Global Haemostatic Methods Following Administration of Bypassing Agents to Patients With Haemophilia With Inhibitors
|
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT02453542 |
|
Recruitment Status :
Recruiting
First Posted : May 25, 2015
Last Update Posted : March 31, 2022
|
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
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.
| Condition or disease |
|---|
| Hemophilia |
| Study Type : | Observational [Patient Registry] |
| Estimated Enrollment : | 20 participants |
| Observational Model: | Cohort |
| Time Perspective: | Prospective |
| Target Follow-Up Duration: | 2 Years |
| Official Title: | Global Haemostatic Methods to Measure the Treatment Effect Following Administration of Bypassing Agents to Patients With Haemophilia With Inhibitors |
| Study Start Date : | March 2015 |
| Estimated Primary Completion Date : | June 2025 |
| Estimated Study Completion Date : | August 2025 |
- 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 ]
Biospecimen Retention: Samples Without DNA
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 7 Years and older (Child, Adult, Older Adult) |
| Sexes Eligible for Study: | Male |
| Accepts Healthy Volunteers: | No |
| Sampling Method: | Probability Sample |
Fifteen patients (adults and children) with hereditary haemophilia with inhibitors.
Five patients (adults) with acquired haemophilia
Inclusion Criteria:
- informed consent
- meets the study population description
Exclusion Criteria:
- no informed consent age<7 years
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02453542
| Contact: Roza Chaireti, MD, PhD | +46 738 5170974 | roza.chaireti@ki.se | |
| Contact: Jovan Antovic, MD, Assoc Prof | +46 8 517 75637 | jovan.antovic@ki.se |
| Sweden | |
| Karolinska University Hospital | Recruiting |
| Solna, Stockholm, Sweden, 17176 | |
| Contact: Roza Chaireti, MD, PhD +46 738517974 roza.chaireti@ki.se | |
| Contact: Margareta Holmström, MD, Assoc Prof +46 8 5177 0000 margareta.holmstrom@ki.se | |
| Principal Investigator: | Roza Chaireti, MD, PhD | Karolinska Institutet |
| Responsible Party: | Roza Chaireti, MD, PhD, Karolinska Institutet |
| ClinicalTrials.gov Identifier: | NCT02453542 |
| Other Study ID Numbers: |
202100-2973 |
| First Posted: | May 25, 2015 Key Record Dates |
| Last Update Posted: | March 31, 2022 |
| Last Verified: | March 2022 |
|
hemophilia inhibitors thrombin |
|
Hemophilia A Blood Coagulation Disorders, Inherited Blood Coagulation Disorders Hematologic Diseases |
Coagulation Protein Disorders Hemorrhagic Disorders Genetic Diseases, Inborn |