Clinical Trial Details
— Status: Completed
Administrative data
NCT number |
NCT06357572 |
Other study ID # |
ENZ13-2024 |
Secondary ID |
|
Status |
Completed |
Phase |
|
First received |
|
Last updated |
|
Start date |
March 26, 2024 |
Est. completion date |
March 27, 2024 |
Study information
Verified date |
April 2024 |
Source |
Enzyre B.V. |
Contact |
n/a |
Is FDA regulated |
No |
Health authority |
|
Study type |
Observational
|
Clinical Trial 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.
Description:
Rationale: Hemophilia is an X-linked hemostatic bleeding disorder characterized by a lack of
coagulation factor VIII activity (HemA) or factor IX activity (HemB). Spontaneous major
bleeding in joints and muscles frequently occur when factor activity levels are low.
Currently, there is a growing evidence that the bleeding phenotype of hemophilia A patients
is not only reflected by the actual factor VIII activity level, as there is a large variety
in bleeding among patients with similar factor VIII activity.
Currently, patients with severe Hemophilia A are treated with either FVIII containing
products or Emicizumab. Initially emicizumab was described for patients who developed
inhibitors as inhibitors do not interfere with emicizumab. Despite, bleeding complications
still may occur and therefore bypassing therapies are still of great importance.
Unfortunately, monitoring treatment with these drugs is a challenge.
Thrombin generation profile may be an additional tool in hemophilia patients to differentiate
between bleeding phenotype, to guide prophylactic replacement therapy and adjust bypassing
therapy, especially in patients treated with emicizumab. Based on recent studies, thrombin
generation as a global hemostasis assay offers an opportunity to assess the hemostatic
capacity of patients, and therefore has much potential for monitoring therapy. To facilitate
this, a hand-held in vitro diagnostic medical device able to simultaneously measure multiple
disease biomarkers with a single drop of blood is currently in development, focusing on
simultaneous measurements of Factor VIII activity and Thrombin generation.
Objective: The primary objective of this study is to demonstrate that 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 Factor VIII Bypassing Agent (FEIBA).
As secondary objective, the study results will be validated with a Thrombin Generation Assay
(TGA) measured in freshly obtained non frozen plasma.
Study design: This is a cross-sectional observational study. All participants are asked to
fill a questionnaire prior to blood collection of four tubes. Blood sample measurements will
be conducted within a two-hour time frame using the version A of the HemA EnzySystem and the
preparation of plasma using a standard TGA assay. Any remaining blood material will be
processed and stored.
Study population: The study population consists of 6 patients treated with FEIBA. In total,
at least 24 samples are tested, four for each patient (t=0, 30, 120 and 240 minutes).