Global Haemostatic Methods Following Administration of Bypassing Agents to Patients With Haemophilia With Inhibitors

NCT ID: NCT02453542

Last Updated: 2025-09-02

Basic Information

• Recruitment Status: RECRUITING
• Total Enrollment: 20 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2015-03-01
• Study Completion Date: 2030-08-31

Brief 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). 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.

Conditions

Hemophilia

Study Design

• Observational Model Type: COHORT
• Study Time Perspective: PROSPECTIVE

Eligibility Criteria

Inclusion Criteria

• informed consent
• meets the study population description

Exclusion Criteria

• no informed consent age<7 years

• Minimum Eligible Age: 7 Years
• Eligible Sex: MALE
• Accepts Healthy Volunteers: No

Sponsors

Karolinska Institutet

OTHER

Sponsor Role: lead

Responsible Party

Roza Chaireti

MD, PhD

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Roza Chaireti, MD, PhD

Role: PRINCIPAL_INVESTIGATOR

Karolinska Institutet

Locations

Karolinska University Hospital

Solna, Stockholm County, Sweden

Site Status: RECRUITING

Countries

Sweden

Central Contacts

Roza Chaireti, MD, PhD

Role: CONTACT

roza.chaireti@ki.se

+46 738 5170974

Jovan Antovic, MD, Assoc Prof

Role: CONTACT

jovan.antovic@ki.se

+46 8 517 75637

Facility Contacts

Roza Chaireti, MD, PhD

Role: primary

roza.chaireti@ki.se

+46 738517974

Margareta Holmström, MD, Assoc Prof

Role: backup

margareta.holmstrom@ki.se

+46 8 5177 0000

Other Identifiers

202100-2973

Identifier Type: -

Identifier Source: org_study_id