Noonan Syndrome
27
12
13
10
Key Insights
Highlights
Success Rate
91% trial completion (above average)
Clinical Risk Assessment
Based on trial outcomes
Moderate Risk
Score: 40/100
3.7%
1 terminated out of 27 trials
90.9%
+4.4% vs benchmark
22%
6 trials in Phase 3/4
20%
2 of 10 completed with results
Key Signals
Data Visualizations
Phase Distribution
Trial Status
Trial Success Rate
Benchmark: 86.5%
Based on 10 completed trials
Clinical Trials (27)
Constitution of a Biological Collection to Study the Pathophysiology in Noonan Syndrome
National Multicentre Study on Lipid Profile in Noonan Syndrome and Related Disorders: Trends by Age, Gender and Genotype
Clinical, Genetic, and Epidemiologic Study of Children and Adults With RASopathies
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
Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial
Palliative Care Needs of Children With Rare Diseases and Their Families
Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Techniques
RASopathy Biorepository
Link Between Abnormal Bleeding and Coagulation Disorders in Noonan Syndromes
A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9
French Registry of Children Treated With Norditropin® for Short Stature Associated With Noonan Syndrome
Lymphatic Phenotype in Noonan Syndrome Spectrum Disorders
Prevalence and Characterization of Pain in RASopathies
MEK Inhibitors for the Treatment of Hypertrophic Cardiomyopathy in Patients With RASopathies
Joint and Hematologic Disorders of Noonan Syndrome: French Descriptive Cross-sectional Study
Solid Tumors in RASopathies
Effect of RAS/MAPK Pathway Hyperactivation on Growth' and Bone' Profile of the RASopathies
GROWing Up With Rare GENEtic Syndromes
Genetic Testing of Noonan Subjects Previously Treated With Norditropin®. An Extension to Trial GHNOO-1658
Treatment With HMG-COA Reductase Inhibitor of Growth and Bone Abnormalities in Children With Noonan Syndrome