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

NCT03695978 | Recruiting

• 1 other identifier: GENA-25
• Study Type: observational
• Target: 200
• Locations: 16 countries
• Sites: 51

Brief Summary

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.

Conditions

Haemophilia A

Outcome Measures

Primary Outcomes (2)

• Annualised rate of breakthrough bleeds to assess efficacy in prophylactic treatment

  Annualised rate of all bleeding events (BEs), including all spontaneous, traumatic and joint BEs

  100 exposure days

• Incidence of Adverse Drug Reactions (ADRs)

  Adverse drug reactions (ADRs) including hypersensitivity reactions will be recorded in by patients in treatment diaries, which will be reviewed at each Follow-up Visit.

  100 exposure days

Secondary Outcomes (2)

• Dosage of FVIII concentrates

  100 exposure days

• Overall assessment of the effectiveness of surgical prophylaxis by the treating physicians

  100 exposure days

Study Arms (3)

Nuwiq

All patients receiving Nuwiq (recombinant FVIII)

Octanate

All patients receiving Octanate (plasma derived FVIII)

Wilate

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

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

A total of 200 patients, either previously untreated patients (PUPs) or minimally treated patients (MTPs), are planned to be documented in the study

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Octapharma: lead

Study Sites (55)

University of Miami Miller School of Medicine

Miami, Florida, 33136, United States

ACTIVE NOT RECRUITING

Ochsner Clinic Foundation

New Orleans, Louisiana, 70121, United States

RECRUITING

Cure 4 The Kids Foundation Children's Specialty Center

Las Vegas, Nevada, 89109, United States

WITHDRAWN

CENIDOR

Salta, Argentina

ACTIVE NOT RECRUITING

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

Baku, AZ1012, Azerbaijan

WITHDRAWN

Republican Scientific Center for Radiation Medicine and Human Ecology

Homyel, 246040, Belarus

WITHDRAWN

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

Minsk, 223053, Belarus

RECRUITING

Hôpital Universitaire des Enfants Reine Fabiola

Brussels, 1020, Belgium

RECRUITING

Cliniques Universitaires Saint-Luc

Brussels, 1200, Belgium

RECRUITING

Cliniques Universitaires Saint-Luc

Brussels, 1200, Belgium

RECRUITING

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

Hamilton, Ontario, 1800NOCLOTS, Canada

WITHDRAWN

Department of Hematology Research Research Transition Facility

Edmonton, T6G 2V2, Canada

COMPLETED

University Hospital Centre Zagreb

Zagreb, Croatia

RECRUITING

Tallinn Childrenś Hospital Clinic of Paediatric Department of Haematology and Oncology

Tallinn, 13419, Estonia

WITHDRAWN

CENTRE HOSPITALIER UNIVERSITAIRE de BESANCON

Besançon, France

RECRUITING

CHRU Tours - Hôpital Trousseau

Chambray-lès-Tours, France

RECRUITING

Centre Régional de Traitement de l'hémophilie

Le Mans, France

RECRUITING

Hopital Simone Veil Groupement Hospitalier Eaubonne-Montmorency

Montmorency, 95160, France

RECRUITING

CHU Hotel Dieu, Centre de Traitment de l'Hemophilie

Nantes, 44093, France

RECRUITING

Hôspital Necker Enfants Malades

Paris, France

RECRUITING

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

Saint-Priest-en-Jarez, 42270, France

RECRUITING

Vivantes - Netzwerk für Gesundheit GmbH Klinikum im Friedrichshain

Berlin, 10249, Germany

ACTIVE NOT RECRUITING

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

Bonn, 53127, Germany

RECRUITING

Coagulation Research Centre GmbH

Duisburg, 47051, Germany

RECRUITING

University Hospital Essen

Essen, Germany

ACTIVE NOT RECRUITING

HZRM GmbH

Frankfurt, Germany

ACTIVE NOT RECRUITING

Heim Pál National Pediatric Institute Department of Oncology and Hematology

Budapest, 1089, Hungary

RECRUITING

University of Debrecen Department of Pediatrics

Debrecen, 4032, Hungary

RECRUITING

Ospedale Pediatrico "Giovani XXIII"

Bari, Italy

RECRUITING

Policlinico Sant'Orsola Malpighi

Bologna, 40138, Italy

RECRUITING

Ospedale San Giacomo

Castelfranco Veneto, 31033, Italy

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, 95123, Italy

RECRUITING

Azienda Ospedaliero Universitaria Careggi

Florence, 50134, Italy

RECRUITING

Ospedale Maggiore Policlinico

Milan, 920122, Italy

RECRUITING

Center for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research Hospital

Milan, Italy

RECRUITING

Centro Emofilia - AUO di Padova

Padua, Italy

WITHDRAWN

Policlinico Umberto I

Rome, 00161, Italy

RECRUITING

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

Torino, Italy

RECRUITING

Ospedale Regina Margherita

Turin, 10126, Italy

WITHDRAWN

Children's Hospital Affiliate of Vilnius Universtity Hospital Santaros Klinikos

Vilnius, LT-08406, Lithuania

RECRUITING

Hospital Infantil de Morelia Eva Sámano de López Mateos

Morelia, Mexico

RECRUITING

Hospital Universitario Dr. José Eleuterio Gonzalez S/N

Nuevo León, Mexico

RECRUITING

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

Nuevo León, Mexico

RECRUITING

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

Moscow, 119049, Russia

WITHDRAWN

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

Saint Petersburg, 191186, Russia

WITHDRAWN

Hospital General Universitario de Alicante Hematología y Hemoterapia

Alicante, 03010, Spain

RECRUITING

Hospital Universitari Vall D'Hebrón, Unitat d'Hemofilia

Barcelona, 02035, Spain

RECRUITING

HRU Malaga

Málaga, Spain

NOT YET RECRUITING

Universitary Hospital Son Espases (Hematology Service)

Palma de Mallorca, Spain

ACTIVE NOT RECRUITING

Hospital Universitario y Politécnico La Fe

Valencia, Spain

ACTIVE NOT RECRUITING

Istanbul University Faculty of Medicine

Fatih, Turkey (Türkiye)

WITHDRAWN

John Radcliffe Hospital, Oxford University Hospitals, NHS Foundation Trust

Headington, Oxford, OX3 9DU, United Kingdom

RECRUITING

Birmingham Children's Hospital NHS Foundation Trust

Birmingham, B46NH, United Kingdom

WITHDRAWN

Great Ormond Street Hospital for Children NHS Trust, Haemophilia Centre

London, WC1N 3JH, United Kingdom

RECRUITING

Newcastle Haemophilia Comprehensive Care Centre, Royal Victoria Infirmary

Newcastle upon Tyne, NE1 4LP, United Kingdom

RECRUITING

Related Publications (1)

• 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.

  PMID: 37180427 DERIVED

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Officials

• Sigurd Knaub, PhD

  Octapharma

  STUDY DIRECTOR

Central Study Contacts

Cristina Solomon, MD

CONTACT

+41 79 585 90 42; Cristina.Solomon@octapharma.com

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: OTHER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: October 3, 2018
• First Posted: October 4, 2018
• Study Start: February 13, 2018
• Primary Completion (Estimated): June 1, 2030
• Study Completion (Estimated): June 1, 2030
• Last Updated: November 10, 2025

Record last verified: 2025-11

Locations

LI (1); GB (4); ES (5); CR (1); ME (3); FR (7); TU (1); RU (2); HU (2); CA (2); BE (2); AR (1); US (3); DE (5); IT (11); AZ (1)