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RecruitingNCT07344480RAS-CMUpdated Feb 18, 2026

Retrospective Natural History Study of RASopathy-associated Cardiomyopathy (RAS-CM)

An observational study in Hypertrophic Cardiomyopathy (HCM), Heart Failure and RASopathy, sponsored by Deutsches Herzzentrum Muenchen. Recruiting at 1 site in Germany. Per ClinicalTrials.gov, last updated 2026-02-18.

Sponsored by Deutsches Herzzentrum Muenchen · Observational

From the registry’s dates

  • Started Jun 2025; still recruiting 1 year 4 months later.
Study type
Observational
Model
Other
Time perspective
Other
Enrollment
100
Sex
All
01

Study summary

RASopathy-associated hypertrophic cardiomyopathy (RAS-CM) is a disease with high morbidity and high mortality if presenting during infancy. Targeted therapies have shown significant activity in preclinical models and case reports. Drugs that target the underlying cause of this disease are now developed in cancer patients. Conducting randomized trials is not possible in severely ill infants with RAS-CM. Existing historical controls from older eras are not sufficient as external controls to support drug development as they lack critical clinical and genetic information to allow comparison with the cohort planned for future clinical trials.

The purpose of this investigator-initiated retrospective natural history study is to collect clinical information and genetic information in patients with RAS-CM. The first goal is to establish a data set that meets regulatory requirements for the use as external control data in a future clinical trial, composing non-randomized, single-arm, open-label study cohorts. The second goal is to obtain natural history information that supports the selection of secondary exploratory endpoints chosen in a clinical trial.

02

Conditions studied

  • Hypertrophic Cardiomyopathy (HCM)
  • Heart Failure
  • RASopathy
03

In context

Cardiomyopathy, Hypertrophic

363 studies on the registry are indexed under Cardiomyopathy, Hypertrophic; 116 are open to participants now.

This study's planned enrollment of 100 is below the median of 259 across 174 observational studies indexed under Cardiomyopathy, Hypertrophic.

Browse Cardiomyopathy, Hypertrophic studies →

Lead sponsor

Deutsches Herzzentrum Muenchen is the lead sponsor of 114 studies on the registry; 10 are open to participants now.

Browse Deutsches Herzzentrum Muenchen studies →

Counted across the registry records on this site, refreshed daily.

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Who can participate

Ages eligible
Child (0–17), Adult (18–64), Older adult (65+)
Sexes eligible
All
Accepts healthy volunteers
No
Sampling method
Non-probability sample

Study population

Patients

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

Inclusion criteria

  • Molecular genetic diagnosis of a RASopathy (i.e., a pathogenic or likely pathogenic variant in one of the RAS-MAPK pathway genes identified, irrespective of when performed)
  • Imaging diagnosis of myocardial hypertrophy (echocardiography) showing a maximal end-diastolic wall thickness of greater than normal (z-score > 2) with or without outflow tract obstruction

  • Admitted to hospital between 01/01/2015 and 06/30/2019 for congestive heart failure* or developing progressive congestive heart failure during any hospital stay within first 6 months of life**

    • Ross score calculated from medical history and physical examination notes in the absence of any other reason prompting hospital admission (e.g., elective procedure, other organ dysfunction, etc.); **: defined by Ross Score greater than 2

Exclusion criteria

Exclusion Criteria:

  • Receiving mechanistic Target of Rapamycin Inhibitor (mTOR inhibitor) and/or Mitogen-Activated Protein Kinase Kinase Inhibitor (MEK inhibitors)
  • Inability to identify or retrospectively calculate the patient´s Ross Score within the first six months of life
05

Study design

Observational model
Other
Time perspective
Other
Enrollment
100 participants (estimated)
Target follow-up
12 Months
Patient registry
Yes

Interventions

  • OtherRetrospective data collection

    Retrospective data collection, observation group are patients with genetic diagnosis of congenital RASopathy with hypertrophic cardiomyopathy and heart failure

06

What researchers measure

Primary outcomes

  1. To define the one-year transplant-free survival rate of patients with infantile-onset RASopathy-associated hypertrophic cardiomyopathy, admitted to the hospital with congestive heart failure by 6 months of age.

    Time frame: Admitted to hospital between 01/01/2015 and 06/30/2019 for congestive heart failure* or developing progressive congestive heart failure during any hospital stay within first 6 months of life**

07

Study locations

1 of 1 sites recruiting
08

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Started Jun 2025 (actual). Primary completion is estimated for Dec 2026, 18 months after the start.

Of 82 observational studies under Cardiomyopathy, Hypertrophic, half reached primary completion within 85 months of starting (the median), counting those still running; a quarter within 43. This study's planned 18 months is below that.

How these figures are worked out, and which studies are left out of them.

Registered
Jan 7, 2026
First posted
Jan 15, 2026
StartActual
Jun 17, 2025
Primary completionEst.
Dec 31, 2026
CompletionEst.
Dec 31, 2026
Last update
Feb 18, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Feb 18, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
09

Registry details

Key details

Study ID
NCT07344480
Lead sponsor
Deutsches Herzzentrum Muenchen
Responsible party
Sponsor
First posted
Jan 15, 2026
Start date
Jun 17, 2025
Primary completion
Dec 31, 2026 (estimated)
Completion
Dec 31, 2026 (estimated)
Last update
Feb 18, 2026

Study contacts

Cordula Prof. Wolf
Contact
wolf@dhm.mhn.de
+49 89 1218-2441

Oversight

FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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