19 completed, 12 recruiting, 6 unknown, 4 active not recruiting. 11 Phase 1, 9 Phase 2, 9 Phase 3, 7 Not applicable.
Follow Noonan Syndrome: new trials by email Literature Review of Noonan Syndrome trials Research Report on Noonan Syndrome
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Clinical Trial of a MEK Inhibitor for RASopathy-Associated Severe Infantile Hypertrophic Cardiomyopathy: Baby MERIT Trametinib - HCM, Noonan Syndrome and Other Rasopathy | Phase 3 | Not yet recruiting | Carelon Research | 25 | 2027-08-01 | 2026-09-14 |
| Link Between Abnormal Bleeding and Coagulation Disorders in Noonan Syndromes Reuse of routine clinical and biological data - Noonan Syndrome | Not yet recruiting | University Hospital, Bordeaux | 100 | 2026-01-01 | 2025-12-02 | |
| 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 Disorders Lonapegsomatropin [SKYTROFA®], Somatropin Pen Injector - Turner Syndrome, Short Stature Homeobox Gene Mutation | Phase 3 | Recruiting | Ascendis Pharma A/S | 186 | 2025-12-12 | 2026-11-05 |
| Palliative Care Needs of Children With Rare Diseases and Their Families Family Centered pediatric palliative care for family caregivers of children with rare diseases. - Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia | Not applicable | Enrolling by invitation | Children's National Research Institute | 480 | 2025-10-02 | 2026-01-28 |
| National Multicentre Study on Lipid Profile in Noonan Syndrome and Related Disorders: Trends by Age, Gender and Genotype RASopathies, Noonan Syndrome | Recruiting | IRCCS Azienda Ospedaliero-Universitaria di Bologna | 200 | 2025-02-26 | 2026-03-11 | |
| A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment Vosoritide Injection - Noonan Syndrome | Phase 2 | Active not recruiting | BioMarin Pharmaceutical | 30 | 2024-11-22 | 2026-09-23 |
| MEK Inhibitors for the Treatment of Hypertrophic Cardiomyopathy in Patients With RASopathies Trametinib tablet, Standard therapy - Cardiomegaly, Noonan Syndrome | Phase 2 | Recruiting | Medical University of Warsaw | 40 | 2024-08-01 | 2024-08-15 |
| Lymphatic Phenotype in Noonan Syndrome Spectrum Disorders Dynamic contrast-enhanced lymphangiography - Lymphatic System Disorders Congenital, Lymphatic Diseases | Not applicable | Completed | Radboud University Medical Center | 9 | 2024-03-27 | 2025-04-25 |
| Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial Waitlist, ACT Intervention - Neurofibromatosis 1, Noonan Syndrome | Not applicable | Recruiting | National Cancer Institute (NCI) | 70 | 2024-01-10 | 2026-10-16 |
| Use of miRNAs in Growth Hormone Deficiency (GHD) Growth Hormone Deficiency | Unknown | University of Parma | 400 | 2023-04-13 | 2024-06-13 | |
| BR790 in Combination With Anlotinib in Adult Subjects With Advanced Non-Small Cell Lung Cancer BR790+anlotinib - Non-Small Cell Lung Cancer | Phase 1 / Phase 2 | Unknown | Shanghai Gopherwood Biotech Co., Ltd. | 90 | 2023-02-01 | 2023-02-10 |
| A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9 Somapacitan - SGA, Turner Syndrome | Phase 3 | Active not recruiting | Novo Nordisk A/S | 47 | 2023-02-01 | 2026-08-13 |
| Registro Nazionale Sindrome Noonan / National Registry of Noonan Syndrome Noonan Syndrome | Recruiting | Istituto Giannina Gaslini | 400 | 2022-10-01 | 2026-10-19 | |
| A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to Grow Somapacitan, Norditropin® - SGA, Turner Syndrome, Noonan Syndrome, ISS | Phase 3 | Active not recruiting | Novo Nordisk A/S | 412 | 2022-08-10 | 2026-06-12 |
| Clinical, Genetic, and Epidemiologic Study of Children and Adults With RASopathies Costello Syndrome, Noonan Syndrome | Recruiting | National Cancer Institute (NCI) | 500 | 2022-04-25 | 2026-10-02 | |
| French Registry of Children Treated With Norditropin® for Short Stature Associated With Noonan Syndrome Norditropin - Noonan Syndrome | Enrolling by invitation | Novo Nordisk A/S | 221 | 2022-03-16 | 2026-10-08 | |
| Constitution of a Biological Collection to Study the Pathophysiology in Noonan Syndrome blood and urine sampling - Noonan Syndrome | Recruiting | University Hospital, Toulouse | 100 | 2022-01-26 | 2026-03-19 | |
| Solid Tumors in RASopathies Molecular characterization of solid tumor in RASopathies - RASopathy, Costello Syndrome | Not applicable | Unknown | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | 100 | 2021-10-12 | 2024-04-04 |
| A Study of Anti-Cancer Therapies Targeting the MAPK Pathway in Patients With Advanced NSCLC ERAS-007, ERAS-601, Osimertinib - Advanced Non-squamous Non-small-cell Lung Cancer | Phase 1 | Completed | Erasca, Inc. | 24 | 2021-09-02 | 2023-07-25 |
| Neuroradiological Features of Noonan Syndrome and Genotype-Phenotype Correlations Brain MRI and MR angiography (MRA) - Noonan Syndrome | Recruiting | Istituto Giannina Gaslini | 60 | 2021-05-15 | 2026-10-19 |
Showing 20 of 49. Sign in to see all of them.
Carcinoma, Non-Small-Cell Lung (8) · Cardiofaciocutaneous syndrome (6) · Colorectal Neoplasms (6) · Costello Syndrome (6) · Genetic Diseases, Inborn (6) · Squamous Cell Carcinoma of Head and Neck (6) · Turner Syndrome (6) · Neurofibromatosis 1 (4) · Dwarfism (3) · Esophageal Neoplasms (3) · Esophageal Squamous Cell Carcinoma (3) · Head and Neck Neoplasms (3) · Legius syndrome (3) · Lung Neoplasms (3)
Cardiovascular Abnormalities (49) · Cardiovascular Diseases (49) · Congenital Abnormalities (49) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (49) · Connective Tissue Diseases (49) · Craniofacial Abnormalities (49) · Heart Defects, Congenital (49) · Heart Diseases (49) · Musculoskeletal Abnormalities (49) · Skin and Connective Tissue Diseases (49) · Musculoskeletal Diseases (48) · Genetic Diseases, Inborn (16) · Neoplasms by Histologic Type (13) · Endocrine System Diseases (12) · Neoplasms (12)
Human Growth Hormone (8) · osimertinib (2) · somapacitan (2) · trametinib (2) · vosoritide (2) · Blood Specimen Collection (1) · Cetuximab (1) · Diagnostic Tests, Routine (1) · Glucose Tolerance Test (1) · Lamotrigine (1) · Lovastatin (1) · Simvastatin (1) · Standard of Care (1) · Waiting Lists (1) · binimetinib (1)
Novo Nordisk A/S (8) · Fondazione Policlinico Universitario Agostino Gemelli IRCCS (3) · Jacobio Pharmaceuticals Co., Ltd. (3) · University Hospital, Toulouse (3) · Erasca, Inc. (2) · Istituto Giannina Gaslini (2) · National Cancer Institute (NCI) (2) · Revolution Medicines, Inc. (2) · Allist Pharmaceuticals, Inc. (1) · Andrew Dauber (1) · Array Biopharma, now a wholly owned subsidiary of Pfizer (1) · Ascendis Pharma A/S (1) · BioMarin Pharmaceutical (1) · Carelon Research (1) · Children's Hospital Medical Center, Cincinnati (1)
United States (20) · France (12) · Italy (12) · Netherlands (6) · Spain (6) · Germany (5) · South Korea (5) · China (4) · Japan (4) · Canada (3) · Poland (3) · Sweden (3) · United Kingdom (3) · Australia (2) · Belgium (2)
Chiara Leoni, MD, PhD (3) · Revolution Medicines, Inc. (2) · Yuankai Shi, MD (2) · Andrew Dauber, MS MMSc (1) · Baohui Han (1) · Douglas R Stewart, M.D. (1) · Joyce Antal (1) · Kathryn N Weaver, MD (1) · Kenneth Attie, MD (1)
PMID 41285480, PMID 16203673, PMID 22508317, PMID 22788856, PMID 23658497, PMID 26903552, PMID 27280591, PMID 27736009, PMID 28522645, PMID 29109363, PMID 31305924, PMID 31972476, PMID 32269181, PMID 32499120, PMID 33571362, PMID 33709288, PMID 33910978, PMID 36947589, PMID 37280688, PMID 38813446, PMID 39290522, PMID 39344930, PMID 39659467, PMID 39700428, PMID 40041314, PMID 40919719, PMID 41712606, PMID 41774755, PMID 42621140
2008 (2), 2005 (1), 2016 (1), 2023 (1), R01HD117137 (1)