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Clinical Trial Details — Status: Not yet recruiting

Administrative data

NCT number NCT04279717
Other study ID # Protocol_3081
Secondary ID
Status Not yet recruiting
Phase
First received
Last updated
Start date February 2020
Est. completion date December 2022

Study information

Verified date February 2020
Source Academia Nacional de Medicina
Contact Analia Sanchez Luceros, PhD, MD
Phone +5491152203235
Email sanchezluceros@gmail.com
Is FDA regulated No
Health authority
Study type Observational [Patient Registry]

Clinical Trial Summary

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


Description:

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.


Recruitment information / eligibility

Status Not yet recruiting
Enrollment 500
Est. completion date December 2022
Est. primary completion date December 2022
Accepts healthy volunteers
Gender All
Age group N/A and older
Eligibility 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

Study Design


Related Conditions & MeSH terms


Intervention

Other:
Observation
No interventions planned: treatment of patients at the discretion of the treating/responsible physician
Observation
No interventions planned: treatment of patients at the discretion of the treating/responsible physician

Locations

Country Name City State
n/a

Sponsors (1)

Lead Sponsor Collaborator
Academia Nacional de Medicina

Outcome

Type Measure Description Time frame Safety issue
Primary Register of VWD patients in Latin America Clinical presentation in hereditary/acquired VWD. Phenotype and genetic diagnosis. assessed up to 33 months
Primary Registration of the bleeding history 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.
Primary Response to Treatment: Follow up of FVIII, VWF:Ag and VWF:RCo 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.
Primary Adverse Events: Number of patients with bleeding events 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.
Secondary Pregnancy outcome: Follow up of FVIII, VWF:Ag and VWF:RCo 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
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