Haemophilia AProtect-NOWNCT03695978
Efficacy, Safety & Utilisation of Nuwiq, Octanate and Wilate in Previously Untreated & Minimally Treated Haemophilia A Patients
An observational study of Haemophilia A, run by Octapharma at 59 sites in 18 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Haemophilia A
- How many people
- 200 participants
- Where
- 59 sitesin 18 countries
- Study dates
- 13 Feb 2018to Jun 2030
- Phase
- Observational
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 Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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
Who may be able to join
- 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
Show all 10Show fewer
- 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)
Who cannot join
- 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
Trial locations and contacts
59 sites in 18 countries. Contact the study team or a site near you to ask about taking part.
59 sites in 18 countries
Italy11 sites, 8 recruiting
- Recruiting
Ospedale Pediatrico "Giovani XXIII"
Bari
- Recruiting
Policlinico Sant'Orsola Malpighi
Bologna, 40138
Lelia Validre, lelia.valdre@aosp.bo.it
- Completed
Ospedale San Giacomo
Castelfranco Veneto, 31033
- Recruiting
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
- Recruiting
Azienda Ospedaliero Universitaria Careggi
Florence, 50134
Giancarlo Castaman, giancarlo.castaman@unifi.it
- Recruiting
Ospedale Maggiore Policlinico
Milan, 920122
Elena Santangostino, elena.santagostino@unimi.it
- Recruiting
Center for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research Hospital
Milan
- Withdrawn
Centro Emofilia - AUO di Padova
Padova
- Recruiting
Policlinico Umberto I
Rome, 00161
Antonio Chistolini, antonio.chistolini@uniroma1.it
chistolini@bce.uniroma1.it
- Recruiting
C.tro Emofilia A.O. Città della salute e della scienza di Torino, Ospedale Regina Margherita
Torino
- Withdrawn
Ospedale Regina Margherita
Turin, 10126
France8 sites, 8 recruiting
- Recruiting
CENTRE HOSPITALIER UNIVERSITAIRE de BESANCON
Besançon
- Recruiting
The Centre Hospitalier Métropole Savoie
Chambéry, 31125 73011
- Recruiting
CHRU Tours - Hôpital Trousseau
Chambray-lès-Tours
- Recruiting
Centre Régional de Traitement de l'hémophilie
Le Mans
- Recruiting
Hopital Simone Veil Groupement Hospitalier Eaubonne-Montmorency
Montmorency, 95160
Hassoun Abel, abel.hassoun@ch-simoneveil.fr
- Recruiting
CHU Hotel Dieu, Centre de Traitment de l'Hemophilie
Nantes, 44093
Marc Fouassier, marc.fouassier@chu-nantes.fr
- Recruiting
Hôspital Necker Enfants Malades
Paris
- Recruiting
CHRU Hopital Nord, Secretariat de pediatre, bat E niv +3
Saint-Priest-en-Jarez, 42270
Claire Berger, claire.berger@chu-st-etienne.fr
United States5 sites, 3 recruiting
- Withdrawn
Cure 4 The Kids Foundation Children's Specialty Center
Las Vegas, Nevada, 89109
- Recruiting
University of Miami Miller School of Medicine
Miami, Florida, 33136
- Active, not recruiting
Ochsner Clinic Foundation
New Orleans, Louisiana, 70121
- Not yet recruiting
The University of Texas Health Science Center at San Antonio
San Antonio, Texas, 78712
- Not yet recruiting
St. Louis University , 221 North Grand Boulevard
St Louis, Missouri, 63103
Germany5 sites, 2 recruiting
- Active, not recruiting
Vivantes - Netzwerk für Gesundheit GmbH Klinikum im Friedrichshain
Berlin, 10249
- Recruiting
Institute of Experimental Haematology and Transfusion Medicine (IHT) University Clinic Bonn (AöR)
Bonn, 53127
Johannes Oldenburg, Johannes.Oldenburg@ukb.uni-bonn.de
- Recruiting
Coagulation Research Centre GmbH
Duisburg, 47051
Susan Halimeh, susan.halimeh@gzrr.de
- Active, not recruiting
University Hospital Essen
Essen
- Active, not recruiting
HZRM GmbH
Frankfurt
Spain5 sites, 3 recruiting
- Recruiting
Hospital General Universitario de Alicante Hematología y Hemoterapia
Alicante, 03010
Pascual Marco Vera, marco.pas@gva.es
- Recruiting
Hospital Universitari Vall D'Hebrón, Unitat d'Hemofilia
Barcelona, 02035
Olga Benitez Hidalgo, 25600173t@vhebron.net
- Not yet recruiting
HRU Malaga
Málaga
- Active, not recruiting
Universitary Hospital Son Espases (Hematology Service)
Palma de Mallorca
- Active, not recruiting
Hospital Universitario y Politécnico La Fe
Valencia
Mexico4 sites, 4 recruiting
- Recruiting
Hospital General de Occidente, Unidad de Hemato-Oncologia Pediatric, Av. Zoquipan 1050, Col Seattle, , Col Seattle
Jalisco
- Not yet recruiting
Hospital Infantil de Morelia Eva Sámano de López Mateos
Morelia
- Not yet recruiting
Hospital Universitario Dr. José Eleuterio Gonzalez S/N
Nuevo León
- Not yet recruiting
SMO and Scientific Services S.A.P.P de C.V
Nuevo León
United Kingdom4 sites, 3 recruiting
- Withdrawn
Birmingham Children's Hospital NHS Foundation Trust
Birmingham, B46NH
- Recruiting
John Radcliffe Hospital, Oxford University Hospitals, NHS Foundation Trust
Headington, Oxford, OX3 9DU
Neha Bhatnagar, Neha.Bhatnagar@ouh.nhs.uk
Georgina Hall, georgina.hall@peadiatrics.ox.ac.uk
- Recruiting
Great Ormond Street Hospital for Children NHS Trust, Haemophilia Centre
London, WC1N 3JH
Raina Liesner, Ri.Liesner@gosh.nhs.uk
- Recruiting
Newcastle Haemophilia Comprehensive Care Centre, Royal Victoria Infirmary
Newcastle upon Tyne, NE1 4LP
Tina Biss, Tina.Biss@nuth.nhs.uk
Belgium3 sites, 3 recruiting
- Recruiting
Hôpital Universitaire des Enfants Reine Fabiola
Brussels, 1020
Phu Quac Le, Phuquoc.LE@huderf.be
- Recruiting
Cliniques Universitaires Saint-Luc
Brussels, 1200
An Van Damme, an.vandamme@uclouvain.be
- Recruiting
Cliniques Universitaires Saint-Luc
Brussels, 1200
Belarus2 sites, 1 recruiting
- Withdrawn
Republican Scientific Center for Radiation Medicine and Human Ecology
Homyel, 246040
- Recruiting
Republican Scientific and Practical Centre of Children Oncology, Hematology and Immunology
Minsk, 223053
Olga Alenjnikowa, aleinikova2004@mail.ru
Canada2 sites
- Completed
Department of Hematology Research Research Transition Facility
Edmonton, T6G 2V2
- Withdrawn
McMaster University, Division of Pediatric Hematology/Oncology Room 3N27
Hamilton, Ontario, 1800NOCLOTS
Hungary2 sites, 2 recruiting
- Recruiting
Heim Pál National Pediatric Institute Department of Oncology and Hematology
Budapest, 1089
Marianna Zombori
- Recruiting
University of Debrecen Department of Pediatrics
Debrecen, 4032
Istvan Szegedi
Russia2 sites
- Withdrawn
Moscow State Government-financed Public Healthcare Institution "Morozovskaya Children Clinical Hospital of Moscow Healthcare Department"
Moscow, 119049
- Withdrawn
Saint-Petersburg State Budget Healthcare Institution "City Out-patient Clinical # 37"
Saint Petersburg, 191186
Azerbaijan1 site
- Withdrawn
Azerbaijan State Advanced Training Institute for Doctors Hematology Department Scientific-Research Center of Hemophilia
Baku, AZ1012
Croatia1 site, 1 recruiting
- Recruiting
University Hospital Centre Zagreb
Zagreb
Estonia1 site
- Withdrawn
Tallinn Childrenś Hospital Clinic of Paediatric Department of Haematology and Oncology
Tallinn, 13419
Lithuania1 site, 1 recruiting
- Recruiting
Children's Hospital Affiliate of Vilnius Universtity Hospital Santaros Klinikos
Vilnius, LT-08406
Sonata Saulyte Trakymiene, sonata.trakymiene@vuvl.lt
sonatrak@gmail.com
Turkey (Türkiye)1 site
- Withdrawn
Istanbul University Faculty of Medicine
Fatih
About this study
Official title: Practical Utilisation of Octapharma FVIII Concentrates in Previously Untreated & Minimally Treated Haemophilia A Patients Entering Routine Clinical Treatment With Nuwiq, Octanate or Wilate - Efficacy & Safety Observational Study-Protect-NOW
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.
Read the detailed description
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
- Haemophilia A
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| 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]) |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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.
Measured: 100 exposure days
Secondary outcomes (2)
Dosage of FVIII concentrates
For each individual FVIII injection the dose will be recorded.
Measured: 100 exposure days
Overall assessment of the effectiveness of surgical prophylaxis by the treating physicians
At the end of the postoperative period, treating physicians will assess the effectiveness of surgical prophylaxis using a scale including the four items: 'excellent,' 'good,' moderate,' and 'none'.
Measured: 100 exposure days
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Oct 2018
- Study start13 Feb 2018
- Primary completionJun 2030 (estimated)
- Study completionJun 2030 (estimated)
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 Haemophilia 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
- Other
- Enrolment
- 200 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Octapharma
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Sigurd Knaub, PhD, Study director, Octapharma
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03695978
- Sponsor's protocol ID
- GENA-25
- Acronym
- Protect-NOW
- Status last verified
- Nov 2025
- First submitted
- 3 Oct 2018
- Last updated
- 2 Jun 2026
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Publications
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.
PubMed 37180427