Efficacy, Safety & Utilisation of Nuwiq, Octanate and Wilate in Previously Untreated & Minimally Treated Haemophilia A Patients

NCT ID: NCT03695978

Last Updated: 2025-11-10

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

RECRUITING

Total Enrollment

200 participants

Study Classification

OBSERVATIONAL

Study Start Date

2018-02-13

Study Completion Date

2030-06-30

Brief Summary

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International, post-authorisation non-interventional study to evaluate real-life effectiveness, safety and utilisation patterns of Octapharma's FVIII concentrates Nuwiq, Octanate, and Wilate in previously untreated and minimally treated severe haemophilia A patients in routine clinical practice.

Detailed Description

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Octapharma's FVIII concentrates have been tested in clinical trials and registered for treatment of haemophilia A; however, as haemophilia A is a rare disease, the numbers of patients treated in studies so far are limited. For previously untreated patients (PUPs), who are typically young children, and for minimally treated patients (MTPs), who have been exposed to only minimal FVIII dosages, there is a general interest to increase the body of data on treatment effectiveness and safety, particularly related to inhibitor development. Also, specifically for PUPs, treatment algorithms are not standardized, e.g. with respect to utilisation, dosage, frequency or optimal start age of FVIII prophylaxis. Real world evidence derived from a non-interventional study (NIS) can describe product utilisation and demonstrate value over a product's life cycle and facilitate benefit-risk assessments. The purpose of this study is thus to evaluate product utilisation, effectiveness and safety, including inhibitor development information, in severe haemophilia A PUPs and MTPs, who have been prescribed Octapharma's FVIII concentrates.

Conditions

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Haemophilia A

Study Design

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Observational Model Type

COHORT

Study Time Perspective

OTHER

Study Groups

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Nuwiq

All patients receiving Nuwiq (recombinant FVIII)

No interventions assigned to this group

Octanate

All patients receiving Octanate (plasma derived FVIII)

No interventions assigned to this group

Wilate

All patients receiving Wilate (plasma derived FVIII/von Willebrand factor \[VWF\])

No interventions assigned to this group

Eligibility Criteria

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Inclusion Criteria

* Male and female patients of any age and ethnicity
* Severe haemophilia A (FVIII:C\<1%)
* Decision to prescribe Octapharma's FVIII concentrate before enrollment into the study
* Either
* No previous treatment with FVIII concentrates or other blood products containing FVIII (PUPs) OR
* Less than 5 Exposure Days (EDs) to FVIII concentrates or other blood products containing FVIII (MTPs), if
* data are available on all previous treatment, AND
* they did not develop an inhibitor at any time point, OR
* they developed an inhibitor during treatment with an Octapharma FVIII concentrate AND continue treatment with THIS Octapharma FVIII concentrate (in the presence or absence of emicizumab).
* Voluntarily given, fully informed written and signed consent obtained before any study-related data documentation is conducted (obtained from the patient's parent/legal guardian)

Exclusion Criteria

* Diagnosis with a coagulation disorder other than haemophilia A
* Concomitant treatment with any systemic immunosuppressive drug
* Participation in an interventional clinical trial during the time period evaluated
* Participation in another non-interventional study of Octapharma
Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Octapharma

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Sigurd Knaub, PhD

Role: STUDY_DIRECTOR

Octapharma

Locations

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University of Miami Miller School of Medicine

Miami, Florida, United States

Site Status ACTIVE_NOT_RECRUITING

Ochsner Clinic Foundation

New Orleans, Louisiana, United States

Site Status RECRUITING

Cure 4 The Kids Foundation Children's Specialty Center

Las Vegas, Nevada, United States

Site Status WITHDRAWN

CENIDOR

Salta, , Argentina

Site Status ACTIVE_NOT_RECRUITING

Azerbaijan State Advanced Training Institute for Doctors Hematology Department Scientific-Research Center of Hemophilia

Baku, , Azerbaijan

Site Status WITHDRAWN

Republican Scientific Center for Radiation Medicine and Human Ecology

Homyel, , Belarus

Site Status WITHDRAWN

Republican Scientific and Practical Centre of Children Oncology, Hematology and Immunology

Minsk, , Belarus

Site Status RECRUITING

Hôpital Universitaire des Enfants Reine Fabiola

Brussels, , Belgium

Site Status RECRUITING

Cliniques Universitaires Saint-Luc

Brussels, , Belgium

Site Status RECRUITING

Cliniques Universitaires Saint-Luc

Brussels, , Belgium

Site Status RECRUITING

McMaster University, Division of Pediatric Hematology/Oncology Room 3N27

Hamilton, Ontario, Canada

Site Status WITHDRAWN

Department of Hematology Research Research Transition Facility

Edmonton, , Canada

Site Status COMPLETED

University Hospital Centre Zagreb

Zagreb, , Croatia

Site Status RECRUITING

Tallinn Childrenś Hospital Clinic of Paediatric Department of Haematology and Oncology

Tallinn, , Estonia

Site Status WITHDRAWN

CENTRE HOSPITALIER UNIVERSITAIRE de BESANCON

Besançon, , France

Site Status RECRUITING

CHRU Tours - Hôpital Trousseau

Chambray-lès-Tours, , France

Site Status RECRUITING

Centre Régional de Traitement de l'hémophilie

Le Mans, , France

Site Status RECRUITING

Hopital Simone Veil Groupement Hospitalier Eaubonne-Montmorency

Montmorency, , France

Site Status RECRUITING

CHU Hotel Dieu, Centre de Traitment de l'Hemophilie

Nantes, , France

Site Status RECRUITING

Hôspital Necker Enfants Malades

Paris, , France

Site Status RECRUITING

CHRU Hopital Nord, Secretariat de pediatre, bat E niv +3

Saint-Priest-en-Jarez, , France

Site Status RECRUITING

Vivantes - Netzwerk für Gesundheit GmbH Klinikum im Friedrichshain

Berlin, , Germany

Site Status ACTIVE_NOT_RECRUITING

Institute of Experimental Haematology and Transfusion Medicine (IHT) University Clinic Bonn (AöR)

Bonn, , Germany

Site Status RECRUITING

Coagulation Research Centre GmbH

Duisburg, , Germany

Site Status RECRUITING

University Hospital Essen

Essen, , Germany

Site Status ACTIVE_NOT_RECRUITING

HZRM GmbH

Frankfurt, , Germany

Site Status ACTIVE_NOT_RECRUITING

Heim Pál National Pediatric Institute Department of Oncology and Hematology

Budapest, , Hungary

Site Status RECRUITING

University of Debrecen Department of Pediatrics

Debrecen, , Hungary

Site Status RECRUITING

Ospedale Pediatrico "Giovani XXIII"

Bari, , Italy

Site Status RECRUITING

Policlinico Sant'Orsola Malpighi

Bologna, , Italy

Site Status RECRUITING

Ospedale San Giacomo

Castelfranco Veneto, , Italy

Site Status COMPLETED

Azienda Ospedaliera-Universitaria Policlinico "Vittorio Emanuele", Centro di Riferimento Regionale per la Prevenzione, Diagnosi e Cura delle Malattie Rare della Coagulazione nel Bambino e neel'Adulto. U.O.C Ematologia con trapianto di Midollo Osseo

Catania, , Italy

Site Status RECRUITING

Azienda Ospedaliero Universitaria Careggi

Florence, , Italy

Site Status RECRUITING

Ospedale Maggiore Policlinico

Milan, , Italy

Site Status RECRUITING

Center for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research Hospital

Milan, , Italy

Site Status RECRUITING

Centro Emofilia - AUO di Padova

Padua, , Italy

Site Status WITHDRAWN

Policlinico Umberto I

Rome, , Italy

Site Status RECRUITING

C.tro Emofilia A.O. Città della salute e della scienza di Torino, Ospedale Regina Margherita

Torino, , Italy

Site Status RECRUITING

Ospedale Regina Margherita

Turin, , Italy

Site Status WITHDRAWN

Children's Hospital Affiliate of Vilnius Universtity Hospital Santaros Klinikos

Vilnius, , Lithuania

Site Status RECRUITING

Hospital Infantil de Morelia Eva Sámano de López Mateos

Morelia, , Mexico

Site Status RECRUITING

Hospital Universitario Dr. José Eleuterio Gonzalez S/N

Nuevo León, , Mexico

Site Status RECRUITING

SMO and Scientific Services S.A.P.P de C.V

Nuevo León, , Mexico

Site Status RECRUITING

Moscow State Government-financed Public Healthcare Institution "Morozovskaya Children Clinical Hospital of Moscow Healthcare Department"

Moscow, , Russia

Site Status WITHDRAWN

Saint-Petersburg State Budget Healthcare Institution "City Out-patient Clinical # 37"

Saint Petersburg, , Russia

Site Status WITHDRAWN

Hospital General Universitario de Alicante Hematología y Hemoterapia

Alicante, , Spain

Site Status RECRUITING

Hospital Universitari Vall D'Hebrón, Unitat d'Hemofilia

Barcelona, , Spain

Site Status RECRUITING

HRU Malaga

Málaga, , Spain

Site Status NOT_YET_RECRUITING

Universitary Hospital Son Espases (Hematology Service)

Palma de Mallorca, , Spain

Site Status ACTIVE_NOT_RECRUITING

Hospital Universitario y Politécnico La Fe

Valencia, , Spain

Site Status ACTIVE_NOT_RECRUITING

Istanbul University Faculty of Medicine

Fatih, , Turkey (Türkiye)

Site Status WITHDRAWN

John Radcliffe Hospital, Oxford University Hospitals, NHS Foundation Trust

Headington, Oxford, United Kingdom

Site Status RECRUITING

Birmingham Children's Hospital NHS Foundation Trust

Birmingham, , United Kingdom

Site Status WITHDRAWN

Great Ormond Street Hospital for Children NHS Trust, Haemophilia Centre

London, , United Kingdom

Site Status RECRUITING

Newcastle Haemophilia Comprehensive Care Centre, Royal Victoria Infirmary

Newcastle upon Tyne, , United Kingdom

Site Status RECRUITING

Countries

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United States Argentina Azerbaijan Belarus Belgium Canada Croatia Estonia France Germany Hungary Italy Lithuania Mexico Russia Spain Turkey (Türkiye) United Kingdom

Central Contacts

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Cristina Solomon, MD

Role: CONTACT

Phone: +41 79 585 90 42

Email: [email protected]

Facility Contacts

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Olga Alenjnikowa

Role: primary

Phu Quac Le

Role: primary

An Van Damme

Role: primary

Hassoun Abel

Role: primary

Marc Fouassier

Role: primary

Claire Berger

Role: primary

Johannes Oldenburg

Role: primary

Susan Halimeh

Role: primary

Lelia Validre

Role: primary

Giancarlo Castaman

Role: primary

Elena Santangostino

Role: primary

Antonio Chistolini

Role: primary

Role: backup

Sonata Saulyte Trakymiene

Role: primary

Role: backup

Pascual Marco Vera

Role: primary

Olga Benitez Hidalgo

Role: primary

Neha Bhatnagar

Role: primary

Georgina Hall

Role: backup

Raina Liesner

Role: primary

Tina Biss

Role: primary

References

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Oldenburg J, Halimeh S, Hall GW, Klamroth R, Vera PM, Jansen M, Mathias M. Design of a Real-World Observational Study in Previously Untreated and Minimally Treated Hemophilia A Patients: Protect-NOW. TH Open. 2023 May 10;7(2):e110-e116. doi: 10.1055/s-0043-1768464. eCollection 2023 Apr.

Reference Type DERIVED
PMID: 37180427 (View on PubMed)

Other Identifiers

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GENA-25

Identifier Type: -

Identifier Source: org_study_id