Real-life Clinical FEIBA Samples Measured Using the Version A of the HemA EnzySystem

NCT06357572 | Completed

• 1 other identifier: ENZ13-2024
• Study Type: observational
• Target: 6
• Locations: 1 country
• Sites: 1

Brief Summary

The goal of this observational study is to assess if the version A of the HemA EnzySystem, a novel portable coagulation testing platform, can be used in patients with hemophilia A treated with Factor VIII Bypassing Agent (FEIBA). The main question\[s\] it aims to answer are:

• Can the version A of the HemA EnzySystem can record thrombin generation within a time frame of 60 min in fresh whole blood samples of patients with hemophilia A treated with FEIBA?
• Are the TGA results of the version A of the HemA EnzySystem in agreement with the TGA results obtained with conventional methods in fresh non frozen plasma? Participants are asked to fill in a questionnaire regarding their general health and hemophilia treatment. Subsequently, blood will be drawn from the patients before, and at 30, 120, and 240 minutes after FEIBA administration. Whole blood is immediately tested using the Version A HemA EnzySystem, and plasma is generated for testing with the Ceveron s100 (Technoclone). Leftover samples are frozen for later additional coagulation testing.

Conditions

Hemophilia A With Inhibitor; Hemophilia A

Outcome Measures

Primary Outcomes (1)

• Time between venipuncture and TGA result

  The primary objective of this study is to demonstrate that the EnzySystem Thrombin Generation assay can record thrombin generation within a time frame of 60 min. Fresh whole blood samples of patients with hemophilia A on prophylactic or on-demand FEIBA treatment are used and tested in a near-patient setting at the Institute of Hematology and Transfusion Medicine (IHiT)

  2 hours

Secondary Outcomes (1)

• Agreement between TGA obtained with the EnzySystem HemA version A and conventional TGA results

  6 hours

Study Arms (1)

Hemophilia A patients prophylactically treated with FEIBA

Diagnostic Test: Thrombin generation assay (EnzySystem HemA version A - whole blood); Diagnostic Test: Thrombin generation assay (Ceveron s100 (Technoclone) - fresh plasma); Diagnostic Test: Additional coagulation tests

Interventions

Thrombin generation assay (EnzySystem HemA version A - whole blood) DIAGNOSTIC_TEST

Whole blood obtained by venipuncture is immediately tested with the EnzySystem HemA version A), before and 0, 30, 120 and 240 minutes after administration of FEIBA.

Hemophilia A patients prophylactically treated with FEIBA

Thrombin generation assay (Ceveron s100 (Technoclone) - fresh plasma) DIAGNOSTIC_TEST

Whole blood is centrifuged to obtain plasma. The fresh plasma is tested using the Ceveron s100.

Hemophilia A patients prophylactically treated with FEIBA

Additional coagulation tests DIAGNOSTIC_TEST

Plasma samples are frozen for later coagulation testing. The following tests are performed: Factor VIII activity von Willebrand Factor antigen levels von Willebrand Factor ristocetin activity levels Prothrombin Fragment 1+2 levels ADAMTS13 activity FVIII antigen levels Nijmegen Hemostasis Assay

Hemophilia A patients prophylactically treated with FEIBA

Eligibility Criteria

• Age: 18 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

Hemophilia A patients at the Institute of Hematology and Transfusion Medicine who are on prophylactic or on-demand FEIBA treatment.

You may qualify if:

• Diagnosed with mild (FVIII activity levels 5-40%), moderate (FVIII activity levels 1-5%) or severe hemophilia A (FVIII activity levels \<1%)
• With inhibitors
• On prophylactic or on-demand treatment with FEIBA (variation in dosing and duration of therapy use)

You may not qualify if:

• use of anticoagulants or platelet antagonists (aspirin or any TAR);
• known allergy to stainless steel;
• a bleeding episode within the last two weeks;
• clinical indication of liver cirrhosis (ultrasonography indication, enlarged spleen, decreased platelet count \<100 G/l);
• Signs of inflammation or infection
• Current use of:
• NSAIDs; antimicrobial medication; thyroid inhibitors or SSRI's;

Sponsors & Collaborators

• Enzyre B.V.: lead
• Instytut Hematologii i Transfuzjologii, Warschau, Poland: collaborator

Study Sites (1)

Institute of Hematology and Blood Transfusion

Warsaw, Mazowieckie Województwo, Poland

Location

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: CROSS SECTIONAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 5, 2024
• First Posted: April 10, 2024
• Study Start: March 26, 2024
• Primary Completion: March 27, 2024
• Study Completion: March 27, 2024
• Last Updated: April 10, 2024

Record last verified: 2024-04

Data Sharing

• IPD Sharing: Will not share

Locations

PL (1)