Noonan Syndrome Clinical Trials

10 recruiting

Noonan Syndrome Trials at a Glance

11 actively recruiting trials for noonan syndrome are listed on ClinicalTrialsFinder across 6 cities in 7 countries. The largest study group is Not Applicable with 3 trials, with the heaviest enrollment activity in Bethesda, Roma, and Aurora. Lead sponsors running noonan syndrome studies include Fondazione Policlinico Universitario Agostino Gemelli IRCCS, National Cancer Institute (NCI), and Children's Hospital Medical Center, Cincinnati.

Browse noonan syndrome trials by phase

Treatments under study

About Noonan Syndrome Clinical Trials

Looking for clinical trials for Noonan Syndrome? There are currently 10 studies actively recruiting participants. Clinical trials offer access to new treatments before they are widely available, and every approved therapy in use today was first tested through a clinical trial.

Below you can browse trials, sign up for alerts when new Noonan Syndrome trials open, and view eligibility criteria for each study. Each listing includes the study phase, locations, and enrollment details.

Frequently Asked Questions

Common questions about Noonan Syndrome clinical trials

A clinical trial is a carefully designed research study that tests new medical treatments, drugs, devices, or approaches in human volunteers. Every approved medication and treatment available today was proven safe and effective through clinical trials.

All clinical trials are reviewed and approved by Institutional Review Boards (IRBs) — independent committees that evaluate patient safety. Trials follow strict protocols, and your health is monitored closely throughout. You can withdraw at any time.

Not necessarily. Many trials compare the new treatment against the current standard of care, meaning all participants receive active treatment. When placebos are used, they are typically combined with standard treatment, not given alone. The trial description will always specify the design.

Under the Affordable Care Act, most private insurers are required to cover routine patient care costs during a clinical trial. The sponsor typically covers the investigational treatment itself. Medicare also covers routine costs for qualifying trials.

Yes. Participation is completely voluntary. You can withdraw at any time, for any reason, without it affecting your access to standard medical care.

Each trial has specific eligibility criteria — including age, diagnosis, disease stage, prior treatments, and general health. Browse the trials listed above and check their eligibility sections. You can also contact the trial site directly to discuss your situation.

Showing 111 of 11 trials

Recruiting

Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders

Cystic FibrosisHuntington DiseaseSickle Cell Disease+12 more
Assistance Publique - Hôpitaux de Paris550 enrolled1 locationNCT06147414
Recruiting

Constitution of a Biological Collection to Study the Pathophysiology in Noonan Syndrome

Noonan Syndrome
University Hospital, Toulouse100 enrolled1 locationNCT05202210
Recruiting

National Multicentre Study on Lipid Profile in Noonan Syndrome and Related Disorders: Trends by Age, Gender and Genotype

Noonan SyndromeRASopathies
IRCCS Azienda Ospedaliero-Universitaria di Bologna200 enrolled14 locationsNCT07464821
Recruiting

Clinical, Genetic, and Epidemiologic Study of Children and Adults With RASopathies

Noonan SyndromeCardiofaciocutaneous SyndromeCostello Syndrome+3 more
National Cancer Institute (NCI)500 enrolled2 locationsNCT04888936
Recruiting
Phase 2

A Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment

Noonan SyndromeTurner SyndromeShort Stature Homeobox- Containing Gene SHOX Deficiency
BioMarin Pharmaceutical54 enrolled24 locationsNCT06668805
Recruiting
Not Applicable

Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial

Neurofibromatosis 1Noonan SyndromeCardiofaciocutaneous Syndrome+2 more
National Cancer Institute (NCI)70 enrolled1 locationNCT05361811
Recruiting

Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques

Noonan SyndromeFabry DiseaseCardiac Amyloidosis+4 more
Chinese Academy of Medical Sciences, Fuwai Hospital1,000 enrolled1 locationNCT07336394
Recruiting

RASopathy Biorepository

RAS MutationNeurofibromatosis 1Noonan Syndrome+11 more
Children's Hospital Medical Center, Cincinnati1,000 enrolled1 locationNCT04395495
Recruiting
Phase 2

MEK Inhibitors for the Treatment of Hypertrophic Cardiomyopathy in Patients With RASopathies

Noonan SyndromeCardiomegaly
Medical University of Warsaw40 enrolled1 locationNCT06555237
Recruiting
Not Applicable

Solid Tumors in RASopathies

Noonan SyndromeCostello SyndromeRASopathy+1 more
Fondazione Policlinico Universitario Agostino Gemelli IRCCS100 enrolled1 locationNCT05761314
Recruiting
Not Applicable

Effect of RAS/MAPK Pathway Hyperactivation on Growth' and Bone' Profile of the RASopathies

Noonan SyndromeCostello SyndromeRASopathy+1 more
Fondazione Policlinico Universitario Agostino Gemelli IRCCS120 enrolled1 locationNCT06331117