Ignite Creation Date:
2024-05-06 @ 8:12 PM
Last Modification Date:
2024-10-26 @ 3:22 PM
Study NCT ID:
NCT06291025
Status:
NOT_YET_RECRUITING
Last Update Posted:
2024-03-04
First Post:
2024-02-26
Brief Title:
Efficacy and Safety of Immunosuppression Caplacizumab and Plasma Infusion Without Therapeutic Plasma Exchange in Immune-mediated Thrombotic Thrombocytopenic Purpura Multicentric Non-inferiority Single-arm Study
Sponsor:
University Hospital Rouen
Organization:
University Hospital Rouen
Study Overview
Official Title:
Efficacy and Safety of Immunosuppression Caplacizumab and Plasma Infusion Without Therapeutic Plasma Exchange in Immune-mediated Thrombotic Thrombocytopenic Purpura Multicentric Non-inferiority Single-arm Study
Status:
NOT_YET_RECRUITING
Status Verified Date:
2024-02
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:
PEX-FREE
Brief Summary:
Immune thrombotic thrombocytopenic purpura iTTP is caused by a severe autoantibody-mediated deficiency of ADAMTS13 leading to an accumulation of ultra-large von Willebrand factor multimers in plasma and finally to microthrombi in blood vessels The current standard of care of iTTP consists in the triple association of daily plasma exchange PEX 60 mlkgday immunosuppressive agents and anti-adhesive treatment Caplacizumab Our group recently reported the outcome of 90 patients with iTTP treated with this triple association and when compared to historical patients the triplet regimen prevented death refractoriness and exacerbations Likewise plasma volumes were reduced by 2 to 3-fold and we could reduce the median number of PEX sessions from 13 to 6 PEX is an invasive and time-consuming procedure associated with catheter and plasma-related complications ranging from 22 to 30 Consequently to alleviate the burden of care in iTTP using a regimen without PEX would represent a major and topical goal Attempts to treat patients with plasma infusion PI without PEX were previously reported and provided evidence that large volumes of PI 20-30 mlkgday improved the initial outcome of iTTP However fluid overload occurred in most cases after 5-7 days limiting the feasibility of this strategy Nevertheless the recent availability of caplacizumab opens the perspective of treating patients with plasma for a shorter period Recently strategies without PEX have been carried out in Jehovahs Witnesses with iTTP 5 Impressively improvement was rapid and comparable to those provided with a standard PEX-based treatment Additionally a treatment combining caplacizumab and immunosuppression only was successfully performed in six iTTP patients with severe neurologic andor cardiac involvement The rapid and durable improvement provides evidence that a regimen without plasma seems feasible However we consider that robust data are still lacking to completely remove plasmatherapy from iTTP management Based on these statements we wish here to address the efficacy and safety of a PEX-free regimen combining PI only 15 mlkgday corticosteroidsrituximab and caplacizumab
Detailed Description:
Immune thrombotic thrombocytopenic purpura iTTP is caused by a severe autoantibody-mediated deficiency of ADAMTS13 leading to an accumulation of ultra-large von Willebrand factor multimers in plasma and finally to microthrombi in blood vessels The current standard of care of iTTP consists in the triple association of daily plasma exchange PEX 60 mlkgday immunosuppressive agents and anti-adhesive treatment Caplacizumab Our group recently reported the outcome of 90 patients with iTTP treated with this triple association and when compared to historical patients the triplet regimen prevented death refractoriness and exacerbations Likewise plasma volumes were reduced by 2 to 3-fold and we could reduce the median number of PEX sessions from 13 to 6 PEX is an invasive and time-consuming procedure associated with catheter and plasma-related complications ranging from 22 to 30 Consequently to alleviate the burden of care in iTTP using a regimen without PEX would represent a major and topical goal Attempts to treat patients with plasma infusion PI without PEX were previously reported and provided evidence that large volumes of PI 20-30 mlkgday improved the initial outcome of iTTP However fluid overload occurred in most cases after 5-7 days limiting the feasibility of this strategy Nevertheless the recent availability of caplacizumab opens the perspective of treating patients with plasma for a shorter period Recently strategies without PEX have been carried out in Jehovahs Witnesses with iTTP 5 Impressively improvement was rapid and comparable to those provided with a standard PEX-based treatment Additionally a treatment combining caplacizumab and immunosuppression only was successfully performed in six iTTP patients with severe neurologic andor cardiac involvement The rapid and durable improvement provides evidence that a regimen without plasma seems feasible However we consider that robust data are still lacking to completely remove plasmatherapy from iTTP management Based on these statements we wish here to address the efficacy and safety of a PEX-free regimen combining PI only 15 mlkgday corticosteroidsrituximab and caplacizumab
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