NEW YORK (October 21, 2026)— Researchers at the Icahn School of Medicine at Mount Sinai will lead a new national clinical trial evaluating a precision medicine approach for infants with Noonan syndrome and related conditions who develop severe hypertrophic cardiomyopathy, a potentially life-threatening condition in which the heart muscle becomes abnormally thick.
Noonan syndrome and related conditions, known as RASopathies, are genetic disorders involving changes in the RAS-MAPK signaling pathway, which plays an important role in cell growth and development. Cardiovascular abnormalities occur in 80 to 90 percent of people with Noonan syndrome, and hypertrophic cardiomyopathy occurs in approximately 20 percent. Some infants develop severe disease and heart failure early in life.
The National Heart, Lung, and Blood Institute (NHLBI), part of the National Institutes of Health (NIH), awarded Mount Sinai $946,567 for the first year of the BabyMERIT trial, titled “Clinical Trial of a MEK Inhibitor for RASopathy-Associated Severe Infantile Hypertrophic Cardiomyopathy.” The trial will evaluate the targeted therapy trametinib in these infants. Mount Sinai will serve as the Clinical Coordinating Center for the multicenter study, which will be conducted through the NHLBI-funded Pediatric Heart Network. Carelon Research/New England Research Institutes, which will serve as the study’s Data Coordinating Center, has also received NIH funding totaling over $4.43 million over six years to support BabyMERIT. The phased award to Mount Sinai includes recommended costs of approximately $4.06 million over six years, subject to approval of subsequent phases. Together, the awards support the clinical and data coordination infrastructure needed to conduct this national trial, and total more than $8.5 million.
Bruce D. Gelb, MD, Gogel Family Professor of the Child Health and Development Institute, Icahn School of Medicine at Mount Sinai, and Jane W. Newburger, MD, MPH, of Boston Children’s Hospital, are the Clinical Coordinating Center Principal Investigators.
“This trial addresses an urgent need for infants with RASopathy-associated hypertrophic cardiomyopathy, a severe condition for which treatment options are limited,” said Dr. Gelb. “By targeting a molecular pathway known to drive these disorders, BabyMERIT will allow us to rigorously evaluate whether trametinib can improve outcomes for these very young patients and provide much-needed prospective evidence to guide their care.”
The BabyMERIT trial builds on evidence that targeting the RAS-MAPK pathway may improve RASopathy-associated hypertrophic cardiomyopathy.
“Infants with severe RASopathy-associated hypertrophic cardiomyopathy can become critically ill very early in life, making it particularly important to develop evidence-based approaches to treatment,” said Dr. Newburger. “The Pediatric Heart Network gives us the ability to bring together investigators and centers across the country to study this rare condition systematically, while collecting rigorous data on both the potential benefits and risks of treatment.”
The trial will enroll 25 infants in the prospective treatment arm. Eligible participants will be older than 28 days and younger than 6 months and will have Noonan syndrome or closely related RASopathies, hypertrophic cardiomyopathy, and severe heart failure requiring hospitalization.
The study will use a single-arm, open-label design and compare outcomes with a real-world evidence cohort of approximately 100 infants who received standard-of-care treatment between 2015 and the present.
Participants will receive trametinib for 12 months, followed by up to 12 months of observation to monitor for relapse. Infants who relapse may be eligible for retreatment for up to another 12 months. The primary endpoint is a composite of death, heart transplantation, or septal myectomy (an open-heart surgery used to treat hypertrophic cardiomyopathy) by 12 months. Researchers will also evaluate heart function, heart failure, biomarkers, relapse and response to retreatment, and adverse events.
The study will include centralized review of cardiac imaging and biomarker testing through Pediatric Heart Network core laboratories, and close monitoring for potential adverse effects affecting the skin, eyes, heart, blood counts, and kidney, liver, and thyroid function.
The study could provide important evidence about whether targeting the underlying molecular pathway can improve outcomes for infants with severe RASopathy-associated hypertrophic cardiomyopathy.
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About the Mount Sinai Health System
Mount Sinai Health System is one of the nation’s leading integrated academic health systems and one of the largest in the New York metropolitan area. Its comprehensive system includes seven hospitals, more than 400 outpatient practices, over 600 research and clinical laboratories, the Icahn School of Medicine at Mount Sinai, the Graduate School of Biomedical Sciences, and the Mount Sinai Phillips School of Nursing. Together, the Health System comprises approximately 48,000 employees, more than 9,000 physicians, and 8,600 nurses.
As a leading learning health system, Mount Sinai combines clinical expertise with scientific discovery to improve patient care while training the next generation of health care and biomedical leaders. The Health System provides care across every stage of life, from prenatal care through geriatrics, while advancing personalized medicine through artificial intelligence, data science, and biomedical research.
Mount Sinai is consistently recognized among the nation’s leading academic health systems for patient care, research, and education. The Mount Sinai Hospital is ranked No. 1 in New York by Newsweek and No. 5 on the magazine’s World’s Best Hospitals list. The Icahn School of Medicine at Mount Sinai ranks No. 9 among U.S. medical schools and No. 1 among freestanding medical schools for National Institutes of Health funding, reflecting the strength of its scientific enterprise and leadership in biomedical research.