HemophiliaIFIENCT02726139
Improved Factor VIII Inhibitor Evaluation
An observational study of Hemophilia and Hemophilia A, run by Boston VA Research Institute, Inc. at 1 site in United States.
Trial summary
Commercial one and two-stage factor VIII assays may not detect some clinically significant inhibitor antibodies. The purpose of the proposed study is to standardize and validate a platelet-based factor VIII activity assay with greater sensitivity to clinically important inhibitory antibodies. Read more about this study
- Who is it for?
- Any age, men only
- Conditions the study is for
- Hemophilia, Hemophilia A
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 15 Jul 2016to 25 Jan 2021
- 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
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Patients with hemophilia A and inhibitory antibodies that have agreed to store plasma and prospectively consented to testing to evaluate bleeding mechanism
Who may be able to join
- hemophilia A
- presence of inhibitory antibodies
Who cannot join
- none
Trial locations and contacts
1 site in 1 country.
United States1 site
Boston VA Research Institute (BVARI)
Boston, Massachusetts, 02130
About this study
Commercial one and two-stage factor VIII assays may not detect some clinically significant inhibitor antibodies. The purpose of the proposed study is to standardize and validate a platelet-based factor VIII activity assay with greater sensitivity to clinically important inhibitory antibodies. Investigators will evaluate the platelet-dependent inhibitory activity vs. conventional inhibitory activity in stored patient plasmas and correlate to bleeding histories
Read the detailed description
Factor VIII activity assays are used to diagnose hemophilia A, to monitor treatment of these patients, to determine when inhibitory antibodies against factor VIII have developed, and to evaluate the activity of engineered pharmaceutical factor VIII products. Factor VIII activity has been measured with one and two-stage assays for more than fifty years. However, all existing assays have major shortcomings that are recognized by the FDA, the International Society for Thrombosis and Haemostasis, and pharmaceutical companies. The major deficiencies that have been identified are: 1) Factor VIII activity levels do not predict the risk of bleeding in patients with inhibitory antibodies. The degree of inhibition in these assays is less than required to explain patient bleeding. 2) The assays are only accurate over a range of 1 - 100% of normal factor VIII activity, while values of 0.1 - 1% are also clinically important. 3) The different approved assays give discrepant values for recombinant pharmaceutical factor VIII products, with a range of 2-fold difference between assays. This can lead to clinically important differences in dosing of factor VIII products from one region to another with corresponding risk of thrombosis or bleeding.
The preliminary data identify an additional deficiency of commercial assays that has not been anticipated. Commercial one and two-stage assays may not detect some clinically significant inhibitor antibodies. Thus, the purpose of the proposed studies is to standardize and validate a platelet-based factor VIII activity assay with greater sensitivity to clinically important inhibitory antibodies.
- Conditions
- Hemophilia, Hemophilia A
- Keywords
- Factor VIII, inhibitor, assay
- Medical subject headings
- Hemophilia A
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 |
|---|---|---|
| Emory University | Samples obtained at and shipped from Emory University | |
| University of Michigan | Samples obtained at and shipped from University of Michigan |
What is being measured
The primary outcome is the main question the study is designed to answer.
Bleeding exceeds risk predicted by factor VIII assay
annual bleeding rate exceeds the rate predicted from the residual factor VIII activity
Measured: 1 year
Bleeding is in accord with the risk assigned by platelet-dependent factor VIII activity
annual bleeding rate is within the predicted range from residual factor VIII activity
Measured: 1 year
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted1 Apr 2016 (estimated)
- Study start15 Jul 2016
- Primary completion25 Jan 2021
- Study completion25 Jan 2021
Why it stopped: No participants enrolled
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for HemophiliaStudies 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
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- Boston VA Research Institute, Inc.
- Sponsor type
- University, hospital or other
- Collaborators
- Shire, Emory University, University of Michigan
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02726139
- Sponsor's protocol ID
- BVARI_GG_2016_1
- Acronym
- IFIE
- Status last verified
- Mar 2016
- First submitted
- 29 Mar 2016
- Last updated
- 25 Feb 2021
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 25 Feb 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Improved Factor VIII Inhibitor Evaluation
- ClinicalTrials.gov ID
- NCT02726139
- Acronym / sponsor ID
- IFIE / BVARI_GG_2016_1
- Conditions
- Hemophilia, Hemophilia A
- Keywords
- Factor VIII, inhibitor, assay
- Study type
- Observational
- Recruitment status
- WithdrawnReason stopped: No participants enrolled
- Enrolment
- 0 participants (actual)
- Lead sponsor
- Boston VA Research Institute, Inc.University, hospital or other
- Collaborators
- Shire, Emory University, University of Michigan
- Study start
- 15 Jul 2016 (actual)
- Primary completion
- 25 Jan 2021 (actual)
- Study completion
- 25 Jan 2021 (actual)
- First posted
- 1 Apr 2016 (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.
Commercial one and two-stage factor VIII assays may not detect some clinically significant inhibitor antibodies. The purpose of the proposed study is to standardize and validate a platelet-based factor VIII activity assay with greater sensitivity to clinically important inhibitory antibodies. Investigators will evaluate the platelet-dependent inhibitory activity vs. conventional inhibitory activity in stored patient plasmas and correlate to bleeding histories
Detailed description
Factor VIII activity assays are used to diagnose hemophilia A, to monitor treatment of these patients, to determine when inhibitory antibodies against factor VIII have developed, and to evaluate the activity of engineered pharmaceutical factor VIII products. Factor VIII activity has been measured with one and two-stage assays for more than fifty years. However, all existing assays have major shortcomings that are recognized by the FDA, the International Society for Thrombosis and Haemostasis, and pharmaceutical companies. The major deficiencies that have been identified are: 1) Factor VIII activity levels do not predict the risk of bleeding in patients with inhibitory antibodies. The degree of inhibition in these assays is less than required to explain patient bleeding. 2) The assays are only accurate over a range of 1 - 100% of normal factor VIII activity, while values of 0.1 - 1% are also clinically important. 3) The different approved assays give discrepant values for recombinant pharmaceutical factor VIII products, with a range of 2-fold difference between assays. This can lead to clinically important differences in dosing of factor VIII products from one region to another with corresponding risk of thrombosis or bleeding.
The preliminary data identify an additional deficiency of commercial assays that has not been anticipated. Commercial one and two-stage assays may not detect some clinically significant inhibitor antibodies. Thus, the purpose of the proposed studies is to standardize and validate a platelet-based factor VIII activity assay with greater sensitivity to clinically important inhibitory antibodies.
Study design
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Emory University | – | –Samples obtained at and shipped from Emory University |
| University of Michigan | – | –Samples obtained at and shipped from University of Michigan |
Who may qualify
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Patients with hemophilia A and inhibitory antibodies that have agreed to store plasma and prospectively consented to testing to evaluate bleeding mechanism
Key inclusion criteria (2 listed)
- hemophilia A
- presence of inhibitory antibodies
Key exclusion criteria (1 listed)
- none
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 exceeds risk predicted by factor VIII assay
Time frame: 1 year
annual bleeding rate exceeds the rate predicted from the residual factor VIII activity
Bleeding is in accord with the risk assigned by platelet-dependent factor VIII activity
Time frame: 1 year
annual bleeding rate is within the predicted range from residual factor VIII activity
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 Feb 2021.
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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.
The record lists no contact. Use the official record to reach the sponsor.
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 and Hemophilia A, run by Boston VA Research Institute, Inc. 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 2 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: withdrawn, as of the sponsor's last update on 25 Feb 2021. 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 was withdrawn before enrolling participants, so there are no results.
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 exceeds risk predicted by factor VIII assay (time frame: 1 year). The study lists 2 primary outcomes in all. 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 25 Feb 2021.