Using MEK inhibitors to treat hypertrophic cardiomyopathy in children with RASopathies
MEK Inhibitors for the Treatment of Hypertrophic Cardiomyopathy in Patients With RASopathies (MEKinRAS) - Randomized Controlled Trial
This study is testing if a new medication called trametinib can help children with RASopathies and hypertrophic cardiomyopathy feel better compared to standard treatment alone.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 40 (estimated) |
| Ages | 1 Day to 18 Years |
| Sex | All |
| Sponsor | Medical University of Warsaw Academic / other |
| Drugs / interventions | trametinib |
| Locations | 1 site (Warsaw) |
| Trial ID | NCT06555237 on ClinicalTrials.gov |
What this trial studies
This study aims to evaluate the effectiveness of trametinib, a MEK kinase inhibitor, in treating hypertrophic cardiomyopathy (HCM) in children with genetic mutations in the RAS/MAPK pathway, such as those seen in Noonan syndrome. It is a randomized, open-label trial involving patients aged 0 to 18 who have been diagnosed with both RASopathy and HCM. Participants will be divided into two groups: one receiving trametinib alongside standard treatment and the other receiving only standard treatment. The study will assess the effectiveness of trametinib over a three-month period, with potential continuation based on results.
Who should consider this trial
Good fit: Ideal candidates are children aged 0 to 18 with a confirmed genetic mutation in the RAS/MAPK pathway and a diagnosis of hypertrophic cardiomyopathy.
Not a fit: Patients who have contraindications to the medications involved or do not have a confirmed diagnosis of RASopathy or hypertrophic cardiomyopathy may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could significantly improve heart function and quality of life for children with hypertrophic cardiomyopathy associated with RASopathies.
How similar studies have performed: While there have been isolated cases of success in treating HCM in patients with RASopathies, this approach is being tested in a well-designed randomized study for the first time.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * patient with diagnosed RASopathy * patient with diagnosed hypertrophic cardiomyopathy * signed innform consent Exclusion Criteria: * contraindications to treatment with propranolol (drug hypersensitivity, atrioventricular block, severe bradycardia) disopyramide (drug hypersensitivity, WPW syndrome, atrioventricular block, QT prolongation) trametinib (drug hypersensitivity) * lack of consent of the child's guardians to participate in the study
Where this trial is running
Warsaw
- Department of Paediatrics, The Medical University of Warsaw, Poland — Warsaw, Poland (Recruiting)
Study contacts
- Principal investigator: MAciej Kołodziej, MD — Medical University of Warsaw
- Study coordinator: Maciej Kołodziej, MD
- Email: maciej.kolodziej@wum.edu.pl
- Phone: 22-317-95-37
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.