2 active not recruiting, 2 completed, 2 terminated, 1 recruiting. 9 Phase 2, 3 Phase 1.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 9 | Active not recruiting 2, Completed 2, Terminated 2, Recruiting 1, Unknown 1, Withdrawn 1 |
| Phase 1 | 3 | Completed 2, Unknown 1 |
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 |
|---|---|---|---|---|---|---|
| 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 |
| 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 |
| Vosoritide for Selected Genetic Causes of Short Stature Vosoritide - Short Stature | Phase 2 | Active not recruiting | Andrew Dauber | 56 | 2020-08-04 | 2026-03-18 |
| Dose-Esc/Exp RMC4630 & Cobi in Relapsed/Refractory Solid Tumors & RMC4630& Osi in EGFR+ Locally Adv/Meta NSCLC RMC-4630, Cobimetinib, Drug: Osimertinib - Solid Tumor | Phase 1 / Phase 2 | Completed | Revolution Medicines, Inc. | 113 | 2019-07-02 | 2023-06-26 |
| Synaptic Plasticity and Cognitive Function in RASopathies Lovastatin, Lamotrigine - Impaired Synaptic Plasticity, Impaired Cognition | Phase 2 | Terminated | Technical University of Munich | 16 | 2019-03-22 | 2023-11-30 |
| A First-in-Human Study of JAB-3068 (SHP2 Inhibitor) in Adult Patients With Advanced Solid Tumors in China JAB-3068 - Non-small Cell Lung Cancer, Head and Neck Cancer | Phase 1 / Phase 2 | Completed | Jacobio Pharmaceuticals Co., Ltd. | 126 | 2018-11-20 | 2024-09-25 |
| Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of MEK162 in Noonan Syndrome Hypertrophic Cardiomyopathy MEK162 - Cardiomegaly | Phase 2 | Withdrawn | Array Biopharma, now a wholly owned subsidiary of Pfizer | 2012-02-01 | 2020-10-06 | |
| Research Study Using Recombinant Human Insulin-Like Growth Factor-1/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 for Children With Noonan Syndrome rhIGF-1/rhIGFBP-3 - Noonan Syndrome | Phase 2 | Terminated | Insmed Incorporated | 24 | 2007-03-30 |
Carcinoma, Non-Small-Cell Lung (3) · Cardiomegaly (2) · Colorectal Neoplasms (2) · Dwarfism (2) · Bone Diseases (1) · Bronchial Neoplasms (1) · Carcinoma, Squamous Cell (1) · Cardiomyopathies (1) · Cardiomyopathy, Hypertrophic (1) · Cognition Disorders (1) · Esophageal Neoplasms (1) · Gastrointestinal Neoplasms (1) · Head and Neck Neoplasms (1) · Hypochondroplasia (1)
Cardiovascular Abnormalities (9) · Cardiovascular Diseases (9) · Congenital Abnormalities (9) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (9) · Connective Tissue Diseases (9) · Craniofacial Abnormalities (9) · Heart Defects, Congenital (9) · Heart Diseases (9) · Musculoskeletal Abnormalities (9) · Skin and Connective Tissue Diseases (9) · Musculoskeletal Diseases (8) · Carcinoma, Bronchogenic (3) · Endocrine System Diseases (3) · Genetic Diseases, Inborn (3) · Neoplasms (3)
vosoritide (2) · Lamotrigine (1) · Lovastatin (1) · Standard of Care (1) · binimetinib (1) · cobimetinib (1) · osimertinib (1) · trametinib (1)
Andrew Dauber (1) · Array Biopharma, now a wholly owned subsidiary of Pfizer (1) · BioMarin Pharmaceutical (1) · Insmed Incorporated (1) · Jacobio Pharmaceuticals Co., Ltd. (1) · Medical University of Warsaw (1) · Revolution Medicines, Inc. (1) · Shanghai Gopherwood Biotech Co., Ltd. (1) · Technical University of Munich (1)
United States (5) · Germany (2) · Australia (1) · Canada (1) · China (1) · France (1) · Italy (1) · Poland (1) · South Korea (1) · Spain (1) · United Kingdom (1)
Andrew Dauber, MS MMSc (1) · Baohui Han (1) · Kenneth Attie, MD (1) · Revolution Medicines, Inc. (1) · Yuankai Shi, MD (1)
PMID 37280688, PMID 38813446, PMID 40041314
2016 (1)