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Life sciences · Phase 3 Trial
ClinicalTrials.gov · September 14, 2026
No summary has been generated for this record yet. What follows is drawn from its source metadata only.
Phase 3 Trial.
No findings were extractable from the material analysed.
Safety was not reported in the material analysed. Check the source before drawing any conclusion about harm.
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Registry record from ClinicalTrials.gov (NCT07817186). This is a study registration, not published results. Lead sponsor: Carelon Research. Recruitment status: NOT_YET_RECRUITING. Phase: PHASE3. Study type: INTERVENTIONAL. Enrollment: 25 participants (ESTIMATED). Conditions: HCM, Noonan Syndrome and Other Rasopathy, Heart Failure (Pediatric). Interventions: DRUG: Trametinib. Primary outcome measures: Time to first occurrence of death, heart transplantation, or LV septal myectomy to relieve LV outflow tract obstruction , From qualifying hospital admission through 12 months after the qualifying hospital admission. Brief 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.