Hemophilia ANCT00936312
Females With Severe or Moderate Hemophilia A or B: an International Multi-center Study
An observational study of Hemophilia A and Hemophilia B, run by Weill Medical College of Cornell University at 11 sites in 9 countries.
Trial summary
This is a multi-center, international study designed to collect clinical, genetic and quality of life information on females with hemophilia, an inherited bleeding disorder. The study is designed to determine whether there are problems and issues unique to females with hemophilia. Read more about this study
- Who is it for?
- Any age, women only
- Conditions the study is for
- Hemophilia A, Hemophilia B
- How many people
- 200 participants
- Where
- 11 sitesin 9 countries
- Study dates
- Mar 2008to Aug 2011
- 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 Any age
- Women only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Females with severe or moderate Hemophilia A (less than or equal to 5%) Females with severe or moderate Hemophilia B (less than or equal to 5%)
Who may be able to join
- Females with severe (FVIII<0.01u/ml) or moderate (FVIII 0.01≤0.05 u/ml) hemophilia A
- Females with severe (FIX<0.01u/ml) or moderate (FIX 0.01≤0.05u/ml) hemophilia B
- Willingness to participate in the study.
Who cannot join
- Subjects who do not meet the inclusion criteria with respect to gender or hemophilia severity.
Trial locations and contacts
11 sites in 9 countries.
11 sites in 9 countries
Italy2 sites
Instituto G. Gaslini
Genova
A. Bianchi Bonomi Hemophilia Center
Milan
United States1 site
Weill Cornell Medical College
New York, New York, 10065
About this study
This is a multi-center, international study designed to collect clinical, genetic and quality of life information on females with hemophilia, an inherited bleeding disorder. The study is designed to determine whether there are problems and issues unique to females with hemophilia.
Read the detailed description
The study involves two questionnaires: one on the diagnosis, symptoms, complications and treatment of each participant to be completed by a staff member; the other is a questionnaire to be completed by the participant on how the disease has affected her life. Finally, for those participants who have not previously had genetic testing, the third part of the study is an optional blood test to determine the genetic cause, what change in the factor VIII or factor IX gene, caused the hemophilia. The test results will be available to those participants who wish to learn their results. With the data we collect we will compile a database to examine the connection between the genetic cause of hemophilia and the course and symptoms of the disease.
- Conditions
- Hemophilia A, Hemophilia B
- Medical subject headings
- 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 |
|---|---|---|
| Females with Hemophilia | Females with severe or moderate Hemophilia A or B | |
| Control group | Male subjects with severe Hemophilia A or B and female subjects with mild (20-60%) Hemophilia A or B. |
What is being measured
The primary outcome is the main question the study is designed to answer.
The molecular and cytogenetic etiology of the condition will be compiled. Clinical manifestation demonstrated by the female hemophiliacs will be compared to published data available. Genotype and phenotype will be correlated.
Measured: 1-2 visits
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Jul 2009 (estimated)
- Study startMar 2008
- Primary completionAug 2011
- Study completionAug 2011
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- More trials run by Weill Medical College of Cornell UniversityThe sponsor's other registered studies.
- 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
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 200 participants (actual)
Sponsor and investigators
- Lead sponsor
- Weill Medical College of Cornell University
- Sponsor type
- University, hospital or other
- Collaborators
- Bayer, University Hospital, Bonn
- Responsible party
- Sponsor
- Investigators
- William B Mitchell, MD, Principal investigator, Weill Medical College of Cornell University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00936312
- Sponsor's protocol ID
- Intl Female Hemophilia Study
- Status last verified
- Dec 2012
- First submitted
- 9 Jul 2009
- Last updated
- 10 Dec 2012
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Dec 2012. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Females With Severe or Moderate Hemophilia A or B: an International Multi-center Study
- ClinicalTrials.gov ID
- NCT00936312
- Acronym / sponsor ID
- Intl Female Hemophilia Study
- Conditions
- Hemophilia A, Hemophilia B
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 200 participants (actual)
- Lead sponsor
- Weill Medical College of Cornell UniversityUniversity, hospital or other
- Collaborators
- Bayer, University Hospital, Bonn
- Study start
- Mar 2008
- Primary completion
- Aug 2011 (actual)
- Study completion
- Aug 2011 (actual)
- First posted
- 10 Jul 2009 (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.
This is a multi-center, international study designed to collect clinical, genetic and quality of life information on females with hemophilia, an inherited bleeding disorder. The study is designed to determine whether there are problems and issues unique to females with hemophilia.
Detailed description
The study involves two questionnaires: one on the diagnosis, symptoms, complications and treatment of each participant to be completed by a staff member; the other is a questionnaire to be completed by the participant on how the disease has affected her life. Finally, for those participants who have not previously had genetic testing, the third part of the study is an optional blood test to determine the genetic cause, what change in the factor VIII or factor IX gene, caused the hemophilia. The test results will be available to those participants who wish to learn their results. With the data we collect we will compile a database to examine the connection between the genetic cause of hemophilia and the course and symptoms of the disease.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Females with Hemophilia | – | –Females with severe or moderate Hemophilia A or B |
| Control group | – | –Male subjects with severe Hemophilia A or B and female subjects with mild (20-60%) Hemophilia A or B. |
Who may qualify
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Females with severe or moderate Hemophilia A (less than or equal to 5%) Females with severe or moderate Hemophilia B (less than or equal to 5%)
Key inclusion criteria (3 listed)
- Females with severe (FVIII<0.01u/ml) or moderate (FVIII 0.01≤0.05 u/ml) hemophilia A
- Females with severe (FIX<0.01u/ml) or moderate (FIX 0.01≤0.05u/ml) hemophilia B
- Willingness to participate in the study.
Key exclusion criteria (1 listed)
- Subjects who do not meet the inclusion criteria with respect to gender or hemophilia severity.
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
The molecular and cytogenetic etiology of the condition will be compiled. Clinical manifestation demonstrated by the female hemophiliacs will be compared to published data available. Genotype and phenotype will be correlated.
Time frame: 1-2 visits
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 10 Dec 2012.
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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.
William B Mitchell, MD principal investigator
Weill Medical College of Cornell University
11 sites in 9 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A and Hemophilia B, run by Weill Medical College of Cornell University at 11 sites in 9 countries.
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 3 inclusion and 1 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 10 Dec 2012. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 11 sites in Germany, Italy, United States, Australia, Israel and 4 more countries. 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?
The molecular and cytogenetic etiology of the condition will be compiled. Clinical manifestation demonstrated by the female hemophiliacs will be compared to published data available. Genotype and phenotype will be correlated. (time frame: 1-2 visits). 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 10 Dec 2012.