- ICH GCP
- Amerikanska kliniska prövningsregistret
- Klinisk prövning NCT04279717
Latin-American Von Willebrand Disease Registry
Establish a Latin-American network of centers and professionals with the aim of:
- To register VWD patients in retrospective/prospective study, using a database, available online, common to all
- To register the bleeding history, the treatment and the events of VWD patients in the region
- To investigate the influence of VWD on quality of life
Studieöversikt
Detaljerad beskrivning
von Willebrand disease (VWD) is the most common autosomal bleeding disorder, mostly inherited as dominant trait. VWD is due to deficiency/abnormality of von Willebrand factor (VWF). The prevalence of VWD is unknown, but estimated as 0.1% to 1% of the general population. Although the autosomal inheritance pattern would suggest an equal distribution of male and female patients, the disease is diagnosed in more females because of female-specific hemostatic challenges: menses, ovulation, pregnancy and childbirth. Diagnosis of VWD is made by assessing personal and family history of bleeding, physical examination and completed with specific laboratory tests.
There is limited information on the epidemiology of VWD in developing countries. Some countries in Latin America have registries of severe disease that, although it is the rarest form, carries the highest costs for regional health systems. So that the prevalence of clinical symptoms and laboratory features of the disease as well as the management of the disease in Latin America is unknown.
The present project aims to establish a network of centers and professionals with the objective to register and investigate all patients with VWD in Latin America, using a database available online common to all, to gain understanding about phenotype, genotype and management of VWD in the region.
Studietyp
Inskrivning (Förväntat)
Kontakter och platser
Studiekontakt
- Namn: Analia Sanchez Luceros, PhD, MD
- Telefonnummer: +5491152203235
- E-post: sanchezluceros@gmail.com
Studera Kontakt Backup
- Namn: Analia Kinen
- E-post: analiakinen@hotmail.com
Deltagandekriterier
Urvalskriterier
Åldrar som är berättigade till studier
- Barn
- Vuxen
- Äldre vuxen
Tar emot friska volontärer
Kön som är behöriga för studier
Testmetod
Studera befolkning
Beskrivning
Inclusion Criteria:
- Historically lowest VWF:Ag and/or VWF:RCo and/or VWF:CB < 0.50 IU/ml and/or FVIII:C < 0.50 IU/ml
- All types of VWD
- All ages
Exclusion Criteria:
- Patient without consent to participate
Studieplan
Hur är studien utformad?
Designdetaljer
- Observationsmodeller: Kohort
- Tidsperspektiv: Tvärsnitt
Kohorter och interventioner
Grupp / Kohort |
Intervention / Behandling |
---|---|
Subjects with von Willbrand Disease Acquired
|
Inga insatser planerade: behandling av patienter efter bedömning av den behandlande/ansvariga läkaren
|
Subjects with von Willbrand Disease Congenital
|
Inga insatser planerade: behandling av patienter efter bedömning av den behandlande/ansvariga läkaren
|
Vad mäter studien?
Primära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
---|---|---|
Register of VWD patients in Latin America
Tidsram: assessed up to 33 months
|
Clinical presentation in hereditary/acquired VWD.
Phenotype and genetic diagnosis.
|
assessed up to 33 months
|
Registration of the bleeding history
Tidsram: From date of selection until the date registration, assessed up to 33 months.
|
Bleeding history is an essential component in the diagnosis of von Willebrand disease (VWD).
ISTH Bleeding Assessment Tool (ISTH-BAT) is used to assist the diagnosis.
|
From date of selection until the date registration, assessed up to 33 months.
|
Response to Treatment: Follow up of FVIII, VWF:Ag and VWF:RCo
Tidsram: Until the end of the registry, an average of 33 months.
|
The aim of therapy is to correct the dual hemostatic defect, due to defective platelet adhesion-aggregation and abnormal coagulation due to Factor VIII (FVIII) deficiency.
The choice of treatment depends on a number of factors, including the severity of the bleed, the procedure planned, the subtype and severity of the disease and the age and morbidity of the patient.
The evaluation of the response to the treatment is going to be through the measure of FVIII, vWF Antigen (VWF:Ag) and vWF ristocetin cofactor (vWF:RCo).
|
Until the end of the registry, an average of 33 months.
|
Adverse Events: Number of patients with bleeding events
Tidsram: until the end of the registry, an average of 33 months.
|
Bleeding disorders and their treatment impact on patients, especially in women, can affect the everyday life of patients and their families.
Measure of number of bleeding events, laboratory results such as Sodium.
|
until the end of the registry, an average of 33 months.
|
Sekundära resultatmått
Resultatmått |
Åtgärdsbeskrivning |
Tidsram |
---|---|---|
Pregnancy outcome: Follow up of FVIII, VWF:Ag and VWF:RCo
Tidsram: Through study completion, an average of 2 years
|
For many women with VWD, pregnancy is a time of few bleeding problems.
Women with Type 3 von Willebrand disease seem to have more frequent miscarriages, especially during the first trimester.
The evaluation of the response to the treatment is going to be through the measure of FVIII, vWF Antigen (VWF:Ag) and vWF ristocetin cofactor (vWF:RCo).
|
Through study completion, an average of 2 years
|
Samarbetspartners och utredare
Sponsor
Studieavstämningsdatum
Studera stora datum
Studiestart (Förväntat)
Primärt slutförande (Förväntat)
Avslutad studie (Förväntat)
Studieregistreringsdatum
Först inskickad
Först inskickad som uppfyllde QC-kriterierna
Första postat (Faktisk)
Uppdateringar av studier
Senaste uppdatering publicerad (Faktisk)
Senaste inskickade uppdateringen som uppfyllde QC-kriterierna
Senast verifierad
Mer information
Termer relaterade till denna studie
Ytterligare relevanta MeSH-villkor
Andra studie-ID-nummer
- Protocol_3081
Plan för individuella deltagardata (IPD)
Planerar du att dela individuella deltagardata (IPD)?
IPD-delning som stöder informationstyp
- Studieprotokoll
- Klinisk studierapport (CSR)
Läkemedels- och apparatinformation, studiedokument
Studerar en amerikansk FDA-reglerad läkemedelsprodukt
Studerar en amerikansk FDA-reglerad produktprodukt
Denna information hämtades direkt från webbplatsen clinicaltrials.gov utan några ändringar. Om du har några önskemål om att ändra, ta bort eller uppdatera dina studieuppgifter, vänligen kontakta register@clinicaltrials.gov. Så snart en ändring har implementerats på clinicaltrials.gov, kommer denna att uppdateras automatiskt även på vår webbplats .
Kliniska prövningar på Von Willebrands sjukdom
-
St. James's Hospital, IrelandOkänd
-
Fondazione IRCCS Ca' Granda, Ospedale Maggiore...RekryteringLåg Von Willebrand-faktorItalien
-
Baylor College of MedicineShireAktiv, inte rekryterande
-
Baxalta now part of ShireAvslutadVon Willebrands sjukdomFörenta staterna, Tyskland, Storbritannien, Italien, Österrike, Kanada
-
University Hospital, CaenRekryteringVon Willebrands sjukdom, typ 2BFrankrike
-
Fondazione Angelo Bianchi BonomiSintesi Research SrlAktiv, inte rekryterandeTyp 3 Von Willebrands sjukdomFinland, Frankrike, Tyskland, Ungern, Iran, Islamiska republiken, Italien, Nederländerna, Spanien, Sverige, Storbritannien
-
Archemix Corp.Indragen
-
OctapharmaRekryteringVWD - Von Willebrands sjukdomFrankrike
-
TakedaTillgängligtVon Willebrands sjukdom (VWD)
-
Tirol Kiniken GmbHLFB BIOMEDICAMENTSOkänd
Kliniska prövningar på Observation
-
Masonic Cancer Center, University of MinnesotaAvslutadAkut leukemi | Kemoterapi-inducerad tarmbarriärskadaFörenta staterna
-
Centre Hospitalier Régional d'OrléansAvslutad
-
Dokuz Eylul UniversityIzmir Katip Celebi UniversityAvslutad
-
Universidad Autonoma de MadridOkänd
-
University of BergenEuropean Society of Intensive Care MedicineAvslutadKritisk sjukdom | Gammal ålder; Svaghet | ÖverlevnadNorge
-
Iain BressendorffDitte Hansen MD PhD; Casper Rydahl MD; Anders Nordholm MD PhD; Eva Gravesen...Har inte rekryterat ännuNefrotiskt syndrom | Minimal förändringssjukdom | Membranös nefropati | Primär fokal segmentell glomeruloskleros
-
MultiCare Health System Research InstituteRekrytering
-
Royal Marsden NHS Foundation TrustHar inte rekryterat ännu
-
Second Affiliated Hospital, School of Medicine,...Jinhua People's Hospital; Jinhua Municipal Central Hospital; Yiwu Central... och andra samarbetspartnersRekrytering
-
Mohamed ElsawyRekrytering