Ignite Creation Date:
2024-05-06 @ 6:00 PM
Last Modification Date:
2024-10-26 @ 2:39 PM
Study NCT ID:
NCT05500807
Status:
RECRUITING
Last Update Posted:
2023-03-01
First Post:
2022-08-12
Brief Title:
Emicizumab for Severe Von Willebrand Disease VWD and VWDHemophilia A
Sponsor:
Bleeding and Clotting Disorders Institute Peoria Illinois
Organization:
Bleeding and Clotting Disorders Institute Peoria Illinois
Study Overview
Official Title:
Emicizumab for Severe VON Willebrand Disease VWD and VWDHemophilia A
Status:
RECRUITING
Status Verified Date:
2023-02
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:
BCDI-XII
Brief Summary:
Von Willebrand Disease VWD is the most common inherited bleeding disorder affecting up to 01 of the population is usually characterized by mucocutaneous bleeding HMB surgical bleeding or other hemostatic challenges Severe bleeding events require VWF concentrates administered solely through intravenous access Emicizumab Hemlibra is a monoclonal bispecific antibody developed to bind activated FIX and FX and mimic FVIII cofactor functionality Hemlibra is administered via subcutaneous injection rather than intravenous infusion The hypothesis of this study is that Emicizumab is safe and efficacious for prophylaxis in severe VWD and concomitant VWDhemophilia patients
Detailed Description:
Von Willebrand Disease VWD is the most common inherited bleeding disorder affecting up to 01 of the population is usually characterized by mucocutaneous bleeding HMB surgical bleeding or other hemostatic challenges VWD currently has few therapies generally useful in management of bleeding events including antifibrinolytics desmopressin DDAVP and VWF concentrates which may be plasma-derived VWF with and without FVIII or recombinant Minor bleeding may be successfully treated with antifibrinolytics and DDAVP however more severe bleeding requires VWF concentrates that are administered solely through intravenous access
Similarly it can be challenging to treat concomitant bleeding disorders with the existing therapeutic options available and patients with concurrent VWD and hemophilia A primarily have VWFFVIII concentrate or desmopression DDAVP available for treatment It has been well-recognized that patients caregivers and medical providers desire additional simplified therapeutic options that are not intravenous to treat severe bleeding disorders Therefore a simplified subcutaneous therapeutic that prevents bleeding would be strongly desired Though its use in the hemophilia A population is growing additional potential emicizumab applications for hemostatic control in other hemostatic disorders remain unknown A recent case report highlighted the hemostatic efficacy of emicizumab off-label use in type 3 von Willebrand Disease VWD another severe bleeding disorder This pediatric patient had type 3 VWD with alloantibodies and a bleeding phenotype similar to hemophilia A with inhibitor patients requiring suboptimal bleeding management with rFVIIa and activated prothrombin complex concentrates aPCC Emicizumab prophylaxis was initiated and the patient no longer required aPCC prophylaxis and rare use of rFVIIa for acute bleeding events only 1 trauma-induced soft tissue hematoma at the time of publication The authors concluded their report suggested the bleeding phenotype in type 3 VWD is expressed mainly due to factor VIII deficiency This study suggests a potential additional application for emicizumab in severe VWD
A pilot multicenter prospective open-label study of emicizumab prophylaxis in severe VWD and concomitant VWDhemophilia A Patients will have a one-year retrospective chart review of annualized bleed rate and hemostatic therapies collected at the time of enrollment Patients will then be treated with emicizumab with 3mgkg weekly for 4 weeks loading dose followed by once weekly prophylaxis of 15mgkg for 1 year Per emicizumab FDA-approved prescribing information for hemophilia A dose up-titration to 3 mgkg once weekly will be allowed after 24 weeks on HEMLIBRA prophylaxis in case of suboptimal efficacy ie 2 spontaneous and clinically significant bleeds Treatment records will be maintained along with bleeding event logs Breakthrough bleeding events may be treated with the patients usual on-demand therapy with antifibrinolytics or VWFFVIII concentrates per clinician discretion Patient reported outcome assessments will be collected throughout the clinical study to collect impact of the treatment on the individual patients assessing quality of life physical emotional social and general symptoms
Similarly it can be challenging to treat concomitant bleeding disorders with the existing therapeutic options available and patients with concurrent VWD and hemophilia A primarily have VWFFVIII concentrate or desmopression DDAVP available for treatment It has been well-recognized that patients caregivers and medical providers desire additional simplified therapeutic options that are not intravenous to treat severe bleeding disorders Therefore a simplified subcutaneous therapeutic that prevents bleeding would be strongly desired Though its use in the hemophilia A population is growing additional potential emicizumab applications for hemostatic control in other hemostatic disorders remain unknown A recent case report highlighted the hemostatic efficacy of emicizumab off-label use in type 3 von Willebrand Disease VWD another severe bleeding disorder This pediatric patient had type 3 VWD with alloantibodies and a bleeding phenotype similar to hemophilia A with inhibitor patients requiring suboptimal bleeding management with rFVIIa and activated prothrombin complex concentrates aPCC Emicizumab prophylaxis was initiated and the patient no longer required aPCC prophylaxis and rare use of rFVIIa for acute bleeding events only 1 trauma-induced soft tissue hematoma at the time of publication The authors concluded their report suggested the bleeding phenotype in type 3 VWD is expressed mainly due to factor VIII deficiency This study suggests a potential additional application for emicizumab in severe VWD
A pilot multicenter prospective open-label study of emicizumab prophylaxis in severe VWD and concomitant VWDhemophilia A Patients will have a one-year retrospective chart review of annualized bleed rate and hemostatic therapies collected at the time of enrollment Patients will then be treated with emicizumab with 3mgkg weekly for 4 weeks loading dose followed by once weekly prophylaxis of 15mgkg for 1 year Per emicizumab FDA-approved prescribing information for hemophilia A dose up-titration to 3 mgkg once weekly will be allowed after 24 weeks on HEMLIBRA prophylaxis in case of suboptimal efficacy ie 2 spontaneous and clinically significant bleeds Treatment records will be maintained along with bleeding event logs Breakthrough bleeding events may be treated with the patients usual on-demand therapy with antifibrinolytics or VWFFVIII concentrates per clinician discretion Patient reported outcome assessments will be collected throughout the clinical study to collect impact of the treatment on the individual patients assessing quality of life physical emotional social and general symptoms
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
True
Is a FDA Regulated Device?:
False
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
True
Is an FDA AA801 Violation?:
None