HemophiliaNCT05617209
In Vitro Correction of Thrombin Generation by Concizumab (Anti-TFPI) for Severe Hemophilia Patients
An observational study of Hemophilia, run by Centre Hospitalier Universitaire de Saint Etienne at 1 site in France.
Trial summary
Studies have shown, in haemophilia patients, the effectiveness of Anti TFPI antibody (aTFPIAb) to prevent joint and muscular bleeding (tissues poor in Tissue Factor (TF)). However, cases of cerebral thrombosis (tissues rich in TF) have been observed in some patients treated with this antibody. Read more about this study
- Who is it for?
- Ages 12 to 65 years, men only
- Conditions the study is for
- Hemophilia
- What is being tested?
- thrombin generation measurement
- How many people
- 20 participants
- Where
- 1 siteFrance
- Study dates
- 7 Sep 2021to 5 Jan 2023
- 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 12 to 65 years
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Haemophilia A and B patients
Who may be able to join
- Severe or moderate haemophilia A or B patient with FVIII or FIX <5% with or without prophylaxis
- Affiliated or beneficiary of a social security regimen
- Signature of consent
Who cannot join
- Haemophilia patients aged 12 years or less with a weight less than 30 kg (taking into account the volume of blood collection).
- Patients who received factor VIII concentrates less than 48 hours or factor IX concentrates less than 96 hours prior to the routine blood draw at the Hemophilia Clinic. 96 hours prior to the routine blood draw at the Hemophilia Centre
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Tissue Factor Pathway Inhibitor (TFPI) and Haemorrhagic Manifestations in Haemophilia A and B Patients
Studies have shown, in haemophilia patients, the effectiveness of Anti TFPI antibody (aTFPIAb) to prevent joint and muscular bleeding (tissues poor in Tissue Factor (TF)). However, cases of cerebral thrombosis (tissues rich in TF) have been observed in some patients treated with this antibody. Because an inter-individual variation in thrombin generation correction by aTFPIAb, an aTFPIAb concentration effective at low TF concentration could be at the same time thrombogenic at high TF concentration.
Read the detailed description
The aim of this study is to determine, for each patient, the minimum concentration of aTFPIAb necessary to restore thrombin generation in the presence of low (1pM) TF concentration and to evaluate at this concentration of aTFPIAb a potential thrombogenic effect in the presence of a high (10pM) TF concentration.
- Conditions
- Hemophilia
- Being tested
- thrombin generation measurement
- Keywords
- anti-TFPI antibody, thrombin generation, tissue factor concentration
- 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 measurementOther
Thrombin generation will be measured in Platelets rich plasma induced either by 1 pM TF or 10 pM TF concentration in presence of different aTFPIAb concentrations (0; 0.5 µg/ml; 0.75 µg/ml; 1.0 µg/ml; 2.0 µg/ml; 4.0 µg/ml)
| Study group | Role | What they receive |
|---|---|---|
| Severe and/or moderate haemophilia A and B | At the end of the consultation and follow-up of the disease, an additional blood sample of 35 ml (8 tubes) will be taken Receives: Other: thrombin generation measurement |
What is being measured
The primary outcome is the main question the study is designed to answer.
minimum concentration of anti-TFPI antibody
Determine the minimum concentration of anti-TFPI antibody (µg/ml) that normalizes the peak to 1 pM FT
Measured: day 1
Secondary outcomes (1)
Measurement of the clot structure
Measurement of the clot structure (density fibers in g/cm3) by scanning electron microscopy
Measured: day 1
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Nov 2022
- Study start7 Sep 2021
- Primary completion5 Jan 2023
- Study completion5 Jan 2023
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.
- More trials run by Centre Hospitalier Universitaire de Saint EtienneThe 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
- Other
- Time perspective
- Prospective
- Enrolment
- 20 participants (actual)
Sponsor and investigators
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Brigitte Tardy, MD, Principal investigator, Centre Hospitalier Universitaire de Saint Etienne
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05617209
- Sponsor's protocol ID
- 21CH036
- Status last verified
- Nov 2024
- First submitted
- 8 Nov 2022
- Last updated
- 27 Nov 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 27 Nov 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Tissue Factor Pathway Inhibitor (TFPI) and Haemorrhagic Manifestations in Haemophilia A and B Patients
- ClinicalTrials.gov ID
- NCT05617209
- Acronym / sponsor ID
- 21CH036
- Conditions
- Hemophilia
- Keywords
- anti-TFPI antibody, thrombin generation, tissue factor concentration
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 20 participants (actual)
- Lead sponsor
- Centre Hospitalier Universitaire de Saint EtienneUniversity, hospital or other
- Study start
- 7 Sep 2021 (actual)
- Primary completion
- 5 Jan 2023 (actual)
- Study completion
- 5 Jan 2023 (actual)
- First posted
- 15 Nov 2022 (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.
Studies have shown, in haemophilia patients, the effectiveness of Anti TFPI antibody (aTFPIAb) to prevent joint and muscular bleeding (tissues poor in Tissue Factor (TF)). However, cases of cerebral thrombosis (tissues rich in TF) have been observed in some patients treated with this antibody. Because an inter-individual variation in thrombin generation correction by aTFPIAb, an aTFPIAb concentration effective at low TF concentration could be at the same time thrombogenic at high TF concentration.
Detailed description
The aim of this study is to determine, for each patient, the minimum concentration of aTFPIAb necessary to restore thrombin generation in the presence of low (1pM) TF concentration and to evaluate at this concentration of aTFPIAb a potential thrombogenic effect in the presence of a high (10pM) TF concentration.
Study design
- Observational model
- Other
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Severe and/or moderate haemophilia A and B | – | Other: thrombin generation measurementAt the end of the consultation and follow-up of the disease, an additional blood sample of 35 ml (8 tubes) will be taken |
Who may qualify
- Ages
- 12 to 65 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Haemophilia A and B patients
Key inclusion criteria (3 listed)
- Severe or moderate haemophilia A or B patient with FVIII or FIX <5% with or without prophylaxis
- Affiliated or beneficiary of a social security regimen
- Signature of consent
Key exclusion criteria (2 listed)
- Haemophilia patients aged 12 years or less with a weight less than 30 kg (taking into account the volume of blood collection).
- Patients who received factor VIII concentrates less than 48 hours or factor IX concentrates less than 96 hours prior to the routine blood draw at the Hemophilia Clinic. 96 hours prior to the routine blood draw at the Hemophilia Centre
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 measurementThrombin generation will be measured in Platelets rich plasma induced either by 1 pM TF or 10 pM TF concentration in presence of different aTFPIAb concentrations (0; 0.5 µg/ml; 0.75 µg/ml; 1.0 µg/ml; 2.0 µg/ml; 4.0 µg/ml) | Other | Severe and/or moderate haemophilia A and B |
Primary outcome
minimum concentration of anti-TFPI antibody
Time frame: day 1
Determine the minimum concentration of anti-TFPI antibody (µg/ml) that normalizes the peak to 1 pM FT
1 secondary outcome
Measurement of the clot structure
Time frame: day 1
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 27 Nov 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.
Brigitte Tardy, MD principal investigator
Centre Hospitalier Universitaire de Saint Etienne
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, run by Centre Hospitalier Universitaire de Saint Etienne at 1 site in France.
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 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 27 Nov 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 France. 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?
minimum concentration of anti-TFPI antibody (time frame: day 1). 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 27 Nov 2024.