Ignite Creation Date:
2024-05-06 @ 1:32 AM
Last Modification Date:
2024-10-26 @ 11:05 AM
Study NCT ID:
NCT01830361
Status:
COMPLETED
Last Update Posted:
2020-08-06
First Post:
2013-03-25
Brief Title:
Trial to Assess the Efficacy of Midostaurin PKC412 in Patients With c-KIT or FLT3-ITD Mutated t821 AML
Sponsor:
Technische Universität Dresden
Organization:
Technische Universität Dresden
Study Overview
Official Title:
A Single-arm Phase II Trial to Assess the Efficacy of Midostaurin PKC412 Added to Standard Primary Therapy in Patients With Newly Diagnosed c-KIT or FLT3-ITD Mutated t821 AML
Status:
COMPLETED
Status Verified Date:
2020-08
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:
MIDOKIT
Brief Summary:
To assess the efficacy of tyrosine-kinase inhibitor midostaurin in c-KIT or FLT3-ITD mutated t821 AML To assess the efficacy of midostaurin depending on the type of c-KIT mutation
Detailed Description:
AML patients displaying t821 have a relatively favourable outcome Nevertheless only approximately 50 of patients carrying this cytogenetic aberration are alive at 5 years This suggests that some patients have more aggressive leukemic phenotypes and indicates the need for treatment optimization with novel therapies
The mutated KIT gene as well as the FLT3-ITD mutation have recently been identified as factors most likely to explain the heterogeneous clinical outcomes within the group of t821 AML The FLT3 and c-KIT genes encode type III receptor tyrosine kinases RTK with important and partly redundant functions in early hematopoietic stem cells Various activating mutations have been described for both genes For c-KIT the incidence ranges from 17 to 48 depending on the source population and type of mutations determined It has been consistently shown that in AMLs with t821 mutated c-KIT is associated with a dramatically increased risk of relapse and reduced overall survival compared to their unmutated counterparts The FLT3-ITD mutation has a similar negative effect on prognosis in the patient group of t821 mutated AMLs as c-KIT
PKC412 midostaurin is known to inhibit the c-KIT RTK activity as well as the FLT3 kinase both in patients with ITD and TKD mutations It should therefore be possible to abrogate the negative impact of pathologically increased c-KIT or FLT3-ITD activity on relapse and overall survival by using midostaurin in this patient population Aim of the proposed clinical trial is to prove the efficacy of midostaurin in c-KIT or FLT3-ITD mutated t821- AMLs in an open-label one-arm design
The mutated KIT gene as well as the FLT3-ITD mutation have recently been identified as factors most likely to explain the heterogeneous clinical outcomes within the group of t821 AML The FLT3 and c-KIT genes encode type III receptor tyrosine kinases RTK with important and partly redundant functions in early hematopoietic stem cells Various activating mutations have been described for both genes For c-KIT the incidence ranges from 17 to 48 depending on the source population and type of mutations determined It has been consistently shown that in AMLs with t821 mutated c-KIT is associated with a dramatically increased risk of relapse and reduced overall survival compared to their unmutated counterparts The FLT3-ITD mutation has a similar negative effect on prognosis in the patient group of t821 mutated AMLs as c-KIT
PKC412 midostaurin is known to inhibit the c-KIT RTK activity as well as the FLT3 kinase both in patients with ITD and TKD mutations It should therefore be possible to abrogate the negative impact of pathologically increased c-KIT or FLT3-ITD activity on relapse and overall survival by using midostaurin in this patient population Aim of the proposed clinical trial is to prove the efficacy of midostaurin in c-KIT or FLT3-ITD mutated t821- AMLs in an open-label one-arm design
Study Oversight
Has Oversight DMC:
None
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?:
None
Secondary IDs
| Secondary ID | Type | Domain | Link |
|---|---|---|---|
| 2011-002567-17 | EUDRACT_NUMBER | None | None |