HemophiliaNCT02453542
Global Haemostatic Methods Following Administration of Bypassing Agents to Patients With Haemophilia With Inhibitors
An observational study of Hemophilia, run by Karolinska Institutet at 1 site in Sweden.
Trial summary
Background The treatment of haemophilia A and B has been revolutionized by the use of factor concentrate, both as prophylaxis and to treat bleeding episodes (on-demand treatment). Read more about this study
- Who is it for?
- Ages 7 years and older, men only
- Conditions the study is for
- Hemophilia
- How many people
- 20 participants
- Where
- 1 siteSweden
- Study dates
- 1 Mar 2015to Aug 2030
- How long will I take part?
- 2 Years
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 7 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Fifteen patients (adults and children) with hereditary haemophilia with inhibitors. Five patients (adults) with acquired haemophilia
Who may be able to join
- informed consent
- meets the study population description
Who cannot join
- no informed consent age<7 years
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Sweden1 site, 1 recruiting
- Recruiting
Karolinska University Hospital
Solna, Stockholm County, 17176
Roza Chaireti, MD, PhD, +46 738517974, roza.chaireti@ki.se
Margareta Holmström, MD, Assoc Prof, +46 8 5177 0000, margareta.holmstrom@ki.se
About this study
Official title: Global Haemostatic Methods to Measure the Treatment Effect Following Administration of Bypassing Agents to Patients With Haemophilia With Inhibitors
Background
The treatment of haemophilia A and B has been revolutionized by the use of factor concentrate, both as prophylaxis and to treat bleeding episodes (on-demand treatment). However, despite its advantages, repeated treatment with factor concentrate can lead to development of inhibitors (antibodies) towards the coagulation factor in the concentrate. Another patient group in which the bleeding symptoms are difficult to treat because of inhibitors towards coagulation factors, most commonly FVIII, is patients with acquired haemophilia. Patients with high antibody titers exhibit a deficient or no response to factor concentrates and usually need treatment with bypassing agents, namely factor eight inhibitor bypassing agent (FEIBA®, Baxter) och recombinant activated factor VII (rFVIIa, Novo-Seven®, Novo Nordisk). The effect of the treatment cannot be accurately monitored by traditional coagulation tests.
Read more
The aim of the study is to evaluate the utility of the global haemostatic methods in patients with haemophilia with inhibitors. The objective is to improve the monitoring of the treatment effect and thus increase the safety of the patient and the effectiveness of the treatment.
Patients and methods
Patients
The primary cohort will consist of fifteen patients with inherited haemophilia with inhibitors as well as five adult patients with acquired haemophilia who are followed up at the Coagulation Department of the Karolinska University Hospital, Stockholm, Sweden.
Blood samples will be collected from those patients at specific time points (see Design of the study) during the course of two years (for each patient). The treatment (type, dose, duration) will be determined by the treating physician.
Methods (selection)
- Thrombin generation (Calibrated Automated Thrombogram, CAT® and a commercial kit from Siemens®).
- Overall haemostatic potential (OHP)
Design of the study
Timeframe for blood sampling: i) baseline (inclusion in the study), and ii) prior and after administration of bypassing agents to either treat bleeding symptoms or before an invasive procedure or as prophylaxis.
Data analysis
The variations in coagulation markers measured as described above (Methods) will be associated to the clinical symptoms (bleeding), the level of coagulation factors (if measurable) and the titers of the inhibitors.
- Conditions
- Hemophilia
- Keywords
- hemophilia, inhibitors, thrombin
- Medical subject headings
- Hemophilia A
What is being measured
The primary outcome is the main question the study is designed to answer.
Changes in coagulation markers, such as thrombin generation markers (Endogenous Thrombin Potential in nano molar thrombin*minute and peak thrombin in nano molar and fibrin aggregation curves, following administration of bypassing agents.) composite
Measured: participants will be followed up for up to 2 years following inclusion
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 May 2015 (estimated)
- Study start1 Mar 2015
- Primary completionJun 2030 (estimated)
- Study completionAug 2030 (estimated)
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 Karolinska InstitutetThe 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 20 participants (estimated)
- Target duration
- 2 Years
Sponsor and investigators
- Lead sponsor
- Karolinska Institutet
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Roza Chaireti, Karolinska Institutet
- Investigators
- Roza Chaireti, MD, PhD, Principal investigator, Karolinska Institutet
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02453542
- Sponsor's protocol ID
- 202100-2973
- Status last verified
- Aug 2025
- First submitted
- 17 May 2015
- Last updated
- 2 Sep 2025
- Results posted
- No
- Data monitoring committee
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Sep 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Global Haemostatic Methods to Measure the Treatment Effect Following Administration of Bypassing Agents to Patients With Haemophilia With Inhibitors
- ClinicalTrials.gov ID
- NCT02453542
- Acronym / sponsor ID
- 202100-2973
- Conditions
- Hemophilia
- Keywords
- hemophilia, inhibitors, thrombin
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 20 participants (estimated)
- Lead sponsor
- Karolinska InstitutetUniversity, hospital or other
- Study start
- 1 Mar 2015 (actual)
- Primary completion
- Jun 2030 (estimated)
- Study completion
- Aug 2030 (estimated)
- First posted
- 25 May 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.
Background
The treatment of haemophilia A and B has been revolutionized by the use of factor concentrate, both as prophylaxis and to treat bleeding episodes (on-demand treatment). However, despite its advantages, repeated treatment with factor concentrate can lead to development of inhibitors (antibodies) towards the coagulation factor in the concentrate. Another patient group in which the bleeding symptoms are difficult to treat because of inhibitors towards coagulation factors, most commonly FVIII, is patients with acquired haemophilia. Patients with high antibody titers exhibit a deficient or no response to factor concentrates and usually need treatment with bypassing agents, namely factor eight inhibitor bypassing agent (FEIBA®, Baxter) och recombinant activated factor VII (rFVIIa, Novo-Seven®, Novo Nordisk). The effect of the treatment cannot be accurately monitored by traditional coagulation tests.
The aim of the study is to evaluate the utility of the global haemostatic methods in patients with haemophilia with inhibitors. The objective is to improve the monitoring of the treatment effect and thus increase the safety of the patient and the effectiveness of the treatment.
Patients and methods
Patients
The primary cohort will consist of fifteen patients with inherited haemophilia with inhibitors as well as five adult patients with acquired haemophilia who are followed up at the Coagulation Department of the Karolinska University Hospital, Stockholm, Sweden.
Blood samples will be collected from those patients at specific time points (see Design of the study) during the course of two years (for each patient). The treatment (type, dose, duration) will be determined by the treating physician.
Methods (selection)
- Thrombin generation (Calibrated Automated Thrombogram, CAT® and a commercial kit from Siemens®).
- Overall haemostatic potential (OHP)
Design of the study
Timeframe for blood sampling: i) baseline (inclusion in the study), and ii) prior and after administration of bypassing agents to either treat bleeding symptoms or before an invasive procedure or as prophylaxis.
Data analysis
The variations in coagulation markers measured as described above (Methods) will be associated to the clinical symptoms (bleeding), the level of coagulation factors (if measurable) and the titers of the inhibitors.
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Target follow-up
- 2 Years
Who may qualify
- Ages
- 7 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Fifteen patients (adults and children) with hereditary haemophilia with inhibitors. Five patients (adults) with acquired haemophilia
Key inclusion criteria (2 listed)
- informed consent
- meets the study population description
Key exclusion criteria (1 listed)
- no informed consent age<7 years
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
Changes in coagulation markers, such as thrombin generation markers (Endogenous Thrombin Potential in nano molar thrombin*minute and peak thrombin in nano molar and fibrin aggregation curves, following administration of bypassing agents.) composite
Time frame: participants will be followed up for up to 2 years following inclusion
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Sep 2025.
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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.
Roza Chaireti, MD, PhD principal investigator
Karolinska Institutet
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 Karolinska Institutet at 1 site in Sweden.
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: looking for participants now, as of the sponsor's last update on 2 Sep 2025. 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 Sweden. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Roza Chaireti, MD, PhD (+46 738 5170974, roza.chaireti@ki.se), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Changes in coagulation markers, such as thrombin generation markers (Endogenous Thrombin Potential in nano molar thrombin*minute and peak thrombin in nano molar and fibrin aggregation curves, following administration of bypassing agents.) composite (time frame: participants will be followed up for up to 2 years following inclusion). 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 2 Sep 2025.