Ignite Creation Date:
2024-05-06 @ 7:25 PM
Last Modification Date:
2024-10-26 @ 3:06 PM
Study NCT ID:
NCT06009497
Status:
NOT_YET_RECRUITING
Last Update Posted:
2023-08-24
First Post:
2023-08-18
Brief Title:
Romiplostim in Combination With CsA vs CsA in the Treatment of Newly Diagnosed NSAA
Sponsor:
Peking Union Medical College Hospital
Organization:
Peking Union Medical College Hospital
Study Overview
Official Title:
Romiplostim in Combination With Ciclosporin Versus Ciclosporin in the Treatment of Newly Diagnosed Non-severe Aplastic Anemia
Status:
NOT_YET_RECRUITING
Status Verified Date:
2023-08
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:
Aplastic anemia AA is a group of clinical syndromes caused by a significant decrease in bone marrow hematopoietic tissue from different etiologies resulting in hematopoietic failure Treatment options for patients with aplastic anemia are very limited In a phase IIIII multicenter open-label study exploring the efficacy and safety of romiplostim the primary endpoint showed an overall response rate of 84 95 CI 66-95 at week 27 However there are no prospective clinical data exploring whether romiplostim combined with ciclosporin CsA can further improve efficacy than ciclosporin monotherapy in newly diagnosed NSAA Therefore we aimed to compare the efficacy and safety of romiplostim in combination with CsA versus CsA monotherapy
Detailed Description:
Aplastic anemia AA is a group of clinical syndromes caused by a significant decrease in bone marrow hematopoietic tissue from different etiologies resulting in hematopoietic failure Treatment options for patients with aplastic anemia are very limited By binding to the thrombopoietin TPO receptor thrombopoietin receptor agonists TPO-RAs can cause conformational changes in the TPO receptor activate the JAK2STATS pathway and increase megakaryocyte progenitor cells proliferation and platelet production At present TPO-RAs including eltrombopag lusutrombopag romiplostim and avatrombopag have been approved by FDA In a phase IIIII multicenter open-label study exploring the efficacy and safety of romiplostim 31 patients with refractory AA were enrolled The primary endpoint for the proportion of patients who achieved any hematological platelet neutrophil and red blood cell response at week 27 was 84 95 CI 66-95 At week 53 the three-line response rate was 39 95 CI 22-58 However there are no prospective clinical data exploring whether romiplostim combined with ciclosporin CsA can further improve efficacy than ciclosporin monotherapy in newly diagnosed NSAA Since romiplostim and CsA have good safety if it is proved that the combination of the two drugs is superior to ciclosporin monotherapy it can quickly improve blood patterns in NSAA avoid the use of more expensive ATG and provide some help to reduce the economic burden of AA patients
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