Clinical Trial of a MEK Inhibitor for RASopathy-Associated Severe Infantile Hypertrophic Cardiomyopathy: Baby MERIT

Summary

This project seeks to perform a Phase 3 clinical trial to test whether an FDA-approved cancer drug called trametinib is effective in treating young infants with genetic conditions called RASopathies and a severe, life-threatening heart problem called hypertrophic cardiomyopathy. The purpose of this research is to demonstrate that trametinib is effective in preventing these sick babies from dying, receiving a heart transplant or undergoing heart surgery to remove extra heart muscle over a one-year period. In addition, the investigators will determine how often this heart problem comes back when the trametinib treatment is stopped.

Timeline

Start

2027-08-01

Primary completion

2032-06-30

Completion

2032-06-30

Drugs

Indications