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ClinicalTrials.gov public records Last synced May 21, 2026, 8:08 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Showing 1–24 of 25 matching trials from the live ClinicalTrials.gov search.
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Filters: Condition: Noonan Syndrome Clear all
Active, not recruiting Phase 3 Interventional
View directory record Official record
Conditions
Low-grade Glioma, Rapidly Accelerated Fibrosarcoma (RAF) Altered Glioma, Pediatric Low-grade Glioma
Interventions
Tovorafenib, Chemotherapeutic Agent
Drug
Lead sponsor
Day One Biopharmaceuticals, Inc.
Industry
Eligibility
Up to 25 Years
Enrollment
418 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2031
U.S. locations
38
States / cities
Birmingham, Alabama • Phoenix, Arizona • Los Angeles, California + 33 more
Source: ClinicalTrials.gov public record
Updated May 13, 2026 · Synced May 21, 2026, 8:08 PM EDT
Recruiting Phase 3 Interventional
View directory record Official record
Conditions
Turner Syndrome, Short Stature Homeobox Gene Mutation, Idiopathic Short Stature, Small for Gestational Age at Delivery
Interventions
Lonapegsomatropin [SKYTROFA®], Somatropin Pen Injector
Combination Product
Lead sponsor
Ascendis Pharma A/S
Industry
Eligibility
2 Years to 17 Years
Enrollment
186 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
9
States / cities
Sacramento, California • Centennial, Colorado • Orlando, Florida + 6 more
Source: ClinicalTrials.gov public record
Updated May 18, 2026 · Synced May 21, 2026, 8:08 PM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Solid Tumor, Unspecified, Adult
Interventions
RLY-1971
Drug
Lead sponsor
Hoffmann-La Roche
Industry
Eligibility
18 Years and older
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
6
States / cities
Lake Mary, Florida • Sarasota, Florida • Boston, Massachusetts + 1 more
Source: ClinicalTrials.gov public record
Updated Jan 30, 2023 · Synced May 21, 2026, 8:08 PM EDT
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Trisomy 13 Syndrome, Arthrogryposis Congenita Multiplex With Intestinal Atresia, Asparagine Synthetase Deficiency, CHARGE Syndrome, Early Infantile Epileptic Encephalopathy, FOXG1 Syndrome, KBG Syndrome, Noonan Syndrome, Severe Hemophilia A, Short Bowel Syndrome, Beta-Propeller Protein-Associated Neurodegeneration, Brain Injury of Prematurity With Periventricular Leukomalacia, Chromosome 17p13.3 Microdeletion Syndrome, Chromosome 1q43-1q44 Deletion, Cockayne Syndrome, Congenital Diaphragmatic Hernia, End-Stage Renal Disease With Cloacal Anomaly, Mitochondrial Depletion Disorder, Severe Factor VII Deficiency
Interventions
Family Centered pediatric palliative care for family caregivers of children with rare diseases.
Behavioral
Lead sponsor
Children's National Research Institute
Other
Eligibility
12 Months to 99 Years
Enrollment
480 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2025 – 2029
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Jan 27, 2026 · Synced May 21, 2026, 8:08 PM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Non-small Cell Lung Cancer, Colorectal Cancer, Pancreatic Ductal Carcinoma, Esophageal Squamous Cell Carcinoma, Head and Neck Squamous Cell Carcinoma, Breast Cancer, Other Solid Tumors
Interventions
JAB-3312
Drug
Lead sponsor
Jacobio Pharmaceuticals Co., Ltd.
Industry
Eligibility
18 Years and older
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
4
States / cities
Denver, Colorado • Nashville, Tennessee • Houston, Texas + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 28, 2025 · Synced May 21, 2026, 8:08 PM EDT
Not listed Phase 1 Interventional
View directory record Official record
Conditions
Solid Tumors
Interventions
RMC-4630
Drug
Lead sponsor
Revolution Medicines, Inc.
Industry
Eligibility
18 Years and older
Enrollment
133 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
12
States / cities
Scottsdale, Arizona • Duarte, California • Orange, California + 9 more
Source: ClinicalTrials.gov public record
Updated Aug 31, 2022 · Synced May 21, 2026, 8:08 PM EDT
Active, not recruiting Phase 2 Interventional
View directory record Official record
Conditions
Short Stature
Interventions
Vosoritide
Drug
Lead sponsor
Andrew Dauber
Other
Eligibility
3 Years to 10 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2035
U.S. locations
1
States / cities
Washington D.C., District of Columbia
Source: ClinicalTrials.gov public record
Updated Mar 17, 2026 · Synced May 21, 2026, 8:08 PM EDT
Recruiting Phase 2 Interventional
View directory record Official record
Conditions
Noonan Syndrome
Interventions
Vosoritide Injection
Drug
Lead sponsor
BioMarin Pharmaceutical
Industry
Eligibility
3 Years to 11 Years
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2041
U.S. locations
22
States / cities
Los Angeles, California • Orange, California • Aurora, Colorado + 19 more
Source: ClinicalTrials.gov public record
Updated May 10, 2026 · Synced May 21, 2026, 8:08 PM EDT
Active, not recruiting Phase 3 Interventional Results available
View directory record Official record
Conditions
SGA, Turner Syndrome, Noonan Syndrome, ISS
Interventions
Somapacitan
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
10 Years to 18 Years
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
6
States / cities
Birmingham, Alabama • Sacramento, California • Centennial, Colorado + 3 more
Source: ClinicalTrials.gov public record
Updated Nov 20, 2025 · Synced May 21, 2026, 8:08 PM EDT
Withdrawn Phase 2 Interventional
View directory record Official record
Conditions
Cardiomegaly
Interventions
MEK162
Drug
Lead sponsor
Array Biopharma, now a wholly owned subsidiary of Pfizer
Industry
Eligibility
18 Years to 65 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
Started 2012
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Oct 5, 2020 · Synced May 21, 2026, 8:08 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
Costello Syndrome, Noonan Syndrome, Cardiofaciocutaneous Syndrome, Legius Syndrome, Capillary Arteriovenous Malformation Syndrome, RASopathy
Interventions
Not listed
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
1 Month to 99 Years
Enrollment
500 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2035
U.S. locations
2
States / cities
Bethesda, Maryland • Rockville, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced May 21, 2026, 8:08 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers
View directory record Official record
Conditions
RAS Mutation, Neurofibromatosis 1, Noonan Syndrome, Noonan Syndrome With Multiple Lentigines, Noonan Neurofibromatosis Syndrome, Cardiofaciocutaneous Syndrome, Costello Syndrome, Legius Syndrome, Smith-Kingsmore Syndrome, MTOR Gene Mutation, GATOR-1 Gene Mutation, SYNGAP1-Related Intellectual Disability, DLG4, MAPK1 Gene Mutation
Interventions
Not listed
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2017 – 2065
U.S. locations
1
States / cities
Cincinnati, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 17, 2025 · Synced May 21, 2026, 8:08 PM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
BRAF NP_004324.2:p.V600X, KRAS wt Allele, Metastatic Malignant Solid Neoplasm, Stage IV Colorectal Cancer AJCC v7, Stage IVA Colorectal Cancer AJCC v7, Stage IVB Colorectal Cancer AJCC v7, Unresectable Solid Neoplasm
Interventions
Cetuximab, Irinotecan Hydrochloride, Laboratory Biomarker Analysis, Pharmacological Study, Vemurafenib
Biological · Drug · Other
Lead sponsor
M.D. Anderson Cancer Center
Other
Eligibility
18 Years and older
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2022
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 22, 2026 · Synced May 21, 2026, 8:08 PM EDT
Terminated Phase 1 Interventional
View directory record Official record
Conditions
Advanced EGFR Mutant Non Small Cell LungCancer (NSCLC), KRAS G12-mutant NSCLC, Esophageal Squamous Cell Cancer (SCC), Head/Neck SCC, Melanoma, Advanced Gastrointestinal Stromal Tumors (GIST), Advanced NRAS/BRAFT wt Cutaneous Melanoma
Interventions
TNO155, TNO155 in combination with EGF816 (nazartinib)
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years to 99 Years
Enrollment
227 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2025
U.S. locations
4
States / cities
Tampa, Florida • Boston, Massachusetts • New York, New York + 1 more
Source: ClinicalTrials.gov public record
Updated Apr 15, 2026 · Synced May 21, 2026, 8:08 PM EDT
Active, not recruiting Phase 1 Interventional
View directory record Official record
Conditions
Advanced or Metastatic Solid Tumors
Interventions
ERAS-601, Cetuximab, Pembrolizumab
Drug
Lead sponsor
Erasca, Inc.
Industry
Eligibility
18 Years to 99 Years
Enrollment
90 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
12
States / cities
Birmingham, Alabama • Sarasota, Florida • Boston, Massachusetts + 9 more
Source: ClinicalTrials.gov public record
Updated Mar 24, 2026 · Synced May 21, 2026, 8:08 PM EDT
Terminated Phase 2 Interventional Results available
View directory record Official record
Conditions
ALK Fusion Protein Expression, BRAF wt Allele, Invasive Skin Melanoma, MET Fusion Gene Positive, NRAS wt Allele, NTRK1 Fusion Positive, NTRK2 Fusion Positive, NTRK3 Fusion Positive, RET Fusion Positive, ROS1 Fusion Positive, Stage III Cutaneous Melanoma, Stage IIIA Cutaneous Melanoma, Stage IIIB Cutaneous Melanoma, Stage IIIC Cutaneous Melanoma, Stage IV Cutaneous Melanoma
Interventions
Capmatinib, Ceritinib, Entrectinib, Laboratory Biomarker Analysis, Regorafenib
Drug · Other
Lead sponsor
University of California, San Francisco
Other
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
1
States / cities
San Francisco, California
Source: ClinicalTrials.gov public record
Updated Jan 26, 2020 · Synced May 21, 2026, 8:08 PM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Advanced Non-squamous Non-small-cell Lung Cancer
Interventions
ERAS-007, ERAS-601, Osimertinib, Sotorasib
Drug
Lead sponsor
Erasca, Inc.
Industry
Eligibility
18 Years to 99 Years
Enrollment
24 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2023
U.S. locations
11
States / cities
Duarte, California • Orange, California • Santa Monica, California + 7 more
Source: ClinicalTrials.gov public record
Updated Jul 24, 2023 · Synced May 21, 2026, 8:08 PM EDT
Recruiting No phase listed Observational
View directory record Official record
Conditions
Acute Leukemia, Adenomatous Polyposis, Adrenocortical Carcinoma, AML, BAP1 Tumor Predisposition Syndrome, Carney Complex, Choroid Plexus Carcinoma, Constitutional Mismatch Repair Deficiency Syndrome, Diamond-Blackfan Anemia, DICER1 Syndrome, Dyskeratosis Congenita, Emberger Syndrome, Familial Acute Myeloid Leukemia, Familial Adenomatous Polyposis, Fanconi Anemia, Familial Cancer, Familial Wilms Tumor, Familial Neuroblastoma, GIST, Hereditary Breast and Ovarian Cancer, Hereditary Paraganglioma-Pheochromocytoma Syndrome, Hodgkin Lymphoma, Juvenile Polyposis, Li-Fraumeni Syndrome, Lynch Syndrome, MDS, Melanoma Syndrome, Multiple Endocrine Neoplasia Type 1, Multiple Endocrine Neoplasia Type 2, Neuroblastoma, Neurofibromatosis Type 1, Neurofibromatosis Type II, Nevoid Basal Cell Carcinoma Syndrome, Non Hodgkin Lymphoma, Noonan Syndrome and Other Rasopathy, Overgrowth Syndromes, Pancreatic Cancer, Peutz-Jeghers Syndrome, Pheochromocytoma/Paraganglioma, PTEN Hamartoma Tumor Syndrome, Retinoblastoma, Rhabdoid Tumor Predisposition Syndrome, Rhabdomyosarcoma, Rothmund-Thomson Syndrome, Tuberous Sclerosis, Von Hippel-Lindau Disease
Interventions
Not listed
Lead sponsor
St. Jude Children's Research Hospital
Other
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2037
U.S. locations
1
States / cities
Memphis, Tennessee
Source: ClinicalTrials.gov public record
Updated Apr 22, 2026 · Synced May 21, 2026, 8:08 PM EDT
Terminated Phase 2 Interventional
View directory record Official record
Conditions
Noonan Syndrome
Interventions
rhIGF-1/rhIGFBP-3
Drug
Lead sponsor
Insmed Incorporated
Industry
Eligibility
2 Years to 16 Years
Enrollment
24 participants
U.S. locations
2
States / cities
New Hyde Park, New York • Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 29, 2007 · Synced May 21, 2026, 8:08 PM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Joint Health
Interventions
MyCondro 300mg, MyCondro 600mg
Dietary Supplement
Lead sponsor
Lesaffre International
Industry
Eligibility
45 Years and older
Enrollment
240 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2026
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Mar 24, 2026 · Synced May 21, 2026, 8:08 PM EDT
Completed Phase 1 Interventional
View directory record Official record
Conditions
Non-small Cell Lung Cancer, Head and Neck Cancer, Esophageal Cancer, Other Metastatic Solid Tumors
Interventions
JAB-3068
Drug
Lead sponsor
Jacobio Pharmaceuticals Co., Ltd.
Industry
Eligibility
18 Years and older
Enrollment
38 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2022
U.S. locations
4
States / cities
Denver, Colorado • Sarasota, Florida • Nashville, Tennessee + 1 more
Source: ClinicalTrials.gov public record
Updated Dec 15, 2024 · Synced May 21, 2026, 8:08 PM EDT
Active, not recruiting Phase 3 Interventional
View directory record Official record
Conditions
SGA, Turner Syndrome, Noonan Syndrome, ISS
Interventions
Somapacitan, Norditropin®
Drug
Lead sponsor
Novo Nordisk A/S
Industry
Eligibility
2 Years to 10 Years
Enrollment
412 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
35
States / cities
Birmingham, Alabama • Los Angeles, California • Sacramento, California + 32 more
Source: ClinicalTrials.gov public record
Updated Apr 23, 2026 · Synced May 21, 2026, 8:08 PM EDT
Completed Phase 2 Interventional Results available
View directory record Official record
Conditions
Solid Tumor and Hematologic Malignancies
Interventions
MEK162
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
110 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
68
States / cities
Mobile, Alabama • Anchorage, Alaska • Phoenix, Arizona + 57 more
Source: ClinicalTrials.gov public record
Updated Feb 20, 2021 · Synced May 21, 2026, 8:08 PM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers
View directory record Official record
Conditions
Neurofibromatosis 1, Noonan Syndrome, Legius Syndrome, Cardiofaciocutaneous Syndrome, Costello Syndrome
Interventions
Waitlist, ACT Intervention
Behavioral
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
70 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2024 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Feb 18, 2026 · Synced May 21, 2026, 8:08 PM EDT