NCT03199794

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.

Trial Health

93
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
50

participants targeted

Target at P25-P50 for all trials

Timeline
Completed

Started Dec 2016

Longer than P75 for all trials

Geographic Reach
7 countries

26 active sites

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

December 14, 2016

Completed
6 months until next milestone

First Submitted

Initial submission to the registry

June 23, 2017

Completed
4 days until next milestone

First Posted

Study publicly available on registry

June 27, 2017

Completed
4.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 30, 2021

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

July 30, 2021

Completed
Last Updated

July 21, 2022

Status Verified

July 1, 2022

Enrollment Period

4.6 years

First QC Date

June 23, 2017

Last Update Submit

July 18, 2022

Conditions

Outcome Measures

Primary Outcomes (4)

  • Number of AEs and SAEs including seriousness, severity and outcome

    AE - adverse event, SAE - serious adverse event.

    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.

    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.

    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.

    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

    From first administration of Obizur up to 180 days after the last administration of Obizur.

  • Obizur treatment regimen, as available

    From first administration of Obizur up to 180 days after the last administration of Obizur.

  • Other medication administered for haemostatic control, as available

    From first administration of Obizur up to 180 days after the last administration of Obizur.

  • Overall effectiveness assessment for resolution of bleeding

    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

    From first administration of Obizur up to 180 days after the last administration of Obizur.

  • +2 more secondary outcomes

Study Arms (1)

OBIZUR participants

Participants previously treated with OBIZUR and continue to be treated with OBIZUR during the study.

Biological: OBIZUR

Interventions

OBIZURBIOLOGICAL

Treating physician will determine treatment regimen and frequency of laboratory and clinical assessments according to routine clinical practice.

Also known as: Recombinant pFVIII, Porcine Sequence, rpFVIII, Antihemophilic Factor (Recombinant)
OBIZUR participants

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)
Sampling MethodNon-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.

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (26)

University of Florida - Shands

Gainesville, Florida, 32608, United States

Location

Henry Ford Hospital

Detroit, Michigan, 48202, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Cleveland Clinic

Cleveland, Ohio, 44103, United States

Location

University Hospitals Cleveland Medical Center

Cleveland, Ohio, 44106, United States

Location

The University of Texas Health Science Center at Houston

Houston, Texas, 77030, United States

Location

AKH - Medizinische Universität Wien

Vienna, 1090, Austria

Location

CHU de Rouen - Hôpital Charles Nicolle

Rouen, 76000, France

Location

Vivantes Klinikum im Friedrichshain

Berlin, 10249, Germany

Location

Universitaetsklinikum Bonn

Bonn, 53127, Germany

Location

Universitaetsklinikum Carl Gustav Carus TU Dresden

Dresden, 01304, Germany

Location

Klinikum der Johann Wolfgang Goethe-Universitaet

Frankfurt, 60590, Germany

Location

Medizinische Hochschule Hannover

Hanover, 30625, Germany

Location

Azienda Ospedaliera Nazionale Santi Antonio e Biagio e Cesare Arrigo

Alessandria, 15100, Italy

Location

Azienda Ospedaliera Pugliese Ciaccio

Catanzaro, 88100, Italy

Location

Azienda Ospedaliero Universitaria Policlinico Paolo Giaccone

Palermo, 90127, Italy

Location

Fondazione IRCCS Policlinico San Matteo

Pavia, 27100, Italy

Location

Policlinico Universitario Agostino Gemelli

Roma, 00168, Italy

Location

Umberto I Pol. di Roma-Università di Roma La Sapienza

Rome, 00161, Italy

Location

Istituto Clinico Humanitas

Rozzano, 20089, Italy

Location

Radboud University Medical Centre

Nijmegen, 6525 GA, Netherlands

Location

University Hospital Birmingham

Birmingham, B15 2TH, United Kingdom

Location

St James's University Hospital

Leeds, LS9 7TF, United Kingdom

Location

Royal Free Hospital

London, NW3 2QG, United Kingdom

Location

Churchill Hospital

Oxford, OX3 7LE, United Kingdom

Location

Southampton General Hospital

Southampton, SO16 5YA, United Kingdom

Location

Related Publications (1)

  • 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.

Related Links

MeSH Terms

Conditions

Factor 8 deficiency, acquired

Interventions

Factor VIII

Intervention Hierarchy (Ancestors)

Blood Coagulation FactorsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsProtein PrecursorsBiological Factors

Study Officials

  • Study Director

    Shire

    STUDY DIRECTOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 23, 2017

First Posted

June 27, 2017

Study Start

December 14, 2016

Primary Completion

July 30, 2021

Study Completion

July 30, 2021

Last Updated

July 21, 2022

Record last verified: 2022-07

Data Sharing

IPD Sharing
Will share

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.

Shared Documents
STUDY PROTOCOL, SAP, ICF, CSR
Access Criteria
IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.
More information

Locations