Recruiting PHASE2 Cardiomegaly

New treatment option for Cardiomegaly

Official title MEK Inhibitors for the Treatment of Hypertrophic Cardiomyopathy in Patients With RASopathies

ClinicalTrials.gov ID: NCT06555237

What this study is testing

What is Trametinib tablet?

Trametinib tablet is an investigational medicine, given as an once-daily pill taken by mouth, being studied as a potential treatment for cardiomegaly.

Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.

What it's testing
The goal of this study is to evaluate the effectiveness of trametinib treatment in patients with Hyperthropic cardiomyopathy and a genetic mutation in the RAS/MAPK pathway.
  • Phase 2: a mid-size study of how well it works
  • Which group you join is decided by chance.

A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.

Who can take part

Ages 1 to 18

You may be able to join if

  • patient with diagnosed RASopathy
  • patient with diagnosed hypertrophic cardiomyopathy
  • signed innform consent

You likely can't join if

  • contraindications to treatment with propranolol (drug hypersensitivity, atrioventricular block, severe bradycardia) disopyramide (drug...
  • lack of consent of the child's guardians to participate in the study

The study team makes the final eligibility decision.

Where it's taking place

  • Warsaw, Poland

Questions & answers

Do participants get paid in this trial?

This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.

Is it free to join, and do I need insurance?

Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.

How long does this study last?

The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.

Who can join this trial?

This study is enrolling all sexes, 1 day to 18 years. The study team makes the final eligibility decision.

Where is this trial taking place?

Study sites include Warsaw, Poland. Enter your location above to see the nearest site and check your eligibility.

Explore other conditions

BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.