Hemophilia ANCT02582060
Individualizing Hemophilia Prophylaxis Using Thromboelastography
A study testing Thromboelastography/Thromboelastometry-Guided Treatment Regimen in people with Hemophilia A, run by Children's Hospital Los Angeles at 1 site in United States.
Trial summary
Currently dosing for prophylaxis is not individualized, and the general approach is to use a dose of 25-40 units/kg given 3 times per week or every other day. One of the issues with weight-based dosing is the possible over-treatment. Read more about this study
- Who is it for?
- Ages 5 to 70 years, men only
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Thromboelastography/Thromboelastometry-Guided Treatment Regimen
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 1 siteUnited States
- Study dates
- 20 May 2014to 31 Dec 2017
- Phase
- Interventional
- 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 5 to 70 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Males, 5-70 years, inclusive
- Plasma FVIII activity <1% documented (Laboratory result or MD documentation of Severe Hemophilia A diagnosis)
- Currently prescribed prophylaxis treatment regimen infusing ≥3 times a week
- Willing to alter their prophylaxis treatment regimen per study protocol
Who cannot join
- Bleeding disorder(s) other than Hemophilia A
- Current inhibitor (>0.6BU)
- Thrombocytopenia (platelet count <100,000K/µL since it can alter TEG®/ROTEM® results)
- Creatinine >2x the upper limit of normal (indicating potential platelet dysfunction)
- Prothrombin time >3 seconds above the upper limit of normal (indicating potential liver dysfunction)
- Any concurrent clinically significant major disease, frequent bleeding pattern or history of non-compliance that, in the opinion of the investigator, would make the subject unsuitable for enrollment
Show all 9Show fewer
- Participation within the past 30 days in any other clinical study involving investigational drugs
- Planned major surgery within 30 days prior to screening or during the study period
- Current use of any medication known to have effects on the coagulation system
Trial locations and contacts
1 site in 1 country.
About this study
Currently dosing for prophylaxis is not individualized, and the general approach is to use a dose of 25-40 units/kg given 3 times per week or every other day. One of the issues with weight-based dosing is the possible over-treatment. This is likely due to the fact that laboratory tests are not sensitive enough at the low levels to support decision-making. The Thromboelastograph (TEG®) and Thromboelastometry (ROTEM®) are coagulation devices, which assess the dynamics of clot formation over time and have several characteristics which suggest they may provide important information for individualized prophylaxis treatment for our patients.
Read the detailed description
This is a pilot study in children and adults with severe hemophilia A that utilizes the TEG/ROTEM to personalize their prophylaxis treatment.
Primary objectives:
- Assess the feasibility of TEG/ROTEM-guided modification of the current prophylaxis regimen for individuals with severe hemophilia A. Specifically, to:
- Test and refine the operational protocol for using TEG/ROTEM to guide factor dosing
- Estimate the proportion of hemophilia A patients whose dose will be modified based on TEG/ROTEM results
- Monitor short term safety of patients whose dose has been modified.
Secondary objectives
- Perform thrombin generation assays to provide additional evidence supporting the TEG/ROTEM-guided dosing.
- Obtain preliminary data on the economic impact of TEG/ROTEM-guided hemophilia A dosing regimens.
- Obtain preliminary data on the potential improvement of subject/family burden resulting from TEG/ROTEM-guided regimen using generic and specific quality of life tools.
The study period will consist of screening, pharmacokinetic (PK) study and follow-up. For subjects whose prophylaxis treatment regimen is modified, the follow-up period will include 3 follow-up clinic visits and 4 follow-up phone calls; these patients will remain on study for approximately 6 months following completion of the PK-study then will have an end of the study visit (visit 9). Subjects whose treatment regimen is not altered will end their study participation after Visit 2 and continuing with their current prophylaxis regimen.
Study endpoints: A safety review will be conducted after the first 6 subjects complete 30 days with the extended prophylaxis treatment. If no safety issues are identified, the study will proceed, and second safety review will be after an additional 8 subjects complete 30 days of extended treatment. If a subject has two or more spontaneous bleeds in 30 days, the subject will be removed from the study. For subjects who did not have bleeds and completed at least 6 months of the study, ongoing prophylactic treatment will be decided by their treating physician.
- Conditions
- Hemophilia A
- Being tested
- Thromboelastography/Thromboelastometry-Guided Treatment Regimen
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Thromboelastography/Thromboelastometry-Guided Treatment RegimenOther
The results of the TEG/ROTEM assay (specifically the R time/CT) will be used to determine the prophylaxis treatment regimen.
| Study group | Role | What they receive |
|---|---|---|
| Severe Hemophilia A Subjects | Other | The results of the TEG/ROTEM assay (specifically the R time/CT) will be used to determine the prophylaxis treatment regimen. Receives: Other: Thromboelastography/Thromboelastometry-Guided Treatment Regimen |
What is being measured
The primary outcome is the main question the study is designed to answer.
Feasibility of TEG/ROTEM-guided prophylaxis modification for patients, assessed by estimated proportion of patients whose dose is modified.
A total of 60 patients will be enrolled on this study. It is expected that less than 25% of these patients will be eligible for dose/schedule modification based on TEG/ROTEM.
Measured: Ongoing while patients are on study (~ 6 months)
Monitoring short-term safety of patients whose dose has been modified, assessed by monitoring number, type, and severity of bleeds.
The primary endpoint for subject safety will be the occurrence of two serious spontaneous bleeding episodes within 28 days of each other.
Measured: Ongoing while patients are on study (~ 6 months)
Feasibility of TEG/ROTEM-guided prophylaxis modification for patients, assessed by testing and refining operational protocol for using TEG/ROTEM to guide factor dosing.
Measured: Ongoing while patients are on study (~ 6 months)
Secondary outcomes (4)
Thrombin generation assay will be performed to provide additional evidence supporting the TEG/ROTEM-guided dosing.
Measured: 6 months
Assessment of direct costs for all subjects whose treatment was modified using factor consumption 6 months before, and the 6 months during study participation.
Analysis for those subjects who had a modified dosing regimen to determine the per capita reduction in consumption when a modified dosing regimen is achieved.
Measured: 1 year
Assessment of indirect costs for all subjects whose treatment was modified using factor consumption 6 months before, and the 6 months during study participation.
Questionnaire collecting data on time spend on hemophilia-related care per week, caregiver support related to hemophilia, and number of infusions per week.
Measured: 6 months
Improvement of subject/family burden as assessed by the Health-Related Quality of Life.
(HRQoL) Assessments/Analysis
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted21 Oct 2015 (estimated)
- Study start20 May 2014
- Primary completion31 Dec 2017
- Study completion31 Dec 2017
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.
- 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Los Angeles
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Guy Young, Children's Hospital Los Angeles
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02582060
- Sponsor's protocol ID
- 14-00030
- Status last verified
- Feb 2019
- First submitted
- 9 Oct 2015
- Last updated
- 18 Feb 2019
- Results posted
- No
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 18 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Individualizing Hemophilia Prophylaxis Using Thromboelastography
- ClinicalTrials.gov ID
- NCT02582060
- Acronym / sponsor ID
- 14-00030
- Conditions
- Hemophilia A
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Children's Hospital Los AngelesUniversity, hospital or other
- Study start
- 20 May 2014 (actual)
- Primary completion
- 31 Dec 2017 (actual)
- Study completion
- 31 Dec 2017 (actual)
- First posted
- 21 Oct 2015 (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.
Currently dosing for prophylaxis is not individualized, and the general approach is to use a dose of 25-40 units/kg given 3 times per week or every other day. One of the issues with weight-based dosing is the possible over-treatment. This is likely due to the fact that laboratory tests are not sensitive enough at the low levels to support decision-making. The Thromboelastograph (TEG®) and Thromboelastometry (ROTEM®) are coagulation devices, which assess the dynamics of clot formation over time and have several characteristics which suggest they may provide important information for individualized prophylaxis treatment for our patients.
Detailed description
This is a pilot study in children and adults with severe hemophilia A that utilizes the TEG/ROTEM to personalize their prophylaxis treatment.
Primary objectives:
- Assess the feasibility of TEG/ROTEM-guided modification of the current prophylaxis regimen for individuals with severe hemophilia A. Specifically, to:
- Test and refine the operational protocol for using TEG/ROTEM to guide factor dosing
- Estimate the proportion of hemophilia A patients whose dose will be modified based on TEG/ROTEM results
- Monitor short term safety of patients whose dose has been modified.
Secondary objectives
- Perform thrombin generation assays to provide additional evidence supporting the TEG/ROTEM-guided dosing.
- Obtain preliminary data on the economic impact of TEG/ROTEM-guided hemophilia A dosing regimens.
- Obtain preliminary data on the potential improvement of subject/family burden resulting from TEG/ROTEM-guided regimen using generic and specific quality of life tools.
The study period will consist of screening, pharmacokinetic (PK) study and follow-up. For subjects whose prophylaxis treatment regimen is modified, the follow-up period will include 3 follow-up clinic visits and 4 follow-up phone calls; these patients will remain on study for approximately 6 months following completion of the PK-study then will have an end of the study visit (visit 9). Subjects whose treatment regimen is not altered will end their study participation after Visit 2 and continuing with their current prophylaxis regimen.
Study endpoints: A safety review will be conducted after the first 6 subjects complete 30 days with the extended prophylaxis treatment. If no safety issues are identified, the study will proceed, and second safety review will be after an additional 8 subjects complete 30 days of extended treatment. If a subject has two or more spontaneous bleeds in 30 days, the subject will be removed from the study. For subjects who did not have bleeds and completed at least 6 months of the study, ongoing prophylactic treatment will be decided by their treating physician.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Severe Hemophilia A Subjects | Other | Other: Thromboelastography/Thromboelastometry-Guided Treatment RegimenThe results of the TEG/ROTEM assay (specifically the R time/CT) will be used to determine the prophylaxis treatment regimen. |
Who may qualify
- Ages
- 5 to 70 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (4 listed)
- Males, 5-70 years, inclusive
- Plasma FVIII activity <1% documented (Laboratory result or MD documentation of Severe Hemophilia A diagnosis)
- Currently prescribed prophylaxis treatment regimen infusing ≥3 times a week
- Willing to alter their prophylaxis treatment regimen per study protocol
Key exclusion criteria (9 listed)
- Bleeding disorder(s) other than Hemophilia A
- Current inhibitor (>0.6BU)
- Thrombocytopenia (platelet count <100,000K/µL since it can alter TEG®/ROTEM® results)
- Creatinine >2x the upper limit of normal (indicating potential platelet dysfunction)
- Prothrombin time >3 seconds above the upper limit of normal (indicating potential liver dysfunction)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Thromboelastography/Thromboelastometry-Guided Treatment RegimenThe results of the TEG/ROTEM assay (specifically the R time/CT) will be used to determine the prophylaxis treatment regimen. | Other | Severe Hemophilia A Subjects |
Primary outcome
Feasibility of TEG/ROTEM-guided prophylaxis modification for patients, assessed by estimated proportion of patients whose dose is modified.
Time frame: Ongoing while patients are on study (~ 6 months)
A total of 60 patients will be enrolled on this study. It is expected that less than 25% of these patients will be eligible for dose/schedule modification based on TEG/ROTEM.
Monitoring short-term safety of patients whose dose has been modified, assessed by monitoring number, type, and severity of bleeds.
Time frame: Ongoing while patients are on study (~ 6 months)
The primary endpoint for subject safety will be the occurrence of two serious spontaneous bleeding episodes within 28 days of each other.
Feasibility of TEG/ROTEM-guided prophylaxis modification for patients, assessed by testing and refining operational protocol for using TEG/ROTEM to guide factor dosing.
Time frame: Ongoing while patients are on study (~ 6 months)
4 secondary outcomes
Thrombin generation assay will be performed to provide additional evidence supporting the TEG/ROTEM-guided dosing.
Time frame: 6 months
Assessment of direct costs for all subjects whose treatment was modified using factor consumption 6 months before, and the 6 months during study participation.
Time frame: 1 year
Assessment of indirect costs for all subjects whose treatment was modified using factor consumption 6 months before, and the 6 months during study participation.
Time frame: 6 months
Improvement of subject/family burden as assessed by the Health-Related Quality of Life.
Time frame: 6 months
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 18 Feb 2019.
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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?
A study testing Thromboelastography/Thromboelastometry-Guided Treatment Regimen in people with Hemophilia A, run by Children's Hospital Los Angeles 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 9 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 18 Feb 2019. 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?
Feasibility of TEG/ROTEM-guided prophylaxis modification for patients, assessed by estimated proportion of patients whose dose is modified. (time frame: Ongoing while patients are on study (~ 6 months)). The study lists 3 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 18 Feb 2019.