Hemophilia A With InhibitorNCT06357572
Real-life Clinical FEIBA Samples Measured Using the Version A of the HemA EnzySystem
An observational study of Hemophilia A With Inhibitor and Hemophilia A, run by Enzyre B.V. at 1 site in Poland.
Trial summary
The goal of this observational study is to assess if the version A of the HemA EnzySystem, a novel portable coagulation testing platform, can be used in patients with hemophilia A treated with Factor VIII Bypassing Agent (FEIBA). Read more about this study
- Who is it for?
- Ages 18 years and older, men only
- Conditions the study is for
- Hemophilia A With Inhibitor, Hemophilia A
- What is being tested?
- Thrombin generation assay (EnzySystem HemA version A - whole blood), Thrombin generation assay (Ceveron s100 (Technoclone) - fresh plasma) and Additional coagulation tests
- How many people
- 6 participants
- Where
- 1 sitePoland
- Study dates
- 26 Mar 2024to 27 Mar 2024
- 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 18 years and older
- Men only
- No healthy volunteers
- Adult, Older adult
Study population: Hemophilia A patients at the Institute of Hematology and Transfusion Medicine who are on prophylactic or on-demand FEIBA treatment.
Who may be able to join
- Diagnosed with mild (FVIII activity levels 5-40%), moderate (FVIII activity levels 1-5%) or severe hemophilia A (FVIII activity levels <1%)
- With inhibitors
- On prophylactic or on-demand treatment with FEIBA (variation in dosing and duration of therapy use)
Who cannot join
- use of anticoagulants or platelet antagonists (aspirin or any TAR);
- known allergy to stainless steel;
- a bleeding episode within the last two weeks;
- clinical indication of liver cirrhosis (ultrasonography indication, enlarged spleen, decreased platelet count <100 G/l);
- Signs of inflammation or infection
- Current use of:
- NSAIDs; antimicrobial medication; thyroid inhibitors or SSRI's;
Trial locations and contacts
1 site in 1 country.
Poland1 site
Institute of Hematology and Blood Transfusion
Warsaw, Mazowieckie Województwo
About this study
The goal of this observational study is to assess if the version A of the HemA EnzySystem, a novel portable coagulation testing platform, can be used in patients with hemophilia A treated with Factor VIII Bypassing Agent (FEIBA). The main question[s] it aims to answer are:
- Can the version A of the HemA EnzySystem can record thrombin generation within a time frame of 60 min in fresh whole blood samples of patients with hemophilia A treated with FEIBA?
- Are the TGA results of the version A of the HemA EnzySystem in agreement with the TGA results obtained with conventional methods in fresh non frozen plasma?
Participants are asked to fill in a questionnaire regarding their general health and hemophilia treatment. Subsequently, blood will be drawn from the patients before, and at 30, 120, and 240 minutes after FEIBA administration. Whole blood is immediately tested using the Version A HemA EnzySystem, and plasma is generated for testing with the Ceveron s100 (Technoclone). Leftover samples are frozen for later additional coagulation testing.
Read the detailed description
Rationale: Hemophilia is an X-linked hemostatic bleeding disorder characterized by a lack of coagulation factor VIII activity (HemA) or factor IX activity (HemB). Spontaneous major bleeding in joints and muscles frequently occur when factor activity levels are low. Currently, there is a growing evidence that the bleeding phenotype of hemophilia A patients is not only reflected by the actual factor VIII activity level, as there is a large variety in bleeding among patients with similar factor VIII activity.
Currently, patients with severe Hemophilia A are treated with either FVIII containing products or Emicizumab. Initially emicizumab was described for patients who developed inhibitors as inhibitors do not interfere with emicizumab. Despite, bleeding complications still may occur and therefore bypassing therapies are still of great importance. Unfortunately, monitoring treatment with these drugs is a challenge.
Thrombin generation profile may be an additional tool in hemophilia patients to differentiate between bleeding phenotype, to guide prophylactic replacement therapy and adjust bypassing therapy, especially in patients treated with emicizumab. Based on recent studies, thrombin generation as a global hemostasis assay offers an opportunity to assess the hemostatic capacity of patients, and therefore has much potential for monitoring therapy. To facilitate this, a hand-held in vitro diagnostic medical device able to simultaneously measure multiple disease biomarkers with a single drop of blood is currently in development, focusing on simultaneous measurements of Factor VIII activity and Thrombin generation.
Objective: The primary objective of this study is to demonstrate that the version A of the HemA EnzySystem can record thrombin generation within a time frame of 60 min in fresh whole blood samples of patients with hemophilia A treated with Factor VIII Bypassing Agent (FEIBA). As secondary objective, the study results will be validated with a Thrombin Generation Assay (TGA) measured in freshly obtained non frozen plasma.
Study design: This is a cross-sectional observational study. All participants are asked to fill a questionnaire prior to blood collection of four tubes. Blood sample measurements will be conducted within a two-hour time frame using the version A of the HemA EnzySystem and the preparation of plasma using a standard TGA assay. Any remaining blood material will be processed and stored.
Study population: The study population consists of 6 patients treated with FEIBA. In total, at least 24 samples are tested, four for each patient (t=0, 30, 120 and 240 minutes).
- Conditions
- Hemophilia A With Inhibitor, Hemophilia A
- Being tested
- Thrombin generation assay (EnzySystem HemA version A - whole blood), Thrombin generation assay (Ceveron s100 (Technoclone) - fresh plasma), Additional coagulation tests
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Thrombin generation assay (EnzySystem HemA version A - whole blood)Diagnostic test
Whole blood obtained by venipuncture is immediately tested with the EnzySystem HemA version A), before and 0, 30, 120 and 240 minutes after administration of FEIBA.
Thrombin generation assay (Ceveron s100 (Technoclone) - fresh plasma)Diagnostic test
Whole blood is centrifuged to obtain plasma. The fresh plasma is tested using the Ceveron s100.
Additional coagulation testsDiagnostic test
Plasma samples are frozen for later coagulation testing. The following tests are performed:
Factor VIII activity von Willebrand Factor antigen levels von Willebrand Factor ristocetin activity levels Prothrombin Fragment 1+2 levels ADAMTS13 activity FVIII antigen levels Nijmegen Hemostasis Assay
| Study group | Role | What they receive |
|---|---|---|
| Hemophilia A patients prophylactically treated with FEIBA | Not described Receives: Diagnostic Test: Thrombin generation assay (EnzySystem HemA version A - whole blood); Diagnostic Test: Thrombin generation assay (Ceveron s100 (Technoclone) - fresh plasma); Diagnostic Test: Additional coagulation tests |
What is being measured
The primary outcome is the main question the study is designed to answer.
Time between venipuncture and TGA result
The primary objective of this study is to demonstrate that the EnzySystem Thrombin Generation assay can record thrombin generation within a time frame of 60 min. Fresh whole blood samples of patients with hemophilia A on prophylactic or on-demand FEIBA treatment are used and tested in a near-patient setting at the Institute of Hematology and Transfusion Medicine (IHiT)
Measured: 2 hours
Secondary outcomes (1)
Agreement between TGA obtained with the EnzySystem HemA version A and conventional TGA results
Outcome of the TGAlum measured with the version A of the HemA EnzySystem will be compared to the reference TGA which is the standard method of testing at the IHiT to determine patients coagulation status. The reference TGA will be evaluated using the Ceveron by Technoclone
Measured: 6 hours
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted10 Apr 2024
- Study start26 Mar 2024
- Primary completion27 Mar 2024
- Study completion27 Mar 2024
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia A With InhibitorStudies 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
- 6 participants (actual)
Sponsor and investigators
- Lead sponsor
- Enzyre B.V.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Instytut Hematologii i Transfuzjologii, Warschau, Poland
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06357572
- Sponsor's protocol ID
- ENZ13-2024
- Status last verified
- Apr 2024
- First submitted
- 5 Apr 2024
- Last updated
- 10 Apr 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Real-life Clinical FEIBA Samples Measured Using the Version A of the HemA EnzySystem
- ClinicalTrials.gov ID
- NCT06357572
- Acronym / sponsor ID
- ENZ13-2024
- Conditions
- Hemophilia A With Inhibitor, Hemophilia A
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 6 participants (actual)
- Lead sponsor
- Enzyre B.V.Industry (pharma, biotech, devices)
- Collaborators
- Instytut Hematologii i Transfuzjologii, Warschau, Poland
- Study start
- 26 Mar 2024 (actual)
- Primary completion
- 27 Mar 2024 (actual)
- Study completion
- 27 Mar 2024 (actual)
- First posted
- 10 Apr 2024 (actual)
- 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.
The goal of this observational study is to assess if the version A of the HemA EnzySystem, a novel portable coagulation testing platform, can be used in patients with hemophilia A treated with Factor VIII Bypassing Agent (FEIBA). The main question[s] it aims to answer are:
- Can the version A of the HemA EnzySystem can record thrombin generation within a time frame of 60 min in fresh whole blood samples of patients with hemophilia A treated with FEIBA?
- Are the TGA results of the version A of the HemA EnzySystem in agreement with the TGA results obtained with conventional methods in fresh non frozen plasma?
Participants are asked to fill in a questionnaire regarding their general health and hemophilia treatment. Subsequently, blood will be drawn from the patients before, and at 30, 120, and 240 minutes after FEIBA administration. Whole blood is immediately tested using the Version A HemA EnzySystem, and plasma is generated for testing with the Ceveron s100 (Technoclone). Leftover samples are frozen for later additional coagulation testing.
Detailed description
Rationale: Hemophilia is an X-linked hemostatic bleeding disorder characterized by a lack of coagulation factor VIII activity (HemA) or factor IX activity (HemB). Spontaneous major bleeding in joints and muscles frequently occur when factor activity levels are low. Currently, there is a growing evidence that the bleeding phenotype of hemophilia A patients is not only reflected by the actual factor VIII activity level, as there is a large variety in bleeding among patients with similar factor VIII activity.
Currently, patients with severe Hemophilia A are treated with either FVIII containing products or Emicizumab. Initially emicizumab was described for patients who developed inhibitors as inhibitors do not interfere with emicizumab. Despite, bleeding complications still may occur and therefore bypassing therapies are still of great importance. Unfortunately, monitoring treatment with these drugs is a challenge.
Thrombin generation profile may be an additional tool in hemophilia patients to differentiate between bleeding phenotype, to guide prophylactic replacement therapy and adjust bypassing therapy, especially in patients treated with emicizumab. Based on recent studies, thrombin generation as a global hemostasis assay offers an opportunity to assess the hemostatic capacity of patients, and therefore has much potential for monitoring therapy. To facilitate this, a hand-held in vitro diagnostic medical device able to simultaneously measure multiple disease biomarkers with a single drop of blood is currently in development, focusing on simultaneous measurements of Factor VIII activity and Thrombin generation.
Objective: The primary objective of this study is to demonstrate that the version A of the HemA EnzySystem can record thrombin generation within a time frame of 60 min in fresh whole blood samples of patients with hemophilia A treated with Factor VIII Bypassing Agent (FEIBA). As secondary objective, the study results will be validated with a Thrombin Generation Assay (TGA) measured in freshly obtained non frozen plasma.
Study design: This is a cross-sectional observational study. All participants are asked to fill a questionnaire prior to blood collection of four tubes. Blood sample measurements will be conducted within a two-hour time frame using the version A of the HemA EnzySystem and the preparation of plasma using a standard TGA assay. Any remaining blood material will be processed and stored.
Study population: The study population consists of 6 patients treated with FEIBA. In total, at least 24 samples are tested, four for each patient (t=0, 30, 120 and 240 minutes).
Study design
- Observational model
- Case only
- Time perspective
- Cross-sectional
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Hemophilia A patients prophylactically treated with FEIBA | – | Diagnostic Test: Thrombin generation assay (EnzySystem HemA version A - whole blood), Diagnostic Test: Thrombin generation assay (Ceveron s100 (Technoclone) - fresh plasma), Diagnostic Test: Additional coagulation tests |
Who may qualify
- Ages
- 18 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Hemophilia A patients at the Institute of Hematology and Transfusion Medicine who are on prophylactic or on-demand FEIBA treatment.
Key inclusion criteria (3 listed)
- Diagnosed with mild (FVIII activity levels 5-40%), moderate (FVIII activity levels 1-5%) or severe hemophilia A (FVIII activity levels <1%)
- With inhibitors
- On prophylactic or on-demand treatment with FEIBA (variation in dosing and duration of therapy use)
Key exclusion criteria (7 listed)
- use of anticoagulants or platelet antagonists (aspirin or any TAR);
- known allergy to stainless steel;
- a bleeding episode within the last two weeks;
- clinical indication of liver cirrhosis (ultrasonography indication, enlarged spleen, decreased platelet count <100 G/l);
- Signs of inflammation or infection
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 |
|---|---|---|
| Thrombin generation assay (EnzySystem HemA version A - whole blood)Whole blood obtained by venipuncture is immediately tested with the EnzySystem HemA version A), before and 0, 30, 120 and 240 minutes after administration of FEIBA. | Diagnostic test | Hemophilia A patients prophylactically treated with FEIBA |
| Thrombin generation assay (Ceveron s100 (Technoclone) - fresh plasma)Whole blood is centrifuged to obtain plasma. The fresh plasma is tested using the Ceveron s100. | Diagnostic test | Hemophilia A patients prophylactically treated with FEIBA |
| Additional coagulation testsPlasma samples are frozen for later coagulation testing. The following tests are performed: Factor VIII activity von Willebrand Factor antigen levels von Willebrand Factor ristocetin activity levels Prothrombin Fragment 1+2 levels ADAMTS13 activity FVIII antigen levels Nijmegen Hemostasis Assay | Diagnostic test | Hemophilia A patients prophylactically treated with FEIBA |
Primary outcome
Time between venipuncture and TGA result
Time frame: 2 hours
The primary objective of this study is to demonstrate that the EnzySystem Thrombin Generation assay can record thrombin generation within a time frame of 60 min. Fresh whole blood samples of patients with hemophilia A on prophylactic or on-demand FEIBA treatment are used and tested in a near-patient setting at the Institute of Hematology and Transfusion Medicine (IHiT)
1 secondary outcome
Agreement between TGA obtained with the EnzySystem HemA version A and conventional TGA results
Time frame: 6 hours
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 Apr 2024.
- Individual participant dataSharing planned: No
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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 A With Inhibitor and Hemophilia A, run by Enzyre B.V. at 1 site in Poland.
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 7 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 Apr 2024. 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 Poland. 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?
Time between venipuncture and TGA result (time frame: 2 hours). 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 Apr 2024.