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Cancer drug tested against deadly infant heart disease

NCT ID NCT07817186

Disease control Sponsor: Carelon Research Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Sep 14, 2026 · Last updated Sep 15, 2026 · Updated 1 time

Summary

Researchers are testing whether trametinib, an FDA-approved cancer drug, can help infants with RASopathies who have a severe, life-threatening heart condition called hypertrophic cardiomyopathy. The trial enrolls about 25 babies with a confirmed genetic diagnosis. Participants receive daily oral trametinib for up to 12 months, with doses adjusted as they grow. The main goal is to see whether the drug prevents death, heart transplant, or surgery to remove extra heart muscle over one year. After treatment stops, researchers track whether the heart problem returns.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
trametinib, an FDA-approved cancer drug
What this could lead to
If it works, trametinib could become a treatment that keeps infants with RASopathy-linked severe hypertrophic cardiomyopathy alive without needing a heart transplant or surgery.
What could go wrong
This is a small Phase 3 trial in a very sick infant population, so results may not apply broadly. Trametinib can cause side effects, and the heart problem may return after treatment stops.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 25 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Aug 2027

An estimate. Start dates often move.

Expected to finish

Jun 2032

An estimate. End dates often move.

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

28 days to 6 months

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

Inclusion Criteria: * Molecular genetic diagnosis of a RASopathy signaling through the RAS/MAPK pathway as identified by molecular assays performed in a Clinical Laboratory Improvements of 1988 (CLIA) or similarly certified laboratories. Qualifying genotypes will included variants in any gene causing Noonan, Costello or cardiofaciocutaneous syndrome curated as pathogenic or likely pathogenic and consistent with the genetic mechanism (i.e., one allele in genes acting in an autosomal dominant manner and two alleles in trans for the autosomal recessive form of LZTR1-related Noonan syndrome). * Diagnosis of HCM, as defined by LV and interventricular septal wall thickness with a z-score \> 2 by echocardiography * Age ≥ 28 days and ≤ 6 months * Ross classification of HF of III or IV * Hospitalization * In the opinion of the site investigator, ability to comply with study protocol requirements * Signed informed consent for the trial by a parent or legal guardian Exclusion Criteria: * PTPN11 pathogenic/likely pathogenic variants causing NSML * Prior treatment with a MEK inhibitor * Requiring treatment with strong inhibitors of CYP2C19 and CYP3A4, strong inducers of CYP3A4, and substrates of CYP2C9 with a narrow therapeutic index * Platelet count \< 50,000/µL * Neoplastic disorder requiring treatment (e.g., juvenile myelomonocytic leukemia)

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Conditions

The condition(s) this trial relates to.

cardiofaciocutaneous syndrome Costello syndrome heart failure Noonan syndrome Noonan syndrome and Noonan-related syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

HCM Heart failure (pediatric) Noonan syndrome and other rasopathy

How to take part

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  1. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  2. A doctor treating you

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