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

Haemophilia A Clinical Trial

— Protect-NOW  

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT03695978
• Other study ID #: GENA-25
• Status: Recruiting
• Start date: February 13, 2018
• Est. completion date: June 2030

Study information

• Verified date: April 2024
• Source: Octapharma
• Contact: Sigurd Knaub, PhD
• Phone: +41 554512141
• Email: Sigurd.Knaub[@]octapharma.ch
• Is FDA regulated: No
• Study type: Observational

Clinical 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 200
• Est. completion date: June 2030
• Est. primary completion date: June 2030
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Haemophilia A
• Hemophilia A

Locations

Country	Name	City	State
Azerbaijan	Azerbaijan State Advanced Training Institute for Doctors Hematology Department Scientific-Research Center of Hemophilia	Baku
Belarus	Republican Scientific Center for Radiation Medicine and Human Ecology	Gomel
Belarus	Republican Scientific and Practical Centre of Children Oncology, Hematology and Immunology	Minsk
Belgium	Cliniques Universitaires Saint-Luc	Brussels
Belgium	Hôpital Universitaire des Enfants Reine Fabiola	Brussels
Canada	Department of Hematology Research Research Transition Facility	Edmonton
Canada	McMaster University, Division of Pediatric Hematology/Oncology Room 3N27	Hamilton	Ontario
Estonia	Tallinn Childrens Hospital Clinic of Paediatric Department of Haematology and Oncology	Tallinn
France	Centre Régional de Traitement de l'hémophilie	Le Mans
France	Hopital Simone Veil Groupement Hospitalier Eaubonne-Montmorency	Montmorency
France	CHU Hotel Dieu, Centre de Traitment de l'Hemophilie	Nantes
France	Hôspital Necker Enfants Malades	Paris
France	CHRU Hopital Nord, Secretariat de pediatre	Saint-Priest-en-Jarez
Germany	Vivantes - Netzwerk für Gesundheit GmbH Klinikum im Friedrichshain	Berlin
Germany	Institute of Experimental Haematology and Transfusion Medicine (IHT) University Clinic Bonn (AöR)	Bonn
Germany	Coagulation Research Centre GmbH	Duisburg
Hungary	Heim Pál National Pediatric Institute Department of Oncology and Hematology	Budapest
Hungary	University of Debrecen Department of Pediatrics	Debrecen
Italy	Ospedale Pediatrico "Giovani XXIII"	Bari
Italy	Policlinico Sant'Orsola Malpighi	Bologna
Italy	Ospedale San Giacomo	Castelfranco Veneto
Italy	Azienda Ospedaliero Universitaria Careggi	Florence
Italy	Center for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research Hospital	Milan
Italy	Ospedale Maggiore Policlinico	Milan
Italy	Centro Emofilia - AUO di Padova	Padova
Italy	Policlinico Umberto I	Rome
Italy	Ospedale Regina Margherita	Turin
Lithuania	Children's Hospital Affiliate of Vilnius Universtity Hospital Santaros Klinikos	Vilnius
Mexico	Hospital Infantil de Morelia Eva Sámano de López Mateos	Morelia
Mexico	Hospital Universitario Dr. José Eleuterio Gonzalez S/N	Nuevo León
Mexico	SMO and Scientific Services S.A.P.P de C.V	Nuevo León
Russian Federation	Moscow State Government-financed Public Healthcare Institution "Morozovskaya Children Clinical Hospital of Moscow Healthcare Department"	Moscow
Russian Federation	Saint-Petersburg State Budget Healthcare Institution "City Out-patient Clinical # 37"	Saint Petersburg
Spain	Hospital General Universitario de Alicante Hematología y Hemoterapia	Alicante
Spain	Hospital Universitari Vall D'Hebrón, Unitat d'Hemofilia	Barcelona
Turkey	Istanbul University Faculty of Medicine	Fatih
United Kingdom	Birmingham Children's Hospital NHS Foundation Trust	Birmingham
United Kingdom	John Radcliffe Hospital, Oxford University Hospitals, NHS Foundation Trust	Headington	Oxford
United Kingdom	Great Ormond Street Hospital for Children NHS Trust, Haemophilia Centre	London
United Kingdom	Newcastle Haemophilia Comprehensive Care Centre, Royal Victoria Infirmary	Newcastle Upon Tyne
United States	Cure 4 The Kids Foundation Children's Specialty Center	Las Vegas	Nevada

Sponsors (1)

Lead Sponsor	Collaborator
Octapharma

Countries where clinical trial is conducted

United States,  Azerbaijan,  Belarus,  Belgium,  Canada,  Estonia,  France,  Germany,  Hungary,  Italy,  Lithuania,  Mexico,  Russian Federation,  Spain,  Turkey,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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
Primary	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	Dosage of FVIII concentrates	For each individual FVIII injection the dose will be recorded.	100 exposure days
Secondary	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'.	100 exposure days