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

Hemophilia Clinical Trial

Official title:

Global Haemostatic Methods to Measure the Treatment Effect Following Administration of Bypassing Agents to Patients With Haemophilia With Inhibitors

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02453542
• Other study ID #: 202100-2973
• Status: Recruiting
• Start date: March 2015
• Est. completion date: August 2030

Study information

• Verified date: October 2023
• Source: Karolinska Institutet
• Contact: Roza Chaireti, MD, PhD
• Phone: +46 738 5170974
• Email: roza.chaireti[@]ki.se
• Is FDA regulated: No
• Study type: Observational [Patient Registry]

Clinical 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). 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 20
• Est. completion date: August 2030
• Est. primary completion date: June 2030
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 7 Years and older

Eligibility

Inclusion Criteria:

• informed consent

• meets the study population description

Exclusion Criteria:

• no informed consent age<7 years

Related Conditions & MeSH terms

• Hemophilia
• Hemophilia A

Locations

Country	Name	City	State
Sweden	Karolinska University Hospital	Solna	Stockholm

Sponsors (1)

Lead Sponsor	Collaborator
Karolinska Institutet

Country where clinical trial is conducted

Sweden, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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		participants will be followed up for up to 2 years following inclusion