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HCMPhase 3Baby MERITNCT07817186

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

A Phase 3 study testing Trametinib in people with HCM and Noonan Syndrome and Other Rasopathy, run by Carelon Research.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 28 days to 6 months
What is being tested?
Trametinib
Type of treatment
Pill or oral medicine
Could I receive a placebo?
No, everyone receives an active treatment
How many people
25 participants
Where
No sites listed yet
Study dates
1 Aug 2027to 30 Jun 2032
Phase
Phase 3
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 28 days to 6 months
  • All sexes
  • No healthy volunteers
  • Child

Who may be able to join

  • 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

Who cannot join

  • 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)

About this study

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.

Being tested
Trametinib
Keywords
Noonan Syndrome, RAS-HCM, RASopathy, Heart failure, trametinib, MEK inhibitor
Medical subject headings
Noonan Syndrome, Heart Failure

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • TrametinibDrug

    Participants will begin 0.025 mg/kg of oral trametinib once daily for up to 12 months. Dose will be adjusted for weight at each study visit. A 12-month surveillance phase follows cessation of treatment. If the participant experiences a RAS-HCM relapse during the surveillance phase, they will restart oral trametinib once daily for up to 12 additional months.

Study groups in NCT07817186
Study groupRoleWhat they receive
TreatmentExperimentalNot described
Receives: Drug: Trametinib

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Time to first occurrence of death, heart transplantation, or LV septal myectomy to relieve LV outflow tract obstruction

    This is a composite time-to-event outcome. An event is defined as the first occurrence of any of the following: death, heart transplantation, or LV septal myectomy to relieve LV outflow tract obstruction. For participants experiencing more than one component event, only the first event contributes to the primary endpoint. Participants who do not experience any component event are censored according to the prespecified analysis rules. The treatment arms will be compared using Kaplan-Meier methods and the log-rank test.

    Measured: From qualifying hospital admission through 12 months after the qualifying hospital admission

Secondary outcomes (7)

  1. Time from qualifying hospital admission to death

    Time-to-event outcome defined as the time from the qualifying hospital admission to death from any cause. Participants who do not experience death during the assessment period will be censored according to the prespecified analysis rules. Treatment arms will be compared using Kaplan-Meier methods and the log-rank test.

    Measured: From qualifying hospital admission through 12 months after the qualifying hospital admission

  2. Change from baseline in LV posterior wall thickness z-score at 12 months

    Change in LV posterior wall z-score from baseline to 12 months. The change is calculated as the 12-month LV posterior wall z-score minus the baseline LV posterior wall z-score. Treatment groups will be compared using ANCOVA with treatment group as a fixed factor and baseline z-score as a covariate.

    All measurements and z-scores performed centrally at the Boston Children's Hospital Echocardiography Core Lab.

    Measured: Baseline and 12 months after qualifying hospital admission

  3. Ross class at 12 months

    Ross class is an ordinal measure with 4 categories (Class I-IV), with higher classes indicating greater severity of heart failure symptoms. Ross class at 12 months will be compared between the treatment arms using an ordinal regression model adjusted for baseline Ross class.

    Measured: 12 months after qualifying hospital admission

  4. Change from baseline in log-transformed BNP at 12 months

    Change in log-transformed BNP from baseline to 12 months. The change is calculated as the 12-month value minus the baseline value. Treatment groups will be compared using ANCOVA with treatment group as a fixed factor and baseline log-transformed BNP/NT-proBN as a covariate.

    Measured: Baseline and 12 months after qualifying hospital admission

  5. Time to first occurrence of death, heart transplantation, or LV septal myectomy to relieve LV outflow tract obstruction

    This is a composite time-to-event outcome. An event is defined as the first occurrence of any of the following: death, heart transplantation, or LV septal myectomy to relieve LV outflow tract obstruction. For participants experiencing more than one component event, only the first event contributes to the primary endpoint. Participants who do not experience any component event are censored according to the prespecified analysis rules. The treatment arms will be compared using Kaplan-Meier methods and the log-rank test.

    Measured: From qualifying hospital admission through 24 months after the qualifying hospital admission

  6. Relapse-related (descriptive)

    frequency, timing, and nature of HCM relapse after trametinib cessation; echocardiographic and clinical factors at relapse; response of relapsed RAS-HCM to restarting trametinib over 12 months.

    Measured: from hospitalization till 24 months of follow-up

  7. Trametinib-related adverse events

    type, severity (Common Terminology Criteria for Adverse Events [CTCAE] Grade), expectedness, and relationship of all AEs and SAEs to trametinib, with focused listings for dermatologic, ophthalmologic, and cardiac AEs

    Measured: from hospitalization till 24 months of follow-up

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted14 Sep 2026
  2. Study start1 Aug 2027 (estimated)
  3. Primary completion30 Jun 2032 (estimated)
  4. Study completion30 Jun 2032 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
25 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07817186
Sponsor's protocol ID
21067 Baby MERIT
Acronym
Baby MERIT
Status last verified
Jul 2026
First submitted
17 Aug 2026
Last updated
14 Sep 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No

Questions about this trial

What is this clinical trial studying?

A Phase 3 study testing Trametinib in people with HCM and Noonan Syndrome and Other Rasopathy, run by Carelon Research.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 7 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: opening soon, as of the sponsor's last update on 14 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Time to first occurrence of death, heart transplantation, or LV septal myectomy to relieve LV outflow tract obstruction (time frame: From qualifying hospital admission through 12 months after the qualifying hospital admission). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07817186.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Sep 2026.

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