Haemophilia AHOPENCT02781766
TGA (Thrombin Generation Assay) and Prophylaxis in Haemophilia
A study testing Thrombin generation measurement. in people with Haemophilia A, run by Hospices Civils de Lyon at 7 sites in France.
Trial summary
Haemophilic arthropathy is one of the major complications of severe haemophilia. In order to maintain plasma clotting factor activity levels above 1% and avoid spontaneous joint bleeds and other serious bleeding events, prophylactic factor replacement therapy is used. Read more about this study
- Who is it for?
- Ages 2 to 45 years, men only
- Conditions the study is for
- Haemophilia A
- What is being tested?
- Thrombin generation measurement.
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 50 participants
- Where
- 7 sitesFrance
- Study dates
- 8 Aug 2016to 8 Sep 2019
- 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 2 to 45 years
- Men only
- No healthy volunteers
- Child, Adult
Who may be able to join
- Severe haemophilia A (FVIII < 1 IU/dl)
- Currently on prophylactic therapy
- The same prophylaxis regimen in the last six months
- Age: 2 (with a body weight ≥12.5 kg ) - 45 years
- Adequate venous access
- Haemophilia patient's diary or equivalent regularly completed
- Ability of patient or family (for minors) to give informed consent
- Subject with health insurance
Who cannot join
- Age <2 and >45 years
- Haemophilia A with documented inhibitor in the last 12 months
- Clinically symptomatic liver disease (historical evidence documented in patient's medical file)
- Platelet count < 100x109/l
- Poor venous access
- Presence of a documented target joint
- Subject under legal protection measure.
Trial locations and contacts
7 sites in 1 country.
7 sites in 1 country
France7 sites, 7 recruiting
- Not yet recruiting
Hôpital Pellegrin
Bordeaux, 33076
Sabine CASTET, 05 56 79 48 79 ext. +33, sabine-marie.castet@chu-bordeaux.fr
Sabine CASTET
- Recruiting
Unité d'Hémostase Clinique Centre Régional de Traitement des Hémophiles Centre de Référence de Maladies Hémorragiques Hôpital Cardiologique Louis Pradel
Bron, 69500
Yesim DARGAUD, Pr, (0)4.72.11.88.10 ext. +33, ydargaud@univ-lyon1.fr
- Not yet recruiting
CH Metropole Savoie site de Chambéry
Chambéry, 73011
Valérie GAY, 04 72 96 56 65 ext. +33, valerie.gay@ch-metropole-savoie.fr
Valérie GAY
- Not yet recruiting
CHU Dijon Bourgogne Hôpital François Mitterrand
Dijon, 21079
Fabienne GENRE VOLOT, 03 80 29 32 57 ext. +33, fabienne.volot@chu-dijon.fr
Fabienne GENRE VOLOT
- Not yet recruiting
Chu Timone
Marseille, 13385
Hervé CHAMBOST, 04 91 38 67 78 ext. +33, herve.chambost@ap-hm.fr
Hervé CHAMBOST
- Recruiting
CHU de Nantes
Nantes, 44093
Marc TROSSAERT, 04 20 08 74 68 ext. +33, marc.trossaert@chu-nantes.fr
Marc TROSSAERT
- Not yet recruiting
CHU Saint Etienne - Hôpital Nord
Saint-Etienne, 42055
Brigitte TARDY, 04 77 12 78 63 ext. +33, brigitte.tardy@chu-st-etienne.fr
Claire BERGER, 04 77 82 88 08 ext. +33, claire.berger@chu-st-etienne.fr
About this study
Official title: Thrombin Generation Capacity of Patients With Severe Haemophilia Receiving Prophylaxis
Haemophilic arthropathy is one of the major complications of severe haemophilia. In order to maintain plasma clotting factor activity levels above 1% and avoid spontaneous joint bleeds and other serious bleeding events, prophylactic factor replacement therapy is used. Because of the high cost and limited availability of clotting factor concentrates, dosing is a crucial issue for prophylaxis therapy. Several studies reported a better correlation between clinical bleeding tendency of patients with haemophilia and thrombin generation assay results compared to FVIII/FIX levels. However there is no specific data showing that thrombin generation may be a better indicator of the clinical efficacy of prophylaxis compared to the conventional FVIII measurement.
The main objective of this open, multicentre, prospective study is to compare trough thrombin generation capacity and FVIII levels in severe haemophilia patients and compare these two laboratory results with:
Read more
- the number of spontaneous haemarthroses and other spontaneous serious bleeds occurred in the last 6 months
- the number of additional FVIII units used in the last 6 months
This project requires no change in term of type of treatment : During the study, each patient will be treated by his usual clotting factor at the usual regimen (frequency and dosage).
The clinical outcome with the usual prophylaxis regimen will be correlated to TGA results
- Conditions
- Haemophilia A
- Being tested
- Thrombin generation measurement.
- Keywords
- Haemophilia A, prophylaxis, thrombin generation assay, factor VIII, prophylactic therapy
- 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 measurement.Other
| Study group | Role | What they receive |
|---|---|---|
| One arm: patients with severe haemophilia A on prophylaxis | Other | Patients with severe haemophilia A (FVIII < 1 IU/dl), currently on prophylactic therapy , having the same prophylaxis regimen in the last six months, aged between 2 (with a body weight ≥12.5 kg ) and 45 years , with adequate venous access, having patient's diary or equivalent regularly completed and able to give informed consent Receives: Other: Thrombin generation measurement. |
What is being measured
The primary outcome is the main question the study is designed to answer.
The correlation between the trough TGA (ETP) results of severe haemophilia patients who are on prophylaxis and the clinical efficacy of the ongoing prophylaxis regimen will be studied
Measured: The clinical efficacy of prophylaxis will be evaluated on the basis of the number of spontaneous joint bleeds the number of other relevant spontaneous bleeds and the consumption of clotting factor concentrate in the last 6 months prior to TGA.
Secondary outcomes (1)
The correlation between the trough FVIII:C results of severe haemophilia patients who are on prophylaxis and the clinical efficacy of the ongoing prophylaxis regimen will be studied
Measured: The clinical efficacy of prophylaxis will be evaluated on the basis of the number of spontaneous joint bleeds the number of other relevant spontaneous bleeds and the consumption of clotting factor concentrate in the last 6 months prior to FVIII.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 May 2016 (estimated)
- Study start8 Aug 2016
- Primary completion8 Sep 2019 (estimated)
- Study completion8 Sep 2019 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Haemophilia AStudies of the same condition, open and under way.
- More trials run by Hospices Civils de LyonThe 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 50 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Hospices Civils de Lyon
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02781766
- Sponsor's protocol ID
- 2012-765
- Acronym
- HOPE
- Status last verified
- Jul 2019
- First submitted
- 20 May 2016
- Last updated
- 10 Jul 2019
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 10 Jul 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Thrombin Generation Capacity of Patients With Severe Haemophilia Receiving Prophylaxis
- ClinicalTrials.gov ID
- NCT02781766
- Acronym / sponsor ID
- HOPE / 2012-765
- Conditions
- Haemophilia A
- Keywords
- Haemophilia A, prophylaxis, thrombin generation assay, factor VIII, prophylactic therapy
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 50 participants (estimated)
- Lead sponsor
- Hospices Civils de LyonUniversity, hospital or other
- Study start
- 8 Aug 2016 (actual)
- Primary completion
- 8 Sep 2019 (estimated)
- Study completion
- 8 Sep 2019 (estimated)
- First posted
- 24 May 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.
Haemophilic arthropathy is one of the major complications of severe haemophilia. In order to maintain plasma clotting factor activity levels above 1% and avoid spontaneous joint bleeds and other serious bleeding events, prophylactic factor replacement therapy is used. Because of the high cost and limited availability of clotting factor concentrates, dosing is a crucial issue for prophylaxis therapy. Several studies reported a better correlation between clinical bleeding tendency of patients with haemophilia and thrombin generation assay results compared to FVIII/FIX levels. However there is no specific data showing that thrombin generation may be a better indicator of the clinical efficacy of prophylaxis compared to the conventional FVIII measurement.
The main objective of this open, multicentre, prospective study is to compare trough thrombin generation capacity and FVIII levels in severe haemophilia patients and compare these two laboratory results with:
- the number of spontaneous haemarthroses and other spontaneous serious bleeds occurred in the last 6 months
- the number of additional FVIII units used in the last 6 months
This project requires no change in term of type of treatment : During the study, each patient will be treated by his usual clotting factor at the usual regimen (frequency and dosage).
The clinical outcome with the usual prophylaxis regimen will be correlated to TGA results
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| One arm: patients with severe haemophilia A on prophylaxis | Other | Other: Thrombin generation measurement.Patients with severe haemophilia A (FVIII < 1 IU/dl), currently on prophylactic therapy , having the same prophylaxis regimen in the last six months, aged between 2 (with a body weight ≥12.5 kg ) and 45 years , with adequate venous access, having patient's diary or equivalent regularly completed and able to give informed consent |
Who may qualify
- Ages
- 2 to 45 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Severe haemophilia A (FVIII < 1 IU/dl)
- Currently on prophylactic therapy
- The same prophylaxis regimen in the last six months
- Age: 2 (with a body weight ≥12.5 kg ) - 45 years
- Adequate venous access
Key exclusion criteria (7 listed)
- Age <2 and >45 years
- Haemophilia A with documented inhibitor in the last 12 months
- Clinically symptomatic liver disease (historical evidence documented in patient's medical file)
- Platelet count < 100x109/l
- Poor venous access
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 measurement. | Other | One arm: patients with severe haemophilia A on prophylaxis |
Primary outcome
The correlation between the trough TGA (ETP) results of severe haemophilia patients who are on prophylaxis and the clinical efficacy of the ongoing prophylaxis regimen will be studied
Time frame: The clinical efficacy of prophylaxis will be evaluated on the basis of the number of spontaneous joint bleeds the number of other relevant spontaneous bleeds and the consumption of clotting factor concentrate in the last 6 months prior to TGA.
1 secondary outcome
The correlation between the trough FVIII:C results of severe haemophilia patients who are on prophylaxis and the clinical efficacy of the ongoing prophylaxis regimen will be studied
Time frame: The clinical efficacy of prophylaxis will be evaluated on the basis of the number of spontaneous joint bleeds the number of other relevant spontaneous bleeds and the consumption of clotting factor concentrate in the last 6 months prior to FVIII.
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 10 Jul 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.
7 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Thrombin generation measurement. in people with Haemophilia A, run by Hospices Civils de Lyon at 7 sites 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 8 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: status not confirmed, as of the sponsor's last update on 10 Jul 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 7 sites 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?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Yesim DARGAUD, Pr ((0)4.72.11.88.10 ext. +33, ydargaud@univ-lyon1.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
The correlation between the trough TGA (ETP) results of severe haemophilia patients who are on prophylaxis and the clinical efficacy of the ongoing prophylaxis regimen will be studied (time frame: The clinical efficacy of prophylaxis will be evaluated on the basis of the number of spontaneous joint bleeds the number of other relevant spontaneous bleeds and the consumption of clotting factor concentrate in the last 6 months prior to TGA.). 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 Jul 2019.