
Istituto Clinico Humanitas
Rozzano, 20089
United States6 sites
Cleveland Clinic
Cleveland, Ohio, 44103
University Hospitals Cleveland Medical Center
Cleveland, Ohio, 44106
Henry Ford Hospital
Detroit, Michigan, 48202
Duke University Medical Center
Durham, North Carolina, 27710
University of Florida - Shands
Gainesville, Florida, 32608
The University of Texas Health Science Center at Houston
Houston, Texas, 77030
Germany5 sites
Vivantes Klinikum im Friedrichshain
Berlin, 10249
Universitaetsklinikum Bonn
Bonn, 53127
Universitaetsklinikum Carl Gustav Carus TU Dresden
Dresden, 01304
Klinikum der Johann Wolfgang Goethe-Universitaet
Frankfurt, 60590
Medizinische Hochschule Hannover
Hanover, 30625
United Kingdom5 sites
University Hospital Birmingham
Birmingham, B15 2TH
St James's University Hospital
Leeds, LS9 7TF
Royal Free Hospital
London, NW3 2QG
Churchill Hospital
Oxford, OX3 7LE
Southampton General Hospital
Southampton, SO16 5YA
About this study
The study addresses the safety, utilisation and effectiveness of Obizur in the treatment of bleeding episodes in real-life clinical practice in Europe and the United States.
- 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 Recombinant pFVIII, Porcine Sequence, rpFVIII, Antihemophilic Factor (Recombinant)
Treating physician will determine treatment regimen and frequency of laboratory and clinical assessments according to routine clinical practice.
| Study group | Role | What they receive |
|---|---|---|
| OBIZUR participants | Participants previously treated with OBIZUR and continue to be treated with OBIZUR during the study. Receives: Biological: OBIZUR |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of AEs and SAEs including seriousness, severity and outcome
AE - adverse event, SAE - serious adverse event.
Measured: From first administration of Obizur up to 180 days after the last administration of Obizur.
Number of AESIs including seriousness, severity, relationship to therapy, outcome, and treatment discontinuation
Adverse Events of Special Interest (AESI) are as follows: hypersensitivity reactions, thromboembolic events and dose dispensing medication errors.
Measured: From first administration of Obizur up to 180 days after the last administration of Obizur.
Number of thromboembolic events
Thromboembolic events include disseminated intravascular coagulation (DIC), venous thrombosis, pulmonary embolism, myocardial infarction and stroke.
Measured: From first administration of Obizur up to 180 days after the last administration of Obizur.
Number of dose dispensing medication errors
Dose dispensing medication erros include miscalculation of dose while prescribing (calculation of correct dose based on the participant's weight) or administration of the incorrect dose.
Measured: From first administration of Obizur up to 180 days after the last administration of Obizur.
Secondary outcomes (7)
Immunogenicity; newly recognized anti-pFVIII inhibitor or increase in titre of anti-pFVIII inhibitors and evolution of titre over time
Measured: From first administration of Obizur up to 180 days after the last administration of Obizur.
Obizur treatment regimen, as available
This may include details of the Obizur treatment regimen utilized, as available
Measured: From first administration of Obizur up to 180 days after the last administration of Obizur.
Other medication administered for haemostatic control, as available
This may include additional medications, treatments and procedures (other than Obizur) undertaken to control a bleeding episode.
Measured: From first administration of Obizur up to 180 days after the last administration of Obizur.
Overall effectiveness assessment for resolution of bleeding
Resolution of bleeding determined as either bleeding stopped or did not stop. If bleeding did not stop, a reason should be provided.
Measured: From first administration of Obizur up to 180 days after the last administration of Obizur.
Dose per infusion administered to achieve bleeding control, death or change in haemostatic treatment other than Obizur
Bleeding control defined as all bleeding stopped.
Measured: From first administration of Obizur up to 180 days after the last administration of Obizur.
Number of infusions administered to achieve bleeding control, death or change in haemostatic treatment other than Obizur
Bleeding control defined as all bleeding stopped.
Measured: From first administration of Obizur up to 180 days after the last administration of Obizur.
Time to achieve bleeding control, death or change in haemostatic treatment other than Obizur
Bleeding control defined as all bleeding stopped.
Measured: From first administration of Obizur up to 180 days after the last administration of Obizur.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jun 2017
- Study start14 Dec 2016
- Primary completion30 Jul 2021
- Study completion30 Jul 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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
- Prospective
- Enrolment
- 50 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
- NCT03199794
- Sponsor's protocol ID
- 241501
- Other IDs
- EUPAS16055
- Status last verified
- Jul 2022
- First submitted
- 23 Jun 2017
- Last updated
- 21 Jul 2022
- 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.
Publications
Miesbach W, Curry N, Knobl P, Percy C, Santoro R, Schmaier AH, Trautmann-Grill K, Badejo K, Chen J, Nouri M, Oberai P, Klamroth R. Real-world use of recombinant porcine sequence factor VIII in the treatment of acquired hemophilia A: EU PASS. Ther Adv Hematol. 2024 Sep 2;15:20406207241260332. doi: 10.1177/20406207241260332. eCollection 2024.
PubMed 39228858
Study at a glance
From the registry record, last updated by the sponsor on 21 Jul 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective and Retrospective, Non-interventional Study to Evaluate the Safety and Effectiveness of Obizur in Real-life Practice
- ClinicalTrials.gov ID
- NCT03199794
- Acronym / sponsor ID
- 241501
- Conditions
- Acquired Hemophilia A
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 50 participants (actual)
- Lead sponsor
- Baxalta now part of ShireIndustry (pharma, biotech, devices)
- Collaborators
- Baxalta Innovations GmbH, now part of Shire
- Study start
- 14 Dec 2016 (actual)
- Primary completion
- 30 Jul 2021 (actual)
- Study completion
- 30 Jul 2021 (actual)
- First posted
- 27 Jun 2017 (actual)
- 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 study addresses the safety, utilisation and effectiveness of Obizur in the treatment of bleeding episodes in real-life clinical practice in Europe and the United States.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| OBIZUR participants | – | Biological: OBIZURParticipants previously treated with OBIZUR and continue to be treated with OBIZUR during the study. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- A participant with acquired hemophilia (AH) must be prescribed Obizur for the treatment of a bleeding episode by a physician, independent of and prior to the decision to enrol the participant in the study.
Key inclusion criteria (2 listed)
- Adult participant (or legal representative) is willing to provide informed consent
- Participant is being treated or was treated (treatment initiation within 30 days) with Obizur in routine clinical practice
Key exclusion criteria (2 listed)
- Participant has known anaphylactic reactions to the active substance, hamster protein or to any of the following excipients: Polysorbate 80; sodium chloride; calcium chloride dihydrate; sucrose; Tris Base; Tris HCl; Tri-sodium citrate dihydrate; sterilized water for injections
- Participant has participated in a 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 at study entry
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: Recombinant pFVIII, Porcine Sequence, rpFVIII, Antihemophilic Factor (Recombinant)Treating physician will determine treatment regimen and frequency of laboratory and clinical assessments according to routine clinical practice. | Biological | OBIZUR participants |
Primary outcome
Number of AEs and SAEs including seriousness, severity and outcome
Time frame: From first administration of Obizur up to 180 days after the last administration of Obizur.
AE - adverse event, SAE - serious adverse event.
Number of AESIs including seriousness, severity, relationship to therapy, outcome, and treatment discontinuation
Time frame: From first administration of Obizur up to 180 days after the last administration of Obizur.
Adverse Events of Special Interest (AESI) are as follows: hypersensitivity reactions, thromboembolic events and dose dispensing medication errors.
Number of thromboembolic events
Time frame: From first administration of Obizur up to 180 days after the last administration of Obizur.
Thromboembolic events include disseminated intravascular coagulation (DIC), venous thrombosis, pulmonary embolism, myocardial infarction and stroke.
Number of dose dispensing medication errors
Time frame: From first administration of Obizur up to 180 days after the last administration of Obizur.
Dose dispensing medication erros include miscalculation of dose while prescribing (calculation of correct dose based on the participant's weight) or administration of the incorrect dose.
7 secondary outcomes
Immunogenicity; newly recognized anti-pFVIII inhibitor or increase in titre of anti-pFVIII inhibitors and evolution of titre over time
Time frame: From first administration of Obizur up to 180 days after the last administration of Obizur.
Obizur treatment regimen, as available
Time frame: From first administration of Obizur up to 180 days after the last administration of Obizur.
Other medication administered for haemostatic control, as available
Time frame: From first administration of Obizur up to 180 days after the last administration of Obizur.
Overall effectiveness assessment for resolution of bleeding
Time frame: From first administration of Obizur up to 180 days after the last administration of Obizur.
Dose per infusion administered to achieve bleeding control, death or change in haemostatic treatment other than Obizur
Time frame: From first administration of Obizur up to 180 days after the last administration of Obizur.
Number of infusions administered to achieve bleeding control, death or change in haemostatic treatment other than Obizur
Time frame: From first administration of Obizur up to 180 days after the last administration of Obizur.
Time to achieve bleeding control, death or change in haemostatic treatment other than Obizur
Time frame: From first administration of Obizur up to 180 days after the last administration of Obizur.
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 21 Jul 2022.
- Derived from this study: PubMed 39228858Miesbach W, Curry N, Knobl P, Percy C, Santoro R, Schmaier AH, Trautmann-Grill K, Badejo K, Chen J, Nouri M, Oberai P, Klamroth R. Real-world use of recombinant porcine sequence factor VIII in the treatment of acquired hemophilia A: EU PASS. Ther Adv Hematol. 2024 Sep 2;15:20406207241260332. doi: 10.1177/20406207241260332. eCollection 2024.
- 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
26 sites in 7 countries: 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 26 sites in 7 countries.
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 2 inclusion and 2 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 21 Jul 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in Italy, United States, Germany, United Kingdom, Austria and 2 more countries. 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?
Number of AEs and SAEs including seriousness, severity and outcome (time frame: From first administration of Obizur up to 180 days after the last administration of Obizur.). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT03199794, which also lists 1 linked publication.
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 21 Jul 2022.