Efficacy, Safety & Utilisation of Nuwiq, Octanate and Wilate in Previously Untreated & Minimally Treated Haemophilia A Patients (Protect-NOW)

April 22, 2024 updated by: Octapharma

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.

Study Overview

Status

Recruiting

Conditions

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.

Study Type

Observational

Enrollment (Estimated)

200

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Azerbaijan
      • Baku, Azerbaijan, AZ1012
        • Withdrawn
        • Azerbaijan State Advanced Training Institute for Doctors Hematology Department Scientific-Research Center of Hemophilia
  • Belarus
      • Gomel, Belarus, 246040
        • Withdrawn
        • Republican Scientific Center for Radiation Medicine and Human Ecology
      • Minsk, Belarus, 223053
        • Recruiting
        • Republican Scientific and Practical Centre of Children Oncology, Hematology and Immunology
        • Contact:
  • Belgium
      • Brussels, Belgium, 1200
      • Brussels, Belgium, 1020
        • Recruiting
        • Hôpital Universitaire des Enfants Reine Fabiola
        • Contact:
  • Canada
      • Edmonton, Canada, T6G 2V2
        • Withdrawn
        • Department of Hematology Research Research Transition Facility
    • Ontario
      • Hamilton, Ontario, Canada, 1800NOCLOTS
        • Withdrawn
        • McMaster University, Division of Pediatric Hematology/Oncology Room 3N27
  • Estonia
      • Tallinn, Estonia, 13419
        • Withdrawn
        • Tallinn Childrenś Hospital Clinic of Paediatric Department of Haematology and Oncology
  • France
      • Le Mans, France
        • Recruiting
        • Centre Régional de Traitement de l'hémophilie
      • Montmorency, France, 95160
      • Nantes, France, 44093
      • Paris, France
        • Recruiting
        • Hôspital Necker Enfants Malades
      • Saint-Priest-en-Jarez, France, 42270
        • Withdrawn
        • CHRU Hopital Nord, Secretariat de pediatre
  • Germany
      • Berlin, Germany, 10249
        • Recruiting
        • Vivantes - Netzwerk für Gesundheit GmbH Klinikum im Friedrichshain
        • Contact:
      • Bonn, Germany, 53127
        • Recruiting
        • Institute of Experimental Haematology and Transfusion Medicine (IHT) University Clinic Bonn (AöR)
        • Contact:
      • Duisburg, Germany, 47051
  • Hungary
      • Budapest, Hungary, 1089
        • Recruiting
        • Heim Pál National Pediatric Institute Department of Oncology and Hematology
        • Principal Investigator:
          • Marianna Zombori
      • Debrecen, Hungary, 4032
        • Recruiting
        • University of Debrecen Department of Pediatrics
        • Principal Investigator:
          • Istvan Szegedi
  • Italy
      • Bari, Italy
        • Recruiting
        • Ospedale Pediatrico "Giovani XXIII"
      • Bologna, Italy, 40138
      • Castelfranco Veneto, Italy, 31033
      • Florence, Italy, 50134
      • Milan, Italy, 920122
      • Milan, Italy
        • Recruiting
        • Center for Thrombosis and Hemorrhagic Diseases, IRCCS Humanitas Research Hospital
      • Padova, Italy
        • Withdrawn
        • Centro Emofilia - AUO di Padova
      • Rome, Italy, 00161
      • Turin, Italy, 10126
        • Withdrawn
        • Ospedale Regina Margherita
  • Lithuania
      • Vilnius, Lithuania, LT-08406
  • Mexico
      • Morelia, Mexico
        • Recruiting
        • Hospital Infantil de Morelia Eva Sámano de López Mateos
      • Nuevo León, 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
  • Russian Federation
      • Moscow, Russian Federation, 119049
        • Withdrawn
        • Moscow State Government-financed Public Healthcare Institution "Morozovskaya Children Clinical Hospital of Moscow Healthcare Department"
      • Saint Petersburg, Russian Federation, 191186
        • Withdrawn
        • Saint-Petersburg State Budget Healthcare Institution "City Out-patient Clinical # 37"
  • Spain
      • Alicante, Spain, 03010
        • Recruiting
        • Hospital General Universitario de Alicante Hematología y Hemoterapia
        • Contact:
      • Barcelona, Spain, 02035
        • Recruiting
        • Hospital Universitari Vall D'Hebrón, Unitat d'Hemofilia
        • Contact:
  • Turkey
      • Fatih, Turkey
        • Withdrawn
        • Istanbul University Faculty of Medicine
  • United Kingdom
      • Birmingham, United Kingdom, B46NH
        • Withdrawn
        • Birmingham Children's Hospital NHS Foundation Trust
      • London, United Kingdom, WC1N 3JH
        • Recruiting
        • Great Ormond Street Hospital for Children NHS Trust, Haemophilia Centre
        • Contact:
      • Newcastle Upon Tyne, United Kingdom, NE1 4LP
        • Recruiting
        • Newcastle Haemophilia Comprehensive Care Centre, Royal Victoria Infirmary
        • Contact:
    • Oxford
  • United States
    • Nevada
      • Las Vegas, Nevada, United States, 89109
        • Withdrawn
        • Cure 4 The Kids Foundation Children's Specialty Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

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

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Other

Cohorts and Interventions

Group / Cohort
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 the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Annualised rate of breakthrough bleeds to assess efficacy in prophylactic treatment
Time Frame: 100 exposure days
Annualised rate of all bleeding events (BEs), including all spontaneous, traumatic and joint BEs
100 exposure days
Incidence of Adverse Drug Reactions (ADRs)
Time Frame: 100 exposure days
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
Dosage of FVIII concentrates
Time Frame: 100 exposure days
For each individual FVIII injection the dose will be recorded.
100 exposure days
Overall assessment of the effectiveness of surgical prophylaxis by the treating physicians
Time Frame: 100 exposure days
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

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Octapharma

Investigators

  • Study Director: Sigurd Knaub, PhD, Octapharma

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 13, 2018

Primary Completion (Estimated)

June 1, 2030

Study Completion (Estimated)

June 1, 2030

Study Registration Dates

First Submitted

October 3, 2018

First Submitted That Met QC Criteria

October 3, 2018

First Posted (Actual)

October 4, 2018

Study Record Updates

Last Update Posted (Actual)

April 23, 2024

Last Update Submitted That Met QC Criteria

April 22, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • GENA-25

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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