Noonan Syndrome clinical trials
The registry lists 49 studies for Noonan Syndrome across more than 10 countries. 21 are recruiting or about to start. 22 of them are mainly about Noonan Syndrome (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-05.
Get an email when these change. Save the Noonan Syndrome studies recruiting or about to start — once a week, free: studies that open, close, add sites or post results.
See an example email
Example · made-up studies
Breast cancer · Recruiting: 2 came in, 3 changed
What changed in your saved searches, Oct 31 – Nov 6:
A study of drug A after surgery for early breast cancer
Came in · now Recruiting
Drug B with hormone therapy in advanced breast cancer
Changed
· Sites: 4 added, including Seoul and Busan
· Enrollment: 300 → 450 participants
Exercise during chemotherapy
Now Active, not recruiting
One email a week for all your saved searches. Stop any time.
Status
Who runs them
Novo Nordisk A/S (8) · Fondazione Policlinico Universitario Agostino Gemelli IRCCS (3) · University Hospital, Toulouse (3) · Jacobio Pharmaceuticals Co., Ltd. (3) · Istituto Giannina Gaslini (2) · National Cancer Institute (NCI) (2) · Revolution Medicines, Inc. (2) · Erasca, Inc. (2)
What is being tested here
Drugs under test in Noonan Syndrome studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 6
- Dec 2026Neuroradiological Features of Noonan Syndrome and Genotype-Phenotype Correlations · Istituto Giannina Gaslini
- Jan 2027Link Between Abnormal Bleeding and Coagulation Disorders in Noonan Syndromes · University Hospital, Bordeaux
- Jan 2027Constitution of a Biological Collection to Study the Pathophysiology in Noonan Syndrome · University Hospital, Toulouse
- Feb 2027National Multicentre Study on Lipid Profile in Noonan Syndrome and Related Disorders: Trends by Age, Gender and Genotype · IRCCS Azienda Ospedaliero-Universitaria di Bologna
- Mar 2027A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment · BioMarin Pharmaceutical
- Jun 2027Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial · National Cancer Institute (NCI)
Who these studies take
Of the 16 Noonan Syndrome studies recruiting now, 10 (63%) set no upper age limit and 4 (25%) stop at 75 or younger. 13 (81%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 7 (44%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 13 (81%) | 13 (81%) |
| 18 | 14 (88%) | 14 (88%) |
| 30 | 13 (81%) | 13 (81%) |
| 40 | 12 (75%) | 12 (75%) |
| 50 | 12 (75%) | 12 (75%) |
| 65 | 12 (75%) | 12 (75%) |
| 75 | 12 (75%) | 12 (75%) |
| 85 | 12 (75%) | 12 (75%) |
Across all 49 studies, recruiting or not: 39% stop at 75 or younger and 37% at 65; 65% take children; 0% are for women only and 0% for men only; 18% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
What changed recently
From the registry's own updates since Oct 14, 2026.
Newly registered2
- Neuroradiological Features of Noonan Syndrome and Genotype-Phenotype CorrelationsOct 19, 2026NCT07881081
- Registro Nazionale Sindrome Noonan / National Registry of Noonan SyndromeOct 19, 2026NCT07881159
Studies
Recruiting first, then most recently updated. Showing 49 of 49. search them.
- Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient DisordersRecruitingNCT07221851Phase 37 countries
- Registro Nazionale Sindrome Noonan / National Registry of Noonan SyndromeRecruitingNCT078811591 country
- Neuroradiological Features of Noonan Syndrome and Genotype-Phenotype CorrelationsRecruitingNCT078810811 country
- Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled TrialRecruitingNCT05361811Not applicable1 country
- French Registry of Children Treated With Norditropin® for Short Stature Associated With Noonan SyndromeEnrolling by invitationNCT053089271 country
- Clinical, Genetic, and Epidemiologic Study of Children and Adults With RASopathiesRecruitingNCT048889361 country
- Clinical Trial of a MEK Inhibitor for RASopathy-Associated Severe Infantile Hypertrophic Cardiomyopathy: Baby MERITNot yet recruitingNCT07817186Phase 3
- Familial Investigations of Childhood Cancer PredispositionRecruitingNCT030502681 country
- Constitution of a Biological Collection to Study the Pathophysiology in Noonan SyndromeRecruitingNCT052022101 country
- National Multicentre Study on Lipid Profile in Noonan Syndrome and Related Disorders: Trends by Age, Gender and GenotypeRecruitingNCT074648211 country
- Palliative Care Needs of Children With Rare Diseases and Their FamiliesEnrolling by invitationNCT06938542Not applicable1 country
- Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance TechniquesRecruitingNCT073363941 country
- RASopathy BiorepositoryRecruitingNCT043954951 country
- Link Between Abnormal Bleeding and Coagulation Disorders in Noonan SyndromesNot yet recruitingNCT072591351 country
- Prevalence and Characterization of Pain in RASopathiesRecruitingNCT06355622Not applicable1 country
- MEK Inhibitors for the Treatment of Hypertrophic Cardiomyopathy in Patients With RASopathiesRecruitingNCT06555237Phase 21 country
- Use of miRNAs in Growth Hormone Deficiency (GHD)RecruitingNCT064559561 country
- Solid Tumors in RASopathiesRecruitingNCT05761314Not applicable1 country
- Effect of RAS/MAPK Pathway Hyperactivation on Growth' and Bone' Profile of the RASopathiesRecruitingNCT06331117Not applicable1 country
- GROWing Up With Rare GENEtic SyndromesRecruitingNCT044633161 country
- BR790 in Combination With Anlotinib in Adult Subjects With Advanced Non-Small Cell Lung CancerNot yet recruitingNCT05715398Phase 1/2
- A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone TreatmentActive, not recruitingNCT06668805Phase 27 countries
- A Dose Escalation/Expansion Study of ERAS-601 in Patients With Advanced or Metastatic Solid TumorsCompletedNCT04670679Phase 12 countries
- A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9Active, not recruitingNCT05723835Phase 36 countries
- Dose Finding Study of TNO155 in Adult Patients With Advanced Solid TumorsTerminatedNCT03114319Phase 19 countries
- A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to GrowActive, not recruitingNCT05330325Phase 333 countries
- Vosoritide for Selected Genetic Causes of Short StatureActive, not recruitingNCT04219007Phase 21 country
- A First-in-Human, Phase 1 Study of JAB-3312 in Adult Patients With Advanced Solid TumorsCompletedNCT04045496Phase 11 country
- Study of Metabolic Modifications in Children With Noonan SyndromeCompletedNCT02383316Not applicable1 country
- A Study of JAB-3312 in Adult Patients With Advanced Solid Tumors in ChinaCompletedNCT04121286Phase 11 country
- Lymphatic Phenotype in Noonan Syndrome Spectrum DisordersCompletedNCT06267807Not applicable1 country
- A First in Human, Dose Escalation Study of JAB-3068 （SHP2 Inhibitor） in Adult Patients With Advanced Solid TumorsCompletedNCT03518554Phase 11 country
- A First-in-Human Study of JAB-3068 (SHP2 Inhibitor) in Adult Patients With Advanced Solid Tumors in ChinaCompletedNCT03565003Phase 1/21 country
- Joint and Hematologic Disorders of Noonan Syndrome: French Descriptive Cross-sectional StudyCompletedNCT065506351 country
- Synaptic Plasticity and Cognitive Function in RASopathiesTerminatedNCT03504501Phase 21 country
- Genetic Testing of Noonan Subjects Previously Treated With Norditropin®. An Extension to Trial GHNOO-1658CompletedNCT01529944Phase 31 country
- A Study of Anti-Cancer Therapies Targeting the MAPK Pathway in Patients With Advanced NSCLCCompletedNCT04959981Phase 11 country
- Dose-Esc/Exp RMC4630 & Cobi in Relapsed/Refractory Solid Tumors & RMC4630& Osi in EGFR+ Locally Adv/Meta NSCLCCompletedNCT03989115Phase 1/22 countries
- Treatment With HMG-COA Reductase Inhibitor of Growth and Bone Abnormalities in Children With Noonan SyndromeCompletedNCT02713945Phase 31 country
- RLY-1971 in Subjects With Advanced or Metastatic Solid TumorsCompletedNCT04252339Phase 11 country
- Post Marketing Surveillance on Long-term Use With Norditropin® (Short Stature Due to Noonan Syndrome)CompletedNCT034356271 country
- Dose Escalation of RMC-4630 Monotherapy in Relapsed/Refractory Solid TumorsStatus unknownNCT03634982Phase 11 country
- Hormonal Sensitivity in Patients With Noonan and LEOPARD SyndromesCompletedNCT024867311 country
- Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of MEK162 in Noonan Syndrome Hypertrophic CardiomyopathyWithdrawnNCT01556568Phase 22 countries
- Investigating the Long-term Efficacy and Safety of Two Doses of NN-220 (Somatropin) in Short Stature Due to Noonan SyndromeCompletedNCT01927861Phase 31 country
- Observational Prospective Study on Patients Treated With Norditropin®CompletedNCT0096012823 countries
- Somatropin Effect on Linear Growth and Final Height in Subjects With Noonan SyndromeCompletedNCT01529840Phase 31 country
- Effect of MAXOMAT ® on the Growth of Small Children to NOONAN's SyndromeCompletedNCT00452725Phase 31 country
- Research Study Using Recombinant Human Insulin-Like Growth Factor-1/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 for Children With Noonan SyndromeTerminatedNCT00351221Phase 21 country