Clinical trial NCT03695978: Efficacy, Safety & Utilisation of Nuwiq, Octanate and Wilate in Previously Untreated & Minimally Treated Haemophilia A Patients
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
Study type: OBSERVATIONAL
Overall status: RECRUITING
Conditions: Haemophilia A
Lead sponsor: Octapharma
Sponsor class: INDUSTRY
First posted: 2018-10-04
Enrollment: 200
Sex: ALL
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: OTHER
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

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.

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.

Eligibility criteria: 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

Primary outcomes:
measure: Annualised rate of breakthrough bleeds to assess efficacy in prophylactic treatment
timeFrame: 100 exposure days
measure: Incidence of Adverse Drug Reactions (ADRs)
timeFrame: 100 exposure days
Secondary outcomes:
measure: Dosage of FVIII concentrates
timeFrame: 100 exposure days
measure: Overall assessment of the effectiveness of surgical prophylaxis by the treating physicians
timeFrame: 100 exposure days
Other outcomes:
Locations:
city: Las Vegas
country: United States
facility: Cure 4 The Kids Foundation Children's Specialty Center
state: Nevada
city: Baku
country: Azerbaijan
facility: Azerbaijan State Advanced Training Institute for Doctors Hematology Department Scientific-Research Center of Hemophilia
city: Gomel
country: Belarus
facility: Republican Scientific Center for Radiation Medicine and Human Ecology
city: Minsk
country: Belarus
facility: Republican Scientific and Practical Centre of Children Oncology, Hematology and Immunology
city: Brussels
country: Belgium
facility: Hôpital Universitaire des Enfants Reine Fabiola
city: Brussels
country: Belgium
facility: Cliniques Universitaires Saint-Luc
city: Brussels
country: Belgium
facility: Cliniques Universitaires Saint-Luc
city: Hamilton
country: Canada
facility: McMaster University, Division of Pediatric Hematology/Oncology Room 3N27
state: Ontario
city: Edmonton
country: Canada
facility: Department of Hematology Research Research Transition Facility
city: Croatia
country: Croatia
facility: University Hospital Centre Zagreb
city: Tallinn
country: Estonia
facility: Tallinn Childrenś Hospital Clinic of Paediatric Department of Haematology and Oncology
city: Besancon
country: France
facility: CENTRE HOSPITALIER UNIVERSITAIRE de BESANCON
city: Le Mans
country: France
facility: Centre Régional de Traitement de l'hémophilie
city: Montmorency
country: France
facility: Hopital Simone Veil Groupement Hospitalier Eaubonne-Montmorency
city: Nantes
country: France
facility: CHU Hotel Dieu, Centre de Traitment de l'Hemophilie
city: Paris
country: France
facility: Hôspital Necker Enfants Malades
city: Saint-Priest-en-Jarez
country: France
facility: CHRU Hopital Nord, Secretariat de pediatre, bat E niv +3
city: Berlin
country: Germany
facility: Vivantes - Netzwerk für Gesundheit GmbH Klinikum im Friedrichshain
city: Bonn
country: Germany
facility: Institute of Experimental Haematology and Transfusion Medicine (IHT) University Clinic Bonn (AöR)
city: Duisburg
country: Germany
facility: Coagulation Research Centre GmbH
city: Budapest
country: Hungary
facility: Heim Pál National Pediatric Institute Department of Oncology and Hematology
city: Debrecen
country: Hungary
facility: University of Debrecen Department of Pediatrics
city: Bari
country: Italy
facility: Ospedale Pediatrico "Giovani XXIII"
city: Bologna
country: Italy
facility: Policlinico Sant'Orsola Malpighi
city: Castelfranco Veneto
country: Italy
facility: Ospedale San Giacomo
city: Catania
country: Italy
facility: 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
city: Florence
country: Italy
facility: Azienda Ospedaliero Universitaria Careggi
city: Milan
country: Italy
facility: Ospedale Maggiore Policlinico
city: Milan
country: Italy
facility: Center for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research Hospital
city: Padova
country: Italy
facility: Centro Emofilia - AUO di Padova
city: Rome
country: Italy
facility: Policlinico Umberto I
city: Torino
country: Italy
facility: C.tro Emofilia A.O. Città della salute e della scienza di Torino, Ospedale Regina Margherita
city: Turin
country: Italy
facility: Ospedale Regina Margherita
city: Vilnius
country: Lithuania
facility: Children's Hospital Affiliate of Vilnius Universtity Hospital Santaros Klinikos
city: Morelia
country: Mexico
facility: Hospital Infantil de Morelia Eva Sámano de López Mateos
city: Nuevo León
country: Mexico
facility: Hospital Universitario Dr. José Eleuterio Gonzalez S/N
city: Nuevo León
country: Mexico
facility: SMO and Scientific Services S.A.P.P de C.V
city: Moscow
country: Russian Federation
facility: Moscow State Government-financed Public Healthcare Institution "Morozovskaya Children Clinical Hospital of Moscow Healthcare Department"
city: Saint Petersburg
country: Russian Federation
facility: Saint-Petersburg State Budget Healthcare Institution "City Out-patient Clinical # 37"
city: Alicante
country: Spain
facility: Hospital General Universitario de Alicante Hematología y Hemoterapia
city: Barcelona
country: Spain
facility: Hospital Universitari Vall D'Hebrón, Unitat d'Hemofilia
city: Fatih
country: Turkey
facility: Istanbul University Faculty of Medicine
city: Headington
country: United Kingdom
facility: John Radcliffe Hospital, Oxford University Hospitals, NHS Foundation Trust
state: Oxford
city: Birmingham
country: United Kingdom
facility: Birmingham Children's Hospital NHS Foundation Trust
city: London
country: United Kingdom
facility: Great Ormond Street Hospital for Children NHS Trust, Haemophilia Centre
city: Newcastle Upon Tyne
country: United Kingdom
facility: Newcastle Haemophilia Comprehensive Care Centre, Royal Victoria Infirmary
Study officials:
affiliation: Octapharma
name: Sigurd Knaub, PhD
role: STUDY_DIRECTOR
References: