Ignite Creation Date:
2024-10-26 @ 3:37 PM
Last Modification Date:
2024-10-26 @ 3:37 PM
Study NCT ID:
NCT06555237
Status:
RECRUITING
Last Update Posted:
None
First Post:
2024-08-12
Brief Title:
MEK Inhibitors for the Treatment of Hypertrophic Cardiomyopathy in Patients With RASopathies
Sponsor:
None
Organization:
None
Study Overview
Official Title:
MEK Inhibitors for the Treatment of Hypertrophic Cardiomyopathy in Patients With RASopathies MEKinRAS - Randomized Controlled Trial
Status:
RECRUITING
Status Verified Date:
2024-08
Last Known Status:
None
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
No
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
MEKinRAS
Brief Summary:
The goal of this study is to evaluate the effectiveness of trametinib treatment in patients with Hyperthropic cardiomyopathy and a genetic mutation in the RASMAPK pathway
Detailed Description:
Introduction RASopathies are a group of genetic diseases caused by mutations in the mitogen-activated kinase RAS-MAPK pathway These mutations affect many processes and are the cause of numerous genetic syndromes including Noonan syndrome in the course of which severe hypertrophic cardiomyopathy HCM develops MEK kinase inhibitors are used to treat cancers with mutations in the RAS-MAPK pathway in adults So far single cases of HCM treatment in patients with RASopathies have been described with rapid improvement in both laboratory and echocardiographic parameters and regression of myocardial hypertrophy Due to the described effectiveness it is reasonable to verify these effects in a well-designed randomized study on a large group of patients
Objective To evaluate the effectiveness of trametinib treatment in patients with HCM and a genetic mutation in the RASMAPK pathway
Methodology
Randomized open-label study The study will include patients aged 0 to 18 with
mutation in the RASMAPK pathway confirmed by genetic tests
HCM diagnosed by echocardiography
In the first phase of the study 3 months patients will be randomly assigned to one of two groups
the intervention group will receive trametinib and standard treatment beta-blocker and disopyramide
the control group will receive only standard treatment Once this phase is complete patients will be assessed If higher effectiveness is demonstrated in the intervention group in the second phase of the study patients in the intervention group will continue their current treatment and patients in the control group will receive trametinib treatment Each group will receive trametinib for 12 months
Importance of the study The study results will provide grounds for routine introduction of MEK kinase inhibitors for the treatment of patients with HCM due to RASopathy If effectiveness is demonstrated this group will gain a simple non-invasive and causal treatment option
Objective To evaluate the effectiveness of trametinib treatment in patients with HCM and a genetic mutation in the RASMAPK pathway
Methodology
Randomized open-label study The study will include patients aged 0 to 18 with
mutation in the RASMAPK pathway confirmed by genetic tests
HCM diagnosed by echocardiography
In the first phase of the study 3 months patients will be randomly assigned to one of two groups
the intervention group will receive trametinib and standard treatment beta-blocker and disopyramide
the control group will receive only standard treatment Once this phase is complete patients will be assessed If higher effectiveness is demonstrated in the intervention group in the second phase of the study patients in the intervention group will continue their current treatment and patients in the control group will receive trametinib treatment Each group will receive trametinib for 12 months
Importance of the study The study results will provide grounds for routine introduction of MEK kinase inhibitors for the treatment of patients with HCM due to RASopathy If effectiveness is demonstrated this group will gain a simple non-invasive and causal treatment option
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