Hemophilia ANCT00936845
Females With Severe or Moderate Hemophilia A or B: A Multi-Center Study
An observational study of Hemophilia A and Hemophilia B, run by Weill Medical College of Cornell University at 19 sites in 2 countries.
Trial summary
To collect and analyze data on females with hemophilia so as to better define the difference between the study population and the male population with hemophilia. Read more about this study
- Who is it for?
- Ages 1 month and older, women only
- Conditions the study is for
- Hemophilia A, Hemophilia B
- How many people
- 22 participants
- Where
- 19 sitesin 2 countries
- Study dates
- Apr 2005to Jan 2010
- 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 1 month and older
- Women only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Females with severe or moderate (less than 5%) hemophilia A or B.
Who may be able to join
- Females with severe (FVIII<0.01u/dl) or moderate (FVIII-0.01<0.06 u/dl) hemophilia A
- Females with severe (FIX<0.01u/dl) or moderate (FIX-0.01<0.06u/dl) 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
19 sites in 2 countries.
19 sites in 2 countries
United States18 sites
Children's Hospital Medical Center of Akron
Akron, Ohio, 44308
Emory University Hemophilia Program Office
Atlanta, Georgia, 30322
Mountain States Regional Hemophilia and Thrombosis Center
Aurora, Colorado, 80045
Boston Hemophilia Center- Children's Hospital
Boston, Massachusetts, 02115
Hemophilia Center of Western New York - Pediatric
Buffalo, New York, 14222
Northwestern University
Chicago, Illinois, 60611
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Henry Ford Hospital Adult Hemophilia and Thrombosis Treatment Center
Detroit, Michigan, 48202
City of Hope National Medical Center
Duarte, California, 91010
Gulf States Hemophilia and Thrombophilia Center
Houston, Texas, 77030
University of Kentucky Hemophilia Treatment Center
Lexington, Kentucky, 40536
Long Island Jewish Medical Center
New Hyde Park, New York, 11040
Weill Cornell Medical College
New York, New York, 10065
Newark Beth Israel Medical Center
Newark, New Jersey, 07112
Oklahoma Center for Bleeding Disorders
Oklahoma City, Oklahoma, 73104
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Mary M. Gooley Hemophilia Center, Inc.
Rochester, New York, 14621
Georgetown University Hospital
Washington D.C., District of Columbia, 20007
About this study
To collect and analyze data on females with hemophilia so as to better define the difference between the study population and the male population with hemophilia.
Read the detailed description
This study is designed as an epidemiological study with retrospective clinical and genetic data collection and prospective quality of life (QOL) data collections and genotyping of subjects without current molecular diagnosis. Data on the genetic/chromosomal etiology of the condition will be collected through chart review. For those subjects who have not had molecular or cytogenetic analysis, molecular and cytogenetic testing will be offered after genetic counseling. Those subjects who agree to have genetic testing will have 5-10 ml of blood drawn and sent to the cytogenetic laboratory at Weill Cornell Medical College for testing to determine the chromosomal complement, and 5 ml of blood sent to Dr. Arupa Ganguly at the University of Pennsylvania for the identification of a factor VIII or factor IX mutation, and X-chromosome inactivation studies.
- 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. |
What is being measured
The primary outcome is the main question the study is designed to answer.
The prevalence of females with severe or moderate hemophilia A or B in the United States.
Measured: Study Duration
Secondary outcomes (3)
Collect existing data on the genetic and/or chromosomal causes for severe and moderate hemophilia A and B in females in the United States.
Measured: Study Duration
To determine the natural history of hemophilia in females by collecting and analyzing data on the symptoms, treatment patterns, and complications of hemophilia in this cohort.
Measured: Study Duration
Collect and analyze data on the health-related quality of life and psychosocial adaptation to disease of females in the United States.
Measured: Study Duration
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 startApr 2005
- Primary completionJan 2010
- Study completionJan 2010
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
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Weill Medical College of Cornell University
- Sponsor type
- University, hospital or other
- Collaborators
- CSL Behring, University of Pennsylvania
- Responsible party
- Sponsor
- Investigators
- William B Mitchell, MD, Principal investigator, Weill Medical College of Cornell University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00936845
- Sponsor's protocol ID
- Females with Hemophilia
- 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: A Multi-Center Study
- ClinicalTrials.gov ID
- NCT00936845
- Acronym / sponsor ID
- Females with Hemophilia
- Conditions
- Hemophilia A, Hemophilia B
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Weill Medical College of Cornell UniversityUniversity, hospital or other
- Collaborators
- CSL Behring, University of Pennsylvania
- Study start
- Apr 2005
- Primary completion
- Jan 2010 (actual)
- Study completion
- Jan 2010 (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.
To collect and analyze data on females with hemophilia so as to better define the difference between the study population and the male population with hemophilia.
Detailed description
This study is designed as an epidemiological study with retrospective clinical and genetic data collection and prospective quality of life (QOL) data collections and genotyping of subjects without current molecular diagnosis. Data on the genetic/chromosomal etiology of the condition will be collected through chart review. For those subjects who have not had molecular or cytogenetic analysis, molecular and cytogenetic testing will be offered after genetic counseling. Those subjects who agree to have genetic testing will have 5-10 ml of blood drawn and sent to the cytogenetic laboratory at Weill Cornell Medical College for testing to determine the chromosomal complement, and 5 ml of blood sent to Dr. Arupa Ganguly at the University of Pennsylvania for the identification of a factor VIII or factor IX mutation, and X-chromosome inactivation studies.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Females with Hemophilia | – | –Females with severe or moderate Hemophilia A or B. |
Who may qualify
- Ages
- 1 month and older
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Females with severe or moderate (less than 5%) hemophilia A or B.
Key inclusion criteria (3 listed)
- Females with severe (FVIII<0.01u/dl) or moderate (FVIII-0.01<0.06 u/dl) hemophilia A
- Females with severe (FIX<0.01u/dl) or moderate (FIX-0.01<0.06u/dl) 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 prevalence of females with severe or moderate hemophilia A or B in the United States.
Time frame: Study Duration
3 secondary outcomes
Collect existing data on the genetic and/or chromosomal causes for severe and moderate hemophilia A and B in females in the United States.
Time frame: Study Duration
To determine the natural history of hemophilia in females by collecting and analyzing data on the symptoms, treatment patterns, and complications of hemophilia in this cohort.
Time frame: Study Duration
Collect and analyze data on the health-related quality of life and psychosocial adaptation to disease of females in the United States.
Time frame: Study Duration
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.
Related trials for Hemophilia A
RecruitingObservational study
The CACHE Study: Coronary Artery Care in HaEmophilia
Haemophilia, Cardiovascular Diseases
Active, not recruitingObservational study
A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
Hemophilia A or B
RecruitingPhase 1/2
A Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of NXT007 in Persons With Severe or Moderate Hemophilia A
Hemophilia A
Active, not recruitingPhase 3
A Study to Evaluate the Efficacy and Safety of Factor IX Gene Therapy With PF-06838435 in Adult Males With Moderately Severe to Severe Hemophilia B
Hemophilia B
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B
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
19 sites in 2 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 19 sites in 2 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 19 sites in United States, Puerto Rico. 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 prevalence of females with severe or moderate hemophilia A or B in the United States. (time frame: Study Duration). 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.