Key Insights

Highlights

Success Rate

91% trial completion (above average)

Clinical Risk Assessment

Based on trial outcomes

Moderate Risk

Score: 40/100

Termination Rate

3.7%

1 terminated out of 27 trials

Success Rate

90.9%

+4.4% vs benchmark

Late-Stage Pipeline

22%

6 trials in Phase 3/4

Results Transparency

20%

2 of 10 completed with results

Key Signals

2 with results91% success

Data Visualizations

Phase Distribution

15Total
Not Applicable (6)
P 2 (3)
P 3 (6)

Trial Status

Recruiting12
Completed10
Enrolling By Invitation2
Terminated1
Active Not Recruiting1
Not Yet Recruiting1

Trial Success Rate

90.9%

Benchmark: 86.5%

Based on 10 completed trials

Clinical Trials (27)

Showing 20 of 20 trials
NCT05202210RecruitingPrimary

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

NCT07464821Recruiting

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

NCT04888936Recruiting

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

NCT06668805Phase 2Recruiting

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

NCT05361811Not ApplicableRecruiting

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

NCT06938542Not ApplicableEnrolling By Invitation

Palliative Care Needs of Children With Rare Diseases and Their Families

NCT07336394Recruiting

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

NCT04395495Recruiting

RASopathy Biorepository

NCT07259135Not Yet RecruitingPrimary

Link Between Abnormal Bleeding and Coagulation Disorders in Noonan Syndromes

NCT05723835Phase 3Active Not Recruiting

A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9

NCT05308927Enrolling By InvitationPrimary

French Registry of Children Treated With Norditropin® for Short Stature Associated With Noonan Syndrome

NCT06267807Not ApplicableCompleted

Lymphatic Phenotype in Noonan Syndrome Spectrum Disorders

NCT06355622Not ApplicableRecruiting

Prevalence and Characterization of Pain in RASopathies

NCT06555237Phase 2Recruiting

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

NCT06550635CompletedPrimary

Joint and Hematologic Disorders of Noonan Syndrome: French Descriptive Cross-sectional Study

NCT05761314Not ApplicableRecruiting

Solid Tumors in RASopathies

NCT06331117Not ApplicableRecruiting

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

NCT04463316Recruiting

GROWing Up With Rare GENEtic Syndromes

NCT01529944Phase 3Completed

Genetic Testing of Noonan Subjects Previously Treated With Norditropin®. An Extension to Trial GHNOO-1658

NCT02713945Phase 3CompletedPrimary

Treatment With HMG-COA Reductase Inhibitor of Growth and Bone Abnormalities in Children With Noonan Syndrome

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