Clinical trial NCT03199794: Prospective and Retrospective, Non-interventional Study to Evaluate the Safety and Effectiveness of Obizur in Real-life Practice
Official title: Prospective and Retrospective, Non-interventional Study to Evaluate the Safety and Effectiveness of Obizur in Real-life Practice
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: 2017-06-27
Enrollment: 50
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
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.

Brief 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.


Eligibility criteria: Inclusion Criteria:

* 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

Exclusion Criteria:

* 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

Primary outcomes:
measure: Number of AEs and SAEs including seriousness, severity and outcome
timeFrame: From first administration of Obizur up to 180 days after the last administration of Obizur.
measure: Number of AESIs including seriousness, severity, relationship to therapy, outcome, and treatment discontinuation
timeFrame: From first administration of Obizur up to 180 days after the last administration of Obizur.
measure: Number of thromboembolic events
timeFrame: From first administration of Obizur up to 180 days after the last administration of Obizur.
measure: Number of dose dispensing medication errors
timeFrame: From first administration of Obizur up to 180 days after the last administration of Obizur.
Secondary outcomes:
measure: Immunogenicity; newly recognized anti-pFVIII inhibitor or increase in titre of anti-pFVIII inhibitors and evolution of titre over time
timeFrame: From first administration of Obizur up to 180 days after the last administration of Obizur.
measure: Obizur treatment regimen, as available
timeFrame: From first administration of Obizur up to 180 days after the last administration of Obizur.
measure: Other medication administered for haemostatic control, as available
timeFrame: From first administration of Obizur up to 180 days after the last administration of Obizur.
measure: Overall effectiveness assessment for resolution of bleeding
timeFrame: From first administration of Obizur up to 180 days after the last administration of Obizur.
measure: Dose per infusion administered to achieve bleeding control, death or change in haemostatic treatment other than Obizur
timeFrame: From first administration of Obizur up to 180 days after the last administration of Obizur.
measure: Number of infusions administered to achieve bleeding control, death or change in haemostatic treatment other than Obizur
timeFrame: From first administration of Obizur up to 180 days after the last administration of Obizur.
measure: Time to achieve bleeding control, death or change in haemostatic treatment other than Obizur
timeFrame: From first administration of Obizur up to 180 days after the last administration of Obizur.
Other outcomes:
Locations:
city: Gainesville
country: United States
facility: University of Florida - Shands
state: Florida
city: Detroit
country: United States
facility: Henry Ford Hospital
state: Michigan
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic
state: Ohio
city: Cleveland
country: United States
facility: University Hospitals Cleveland Medical Center
state: Ohio
city: Houston
country: United States
facility: The University of Texas Health Science Center at Houston
state: Texas
city: Vienna
country: Austria
facility: AKH - Medizinische Universität Wien
city: Rouen
country: France
facility: CHU de Rouen - Hôpital Charles Nicolle
city: Berlin
country: Germany
facility: Vivantes Klinikum im Friedrichshain
city: Bonn
country: Germany
facility: Universitaetsklinikum Bonn
city: Dresden
country: Germany
facility: Universitaetsklinikum Carl Gustav Carus TU Dresden
city: Frankfurt
country: Germany
facility: Klinikum der Johann Wolfgang Goethe-Universitaet
city: Hannover
country: Germany
facility: Medizinische Hochschule Hannover
city: Alessandria
country: Italy
facility: Azienda Ospedaliera Nazionale Santi Antonio e Biagio e Cesare Arrigo
city: Catanzaro
country: Italy
facility: Azienda Ospedaliera Pugliese Ciaccio
city: Palermo
country: Italy
facility: Azienda Ospedaliero Universitaria Policlinico Paolo Giaccone
city: Pavia
country: Italy
facility: Fondazione IRCCS Policlinico San Matteo
city: Roma
country: Italy
facility: Policlinico Universitario Agostino Gemelli
city: Rome
country: Italy
facility: Umberto I Pol. di Roma-Università di Roma La Sapienza
city: Rozzano
country: Italy
facility: Istituto Clinico Humanitas
city: Nijmegen
country: Netherlands
facility: Radboud University Medical Centre
city: Birmingham
country: United Kingdom
facility: University Hospital Birmingham
city: Leeds
country: United Kingdom
facility: St James's University Hospital
city: London
country: United Kingdom
facility: Royal Free Hospital
city: Oxford
country: United Kingdom
facility: Churchill Hospital
city: Southampton
country: United Kingdom
facility: Southampton General Hospital
Study officials:
affiliation: Shire
name: Study Director
role: STUDY_DIRECTOR
References: