Von Willebrand DiseaseVWDNCT01589848
Study on Von Willebrand Disease and Hemophilia in Cuenca, Ecuador
An observational study of Von Willebrand Disease and Hemophilia A, run by Universidad del Azuay at 2 sites in Ecuador.
Trial summary
1. Hypothesis a. There are patients with von Willebrand Disease in Cuenca. 2. Primary question a. How many women referred with a history of bleeding may have von Willebrand disease? 3. Secondary 1. Associations between the bleeding score and initial laboratory studies 2. Read more about this study
- Who is it for?
- Ages 18 to 50 years, women only
- Conditions the study is for
- Von Willebrand Disease, Hemophilia A, Hemophilia B
- How many people
- 81 participants
- Where
- 2 sitesEcuador
- Study dates
- Mar 2013to Jun 2013
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 50 years
- Women only
- No healthy volunteers
- Adult
Study population: Target population: all women from Cuenca district with a hemorrhage history. Accessible population: Those women referred from professionals with an initial hemorrhage history, who comply with the inclusion criteria and who sign the informed consert form. Sample population: First comers referred with a hemorrhage history until fulfilling the projected sample size. This non-probabilistic purposive sample will be used because it has been reported that it could represent the target population. *2010 Ecuadorian census: 131.163 women ages 18 to 50 years, at Cuenca district.
Who may be able to join
- Subjects living at urban and rural parishes from Cuenca district.
- Subjects referred from professionals of public and private health services, who have filled the initial hemorrhage survey.
- Must be referred women who sign the informed consent form.
- Patients who answer the standardized bleeding score questionnaire (BS).
- Patients who answer the quality of life questionnaire (SF-12).
- Patients who answer the socio-economic survey.
- Patients who accept to provide a venous blood sample for the initial laboratory tests.
Who cannot join
- Women with organic or hormonal bleeding etiologies.
- Patients taking medicines which may affect the coagulation cascade or the number or function of platelets.
- Those women who otherwise do not comply with the inclusion criteria.
Trial locations and contacts
2 sites in 1 country.
Ecuador2 sites
UAzuay
Cuenca, Azuay, 0001
Universidad del Azuay (UDA)
Cuenca, Azuay, 0001
About this study
- Hypothesis
- There are patients with von Willebrand Disease in Cuenca.
- Primary question
- How many women referred with a history of bleeding may have von Willebrand disease?
- Secondary
- Associations between the bleeding score and initial laboratory studies
- What are the differences on subgroups of enrolled patients with the bleeding score?
- Ancillary
- What is the clinical and socio-economic status of women with von Willebrand Disease in Cuenca?
- What is the clinical and socio-economic status of patients with Hemophilia in Cuenca?
Read the detailed description
The incidence of hemophilia A is 1 in 10,000 and the one of hemophilia B is 1 in 30,000 live born males. As the most frequent coagulopathy in the world, von Willebrand disease (vWD) can affect 1 in 1000 children of both genders. The prevalence of vWD is 1-2% and it may be as high as 5-15% among women with menorrhagia. So vWD can be present in 1 of every 100 to 500 individuals.
A world survey published that Ecuador, with a population of 14,790,608 people, has 238 patients with hemophilia, 45 people with von Willebrand disease (vWD) and 13 patients with other bleeding disorders including rare factor deficiencies and platelet disorders.
The Ecuadorian census of 2010 indicates that Cuenca district (canton) has 505,585 inhabitants. According to a local reference, Cuenca has no reports of vWD and it has registered 20-40 patients with hemophilia A and B. However Cuenca may host approximately 10,000 people with vWD because it affects both males and females. One plausible explanation for this lag is that the diagnostic suspicions of vWD had not been proven because of the lack of a special coagulation laboratory in Cuenca that would allow professionals to make a positive diagnosis.
This is the first clinical, demographic and socio-economic study of patients registered with hemophilia and of Cuenca women with possible vWD who are referred from dentists and physicians from public and private health centers. The instruments will be validated surveys and screening coagulation studies.
As far as we know Ecuador has not published studies on coagulopathies. Our aim is to initiate a process to eventually reach the goal to offer adequate diagnostic and therapeutic interventions to human victims of these diseases, within a program of service, teaching and research.
- Conditions
- Von Willebrand Disease, Hemophilia A, Hemophilia B
- Keywords
- vWD
- Medical subject headings
- von Willebrand Diseases, Hemophilia A, Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| von Willebrand women | Referred women who may have von Willebrand Disease |
What is being measured
The primary outcome is the main question the study is designed to answer.
Bleeding Score (BS)
As an adequate bleeding history is fundamental, the Condensed MCMDM-1 Bleeding Questionnaire will be used. Its bleeding score (BS) has a sensitivity of 100%, specificity 87%, positive predictive value 0.2 and negative predictive value 1.0. There was a signifficant difference of BS between the subtypes of vWD: with type 3 >>type 2 >>type 1 vWD (Anova P <0.001).
AS the BS is an index of ordinal values, medians and the Mann-Whitney non-parametric ranksum test will be used, to study the bleeding score (BS) differences among subgroups of enrolled women.
Measured: 1 week
Secondary outcomes (1)
Associations among BS and female subgroup characteristics and initial laboratory tests.
As bleeding may tend to associate in subjects, i.e. epistaxis and menorrhagia, a logistic (log-linear) model will be used. As bleeding can also ocur independently, symptoms will be coded for an adjusted covariate analysis.
To study the relationships among the BS and the quantitative laboratory results, the BS will be divided in quintiles, and an adjusted multiple regression model will be used. Likelihood ratios (LR) for values of BS, with a prevalence of 5% and ROC curves will be applied. According to Tosetto et.al. laboratory data will be expressed in categorical and quantitative scales.
Measured: 1 week
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted2 May 2012 (estimated)
- Study startMar 2013
- Primary completionJun 2013
- Study completionJun 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Von Willebrand DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Enrolment
- 81 participants (actual)
Sponsor and investigators
- Lead sponsor
- Universidad del Azuay
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jaime M. Moreno A., M.D., M.S., F.A.C.P., Universidad del Azuay
- Investigators
- Jaime M. Moreno A., M.D., M.S., Principal investigator, Universidad del Azuay (UAzuay)Jacinto Guillen G., Eng., M.S., Study chair, Universidad del Azuay (UAzuay)Rodrigo Cueva M., Econ., M.S., Study director, Universidad del Azuay (UAzuay)
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01589848
- Sponsor's protocol ID
- 001
- Other IDs
- UAzuay
- Acronym
- VWD
- Status last verified
- Jul 2013
- First submitted
- 30 Apr 2012
- Last updated
- 3 Jul 2013
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 3 Jul 2013. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study on Von Willebrand Disease and Hemophilia in Cuenca, Ecuador
- ClinicalTrials.gov ID
- NCT01589848
- Acronym / sponsor ID
- VWD / 001
- Conditions
- Von Willebrand Disease, Hemophilia A, Hemophilia B
- Keywords
- vWD
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 81 participants (actual)
- Lead sponsor
- Universidad del AzuayUniversity, hospital or other
- Study start
- Mar 2013
- Primary completion
- Jun 2013 (actual)
- Study completion
- Jun 2013 (actual)
- First posted
- 2 May 2012 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
- Hypothesis
- There are patients with von Willebrand Disease in Cuenca.
- Primary question
- How many women referred with a history of bleeding may have von Willebrand disease?
- Secondary
- Associations between the bleeding score and initial laboratory studies
- What are the differences on subgroups of enrolled patients with the bleeding score?
- Ancillary
- What is the clinical and socio-economic status of women with von Willebrand Disease in Cuenca?
- What is the clinical and socio-economic status of patients with Hemophilia in Cuenca?
Detailed description
The incidence of hemophilia A is 1 in 10,000 and the one of hemophilia B is 1 in 30,000 live born males. As the most frequent coagulopathy in the world, von Willebrand disease (vWD) can affect 1 in 1000 children of both genders. The prevalence of vWD is 1-2% and it may be as high as 5-15% among women with menorrhagia. So vWD can be present in 1 of every 100 to 500 individuals.
A world survey published that Ecuador, with a population of 14,790,608 people, has 238 patients with hemophilia, 45 people with von Willebrand disease (vWD) and 13 patients with other bleeding disorders including rare factor deficiencies and platelet disorders.
The Ecuadorian census of 2010 indicates that Cuenca district (canton) has 505,585 inhabitants. According to a local reference, Cuenca has no reports of vWD and it has registered 20-40 patients with hemophilia A and B. However Cuenca may host approximately 10,000 people with vWD because it affects both males and females. One plausible explanation for this lag is that the diagnostic suspicions of vWD had not been proven because of the lack of a special coagulation laboratory in Cuenca that would allow professionals to make a positive diagnosis.
This is the first clinical, demographic and socio-economic study of patients registered with hemophilia and of Cuenca women with possible vWD who are referred from dentists and physicians from public and private health centers. The instruments will be validated surveys and screening coagulation studies.
As far as we know Ecuador has not published studies on coagulopathies. Our aim is to initiate a process to eventually reach the goal to offer adequate diagnostic and therapeutic interventions to human victims of these diseases, within a program of service, teaching and research.
Study design
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| von Willebrand women | – | –Referred women who may have von Willebrand Disease |
Who may qualify
- Ages
- 18 to 50 years
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Adult
- Study population
- Target population: all women from Cuenca district with a hemorrhage history. Accessible population: Those women referred from professionals with an initial hemorrhage history, who comply with the inclusion criteria and who sign the informed consert form. Sample population: First comers referred with a hemorrhage history until fulfilling the projected sample size. This non-probabilistic purposive sample will be used because it has been reported that it could represent the target population. *2010 Ecuadorian census: 131.163 women ages 18 to 50 years, at Cuenca district.
Key inclusion criteria (7 listed)
- Subjects living at urban and rural parishes from Cuenca district.
- Subjects referred from professionals of public and private health services, who have filled the initial hemorrhage survey.
- Must be referred women who sign the informed consent form.
- Patients who answer the standardized bleeding score questionnaire (BS).
- Patients who answer the quality of life questionnaire (SF-12).
Key exclusion criteria (3 listed)
- Women with organic or hormonal bleeding etiologies.
- Patients taking medicines which may affect the coagulation cascade or the number or function of platelets.
- Those women who otherwise do not comply with the inclusion criteria.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Bleeding Score (BS)
Time frame: 1 week
As an adequate bleeding history is fundamental, the Condensed MCMDM-1 Bleeding Questionnaire will be used. Its bleeding score (BS) has a sensitivity of 100%, specificity 87%, positive predictive value 0.2 and negative predictive value 1.0. There was a signifficant difference of BS between the subtypes of vWD: with type 3 >>type 2 >>type 1 vWD (Anova P <0.001). AS the BS is an index of ordinal values, medians and the Mann-Whitney non-parametric ranksum test will be used, to study the bleeding score (BS) differences among subgroups of enrolled women.
1 secondary outcome
Associations among BS and female subgroup characteristics and initial laboratory tests.
Time frame: 1 week
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jul 2013.
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HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB
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Von Willebrand Diseases
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jaime M. Moreno A., M.D., M.S. principal investigator
Universidad del Azuay (UAzuay)
Jacinto Guillen G., Eng., M.S. study chair
Universidad del Azuay (UAzuay)
Rodrigo Cueva M., Econ., M.S. study director
Universidad del Azuay (UAzuay)
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Von Willebrand Disease and Hemophilia A, run by Universidad del Azuay at 2 sites in Ecuador.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 3 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Jul 2013. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Ecuador. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Bleeding Score (BS) (time frame: 1 week). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Jul 2013.