Hemophilia ANCT02093065
Platelet Function in Patients With Hemophilia A
An observational study of Hemophilia A, run by Boston Children's Hospital at 1 site in United States.
Trial summary
Abnormalities in the gene encoding Factor VIII (FVIII) results in hemophilia A, an X-linked recessive bleeding disorder with a prevalence of 1 in 5000 males. Hemophilia A patients are classified into 3 different categories based on residual FVIII activity compared to normal: mild (6-40%), moderate (1-5%) and severe… Read more about this study
- Who is it for?
- Ages 2 to 18 years, men only
- Conditions the study is for
- Hemophilia A
- How many people
- 36 participants
- Where
- 1 siteUnited States
- Study dates
- Mar 2015to 1 Feb 2020
- 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 2 to 18 years
- Men only
- No healthy volunteers
- Child, Adult
Study population: Patients with severe hemophilia A who do not have inhibitors against FVIII and who have a bleeding history of at least 6 months.
Who may be able to join
- Patients with severe hemophilia A who are being prophylactically treated with FVIII.
- Age of at least 2 years.
- Bleeding history of at least 6 months.
- IRB-approved informed consent.
Who cannot join
- Presence of FVIII inhibitors.
- Greater than 7 days since active bleeding.
Trial locations and contacts
1 site in 1 country.
United States1 site
Boston Children's Hospital, Boston Hemophilia Center
Boston, Massachusetts, 02115
About this study
Official title: Decreased Platelet Function as a Cause of Increased Bleeding in Patients With Hemophilia A
Abnormalities in the gene encoding Factor VIII (FVIII) results in hemophilia A, an X-linked recessive bleeding disorder with a prevalence of 1 in 5000 males. Hemophilia A patients are classified into 3 different categories based on residual FVIII activity compared to normal: mild (6-40%), moderate (1-5%) and severe (<1%). This categorization correlates to some degree with bleeding phenotype, but does not completely define it. Some patients with hemophilia A bleed less often than others despite identical plasma FVIII levels. The cause(s) of this phenotype heterogeneity in hemophilia A remains largely unknown, despite a number of studies of possible factors.
Activated platelets, in addition to their role in primary hemostasis, play a major role in secondary hemostasis (coagulation) by providing a phospholipid surface to which coagulation factors bind. A role for platelets in the hemorrhagic propensity of hemophilia A has been suggested in the past, but only a small number of studies have been performed with limitations in assays performed and numbers of patients. The purpose of the present study is to determine whether platelet reactivity in severe hemophilia A patients is associated with past bleeding frequency and/or predicts future bleeding frequency.
- Conditions
- Hemophilia A
- Keywords
- severe hemophilia A, coated platelets, bleeding
- Medical subject headings
- Hemophilia A, Hemorrhage
What is being measured
The primary outcome is the main question the study is designed to answer.
The percentage of coated platelets.
Measured: 2 years
Secondary outcomes (2)
Platelet reactivity.
Measured: 2 years
The number of procoagulant platelet-derived microparticles.
Measured: 2 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Mar 2014 (estimated)
- Study startMar 2015
- Primary completionJun 2018
- Study completion1 Feb 2020
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 Boston Children's HospitalThe 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
- Prospective
- Enrolment
- 36 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boston Children's Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Baxter Healthcare Corporation
- Responsible party
- Principal investigator, Alan D. Michelson, Boston Children's Hospital
- Investigators
- Alan D Michelson, MD, Principal investigator, Boston Children's Hospital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02093065
- Sponsor's protocol ID
- BCH-CPRS-hemophilia A
- Status last verified
- Jul 2020
- First submitted
- 19 Mar 2014
- Last updated
- 14 Jul 2020
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 14 Jul 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Decreased Platelet Function as a Cause of Increased Bleeding in Patients With Hemophilia A
- ClinicalTrials.gov ID
- NCT02093065
- Acronym / sponsor ID
- BCH-CPRS-hemophilia A
- Conditions
- Hemophilia A
- Keywords
- severe hemophilia A, coated platelets, bleeding
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 36 participants (actual)
- Lead sponsor
- Boston Children's HospitalUniversity, hospital or other
- Collaborators
- Baxter Healthcare Corporation
- Study start
- Mar 2015
- Primary completion
- Jun 2018 (actual)
- Study completion
- 1 Feb 2020 (actual)
- First posted
- 20 Mar 2014 (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.
Abnormalities in the gene encoding Factor VIII (FVIII) results in hemophilia A, an X-linked recessive bleeding disorder with a prevalence of 1 in 5000 males. Hemophilia A patients are classified into 3 different categories based on residual FVIII activity compared to normal: mild (6-40%), moderate (1-5%) and severe (<1%). This categorization correlates to some degree with bleeding phenotype, but does not completely define it. Some patients with hemophilia A bleed less often than others despite identical plasma FVIII levels. The cause(s) of this phenotype heterogeneity in hemophilia A remains largely unknown, despite a number of studies of possible factors.
Activated platelets, in addition to their role in primary hemostasis, play a major role in secondary hemostasis (coagulation) by providing a phospholipid surface to which coagulation factors bind. A role for platelets in the hemorrhagic propensity of hemophilia A has been suggested in the past, but only a small number of studies have been performed with limitations in assays performed and numbers of patients. The purpose of the present study is to determine whether platelet reactivity in severe hemophilia A patients is associated with past bleeding frequency and/or predicts future bleeding frequency.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
Who may qualify
- Ages
- 2 to 18 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
- Study population
- Patients with severe hemophilia A who do not have inhibitors against FVIII and who have a bleeding history of at least 6 months.
Key inclusion criteria (4 listed)
- Patients with severe hemophilia A who are being prophylactically treated with FVIII.
- Age of at least 2 years.
- Bleeding history of at least 6 months.
- IRB-approved informed consent.
Key exclusion criteria (2 listed)
- Presence of FVIII inhibitors.
- Greater than 7 days since active bleeding.
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 percentage of coated platelets.
Time frame: 2 years
2 secondary outcomes
Platelet reactivity.
Time frame: 2 years
The number of procoagulant platelet-derived microparticles.
Time frame: 2 years
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 14 Jul 2020.
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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.
Alan D Michelson, MD principal investigator
Boston Children's Hospital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A, run by Boston Children's Hospital at 1 site in United States.
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 4 inclusion and 2 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 14 Jul 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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 percentage of coated platelets. (time frame: 2 years). 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 14 Jul 2020.