Ignite Creation Date:
2024-05-06 @ 8:21 PM
Last Modification Date:
2024-10-26 @ 3:26 PM
Study NCT ID:
NCT06357572
Status:
COMPLETED
Last Update Posted:
2024-04-10
First Post:
2024-04-05
Brief Title:
Real-life Clinical FEIBA Samples Measured Using the Version A of the HemA EnzySystem
Sponsor:
Enzyre BV
Organization:
Enzyre BV
Study Overview
Official Title:
Real-life Clinical FEIBA Samples Measured Using the Version A of the HemA EnzySystem
Status:
COMPLETED
Status Verified Date:
2024-04
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
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 questions 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
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
Detailed 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 t0 30 120 and 240 minutes
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 t0 30 120 and 240 minutes
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
False
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None