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

Overview

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.

Primary outcome measures

• Annualised rate of breakthrough bleeds to assess efficacy in prophylactic treatment [Time frame: 100 exposure days]
• Incidence of Adverse Drug Reactions (ADRs) [Time frame: 100 exposure days]

Secondary outcome measures (2)

• Dosage of FVIII concentrates [Time frame: 100 exposure days]
• Overall assessment of the effectiveness of surgical prophylaxis by the treating physicians [Time frame: 100 exposure days]

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

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Observational model

Cohort

Study locations

Italy · 11 centers

• Ospedale Pediatrico "Giovani XXIII" — Bari
• Policlinico Sant'Orsola Malpighi — Bologna
• Ospedale San Giacomo — Castelfranco Veneto
• Azienda Ospedaliera-Universitaria Policlinico "Vittorio Emanuele", Centro di Riferimento R — Catania
• Azienda Ospedaliero Universitaria Careggi — Florence
• Ospedale Maggiore Policlinico — Milan
• Center for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research Hospital — Milan
• Centro Emofilia - AUO di Padova — Padova
• … and 3 more centers

France · 8 centers

• CENTRE HOSPITALIER UNIVERSITAIRE de BESANCON — Besançon
• The Centre Hospitalier Métropole Savoie — Chambéry
• CHRU Tours - Hôpital Trousseau — Chambray-lès-Tours
• Centre Régional de Traitement de l'hémophilie — Le Mans
• Hopital Simone Veil Groupement Hospitalier Eaubonne-Montmorency — Montmorency
• CHU Hotel Dieu, Centre de Traitment de l'Hemophilie — Nantes
• Hôspital Necker Enfants Malades — Paris
• CHRU Hopital Nord, Secretariat de pediatre, bat E niv +3 — Saint-Priest-en-Jarez

United States · 5 centers

• University of Miami Miller School of Medicine — Miami
• Ochsner Clinic Foundation — New Orleans
• St. Louis University , 221 North Grand Boulevard — St Louis
• Cure 4 The Kids Foundation Children's Specialty Center — Las Vegas
• The University of Texas Health Science Center at San Antonio — San Antonio

Germany · 5 centers

• Vivantes - Netzwerk für Gesundheit GmbH Klinikum im Friedrichshain — Berlin
• Institute of Experimental Haematology and Transfusion Medicine (IHT) University Clinic Bon — Bonn
• Coagulation Research Centre GmbH — Duisburg
• University Hospital Essen — Essen
• HZRM GmbH — Frankfurt

Spain · 5 centers

• Hospital General Universitario de Alicante Hematología y Hemoterapia — Alicante
• Hospital Universitari Vall D'Hebrón, Unitat d'Hemofilia — Barcelona
• HRU Malaga — Málaga
• Universitary Hospital Son Espases (Hematology Service) — Palma de Mallorca
• Hospital Universitario y Politécnico La Fe — Valencia

Mexico · 4 centers

• Hospital General de Occidente, Unidad de Hemato-Oncologia Pediatric, Av. Zoquipan 1050, Co — Jalisco
• Hospital Infantil de Morelia Eva Sámano de López Mateos — Morelia
• Hospital Universitario Dr. José Eleuterio Gonzalez S/N — Nuevo León
• SMO and Scientific Services S.A.P.P de C.V — Nuevo León

United Kingdom · 4 centers

• John Radcliffe Hospital, Oxford University Hospitals, NHS Foundation Trust — Headington
• Birmingham Children's Hospital NHS Foundation Trust — Birmingham
• Great Ormond Street Hospital for Children NHS Trust, Haemophilia Centre — London
• Newcastle Haemophilia Comprehensive Care Centre, Royal Victoria Infirmary — Newcastle upon Tyne

Belgium · 3 centers

• Hôpital Universitaire des Enfants Reine Fabiola — Brussels
• Cliniques Universitaires Saint-Luc — Brussels
• Cliniques Universitaires Saint-Luc — Brussels

Belarus · 2 centers

• Republican Scientific Center for Radiation Medicine and Human Ecology — Homyel
• Republican Scientific and Practical Centre of Children Oncology, Hematology and Immunology — Minsk

Canada · 2 centers

• McMaster University, Division of Pediatric Hematology/Oncology Room 3N27 — Hamilton
• Department of Hematology Research Research Transition Facility — Edmonton

Hungary · 2 centers

• Heim Pál National Pediatric Institute Department of Oncology and Hematology — Budapest
• University of Debrecen Department of Pediatrics — Debrecen

Russia · 2 centers

• Moscow State Government-financed Public Healthcare Institution "Morozovskaya Children Clin — Moscow
• Saint-Petersburg State Budget Healthcare Institution "City Out-patient Clinical # 37" — Saint Petersburg

Argentina · 1 center

• CENIDOR — Salta

Azerbaijan · 1 center

• Azerbaijan State Advanced Training Institute for Doctors Hematology Department Scientific- — Baku

Croatia · 1 center

• University Hospital Centre Zagreb — Zagreb

Estonia · 1 center

• Tallinn Childrenś Hospital Clinic of Paediatric Department of Haematology and Oncology — Tallinn

Lithuania · 1 center

• Children's Hospital Affiliate of Vilnius Universtity Hospital Santaros Klinikos — Vilnius

Turkey (Türkiye) · 1 center

• Istanbul University Faculty of Medicine — Fatih

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. PMID 37180427

Identifiers

NCT: NCT03695978 · GENA-25

Primary sources (government registries)

• ClinicalTrials.gov (US NIH)

View this study on ClinicalTrials.gov ↗