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En observasjonsstudie av deltakere med type 3 von Willebrand sykdom på profylaktisk standard-of-care-behandling (WILL-EMI NIS)

5. november 2026 oppdatert av: Hoffmann-La Roche

En multisenter ikke-intervensjonell studie som evaluerer blødninger og helserelatert livskvalitet hos pasienter med type 3 von Willebrand sykdom på profylaktisk standardbehandlingsbehandling

This non-interventional study (NIS) is designed to collect information on the effectiveness and safety of treatment received in routine clinical care, as well as measure the health-related quality of life (HRQoL) of participants with Type 3 von Willebrand disease (VWD) receiving prophylactic therapy per local standard of care (SOC) over an observation period of at least 24 weeks.

Studieoversikt

Status

Aktiv, ikke rekrutterende

Forhold

Von Willebrands sykdom, type 3

Intervensjon / Behandling

Studietype

Observasjonsmessig

Registrering (Faktiske)

Kontakter og plasseringer

Denne delen inneholder kontaktinformasjon for de som utfører studien, og informasjon om hvor denne studien blir utført.

Studiesteder

Belgia
- - Leuven, Belgia, 3000
    - UZ Leuven Gasthuisberg
Canada
- Ontario
  - Toronto, Ontario, Canada, M5G 1X8
    - The Hospital for Sick Children
- Quebec
  - Montreal, Quebec, Canada, H4A 3J1
    - McGill University Health Center
Colombia
- - Medellín, Colombia
    - IPS SURA Industriales Medellín
Forente stater
- California
  - Sacramento, California, Forente stater, 95817
    - UC Davis
- Minnesota
  - Minneapolis, Minnesota, Forente stater, 55455
    - University of Minnesota Medical Center
- Missouri
  - St Louis, Missouri, Forente stater, 63110
    - Washington University School of Medicine
Frankrike
- - Lille, Frankrike, 59037
    - Hopital Claude Huriez - CHU Lille
  - Paris, Frankrike, 75015
    - Groupe Hospitalier Necker Enfants Malades
Italia
- Lazio
  - Rome, Lazio, Italia, 00161
    - Universita' Degli Studi La Sapienza-Ist.Di Ematologia
- Lombardy
  - Milan, Lombardy, Italia, 20122
    - IRCCS Ca' Granda Ospedale Maggiore Policlinico
- Tuscany
  - Florence, Tuscany, Italia, 50134
    - AOU Careggi
Japan
- - Fukuoka, Japan, 830-0011
    - Kurume University Hospital
  - Tokyo, Japan, 160-0023
    - Tokyo Medical University Hospital
Nederland
- - Rotterdam, Nederland, 3015 GD
    - Erasmus MC
Polen
- - Warsaw, Polen, 02-776
    - Instytut Hematologii i Transfuzjologii
Spania
- - Madrid, Spania, 28046
    - Hospital Universitario la Paz
  - Seville, Spania, 41013
    - Hospital Universtiario Virgen del Rocio
Storbritannia
- - London, Storbritannia, WC1N 3JH
    - Great Ormond Street Hospital
  - London, Storbritannia, SE1 7EH
    - St Thomas' Hospital
  - Manchester, Storbritannia, M13 9WL
    - Manchester Royal Infirmary
Sverige
- - Gothenburg, Sverige, S-413 45
    - Sahlgrenska Universitetssjukhuset
Tyskland
- - Bonn, Tyskland, 53127
    - Universitätsklinikum Bonn
  - Duisburg, Tyskland, 47051
    - Gerinnungszentrum Rhein-Ruhr;Gerinnungsambulanz
  - Frankfurt/M., Tyskland, 60590
    - Hämophiliezentrum Med. Klinik III/Institut für Transfusionsmedizin

Deltakelseskriterier

Forskere ser etter personer som passer til en bestemt beskrivelse, kalt kvalifikasjonskriterier. Noen eksempler på disse kriteriene er en persons generelle helsetilstand eller tidligere behandlinger.

Kvalifikasjonskriterier

Alder som er kvalifisert for studier

2 år og eldre (Barn, Voksen, Eldre voksen)

Tar imot friske frivillige

Nei

Kjønn som er kvalifisert for studier

Alle

Prøvetakingsmetode

Ikke-sannsynlighetsprøve

Studiepopulasjon

This study will observe approximately 40 participants with Type 3 VWD (aged 2 years and older) that are currently being treated with SOC prophylactic therapy.

Beskrivelse

Inclusion Criteria:

Confirmed diagnosis of Type 3 von Willebrand disease (VWD), based on medical records
Adequate hematologic, hepatic, and renal function
Documented and confirmed previous use of SOC prophylactic therapy for VWD (1-3 times weekly, as per prescribed dose) and anticipation to remain on the same regimen during the study
For participants of childbearing potential: agreement to remain abstinent or adhere to the contraception requirements

Exclusion Criteria:

Inherited or acquired bleeding disorder other than Congenital Type 3 VWD
History of gastrointestinal bleeding within 18 months prior to enrollment, or any previous diagnosis of angiodysplasia
History of intracranial hemorrhage
Previous or current treatment for thromboembolic disease or signs of thromboembolic disease
Other conditions (e.g., certain autoimmune diseases) that may increase risk of bleeding or thrombosis
History of clinically significant hypersensitivity associated with monoclonal antibody therapies or components of the emicizumab injection
Use of systemic immunomodulators (e.g., interferon) at enrollment or planned use during the study, with the exception of anti-retroviral therapy

Studieplan

Denne delen gir detaljer om studieplanen, inkludert hvordan studien er utformet og hva studien måler.

Hvordan er studiet utformet?

Designdetaljer

Observasjonsmodeller: COHORT
Tidsperspektiver: PROSPECTIVE

Antall grupper / kohorter

Kohorter og intervensjoner

Gruppe / Kohort	Intervensjon / Behandling
Participants with Type 3 VWD Treated with Prophylactic SOC Participants with Type 3 VWD, aged 2 years and above, who are currently on standard of care (SOC) prophylactic therapy are anticipated to remain on the chosen SOC regimen during the study. Dosing and treatment duration of any studied medicinal products collected as part of this study are at the discretion of the treating physician in accordance with local labeling or local treatment guidelines.	Legemiddel: Von Willebrand Factor Concentrates Used according to local labeling or local treatment guidelines. Legemiddel: Von Willebrand Factor Concentrates and Factor VIII Concentrates Used according to local labeling or local treatment guidelines. Legemiddel: Factor VIII Concentrates Used according to local labeling or local treatment guidelines. Legemiddel: Recombinant Activated Factor VII Used according to local labeling or local treatment guidelines. Legemiddel: Activated Prothrombin Complex Concentrate Used according to local labeling or local treatment guidelines.

Gruppe / Kohort

Intervensjon / Behandling

Participants with Type 3 VWD Treated with Prophylactic SOC

Participants with Type 3 VWD, aged 2 years and above, who are currently on standard of care (SOC) prophylactic therapy are anticipated to remain on the chosen SOC regimen during the study. Dosing and treatment duration of any studied medicinal products collected as part of this study are at the discretion of the treating physician in accordance with local labeling or local treatment guidelines.

Legemiddel: Von Willebrand Factor Concentrates