- 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
Search Results
Search by objective public record fields.
Search by query text, NCT ID, condition, intervention, sponsor, city, state, recruitment status, phase, study type, healthy volunteer eligibility, sex, or age. Results are retrieved from ClinicalTrials.gov and synchronized into the directory. Search pages remain noindex by default.
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.
Local D1 index available.
- 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
- 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
- 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
- Conditions
- Solid Tumor
- Interventions
- RMC-4630, Cobimetinib, Drug: Osimertinib
- Drug
- Lead sponsor
- Revolution Medicines, Inc.
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 113 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 21
- States / cities
- Scottsdale, Arizona • Duarte, California • Orange, California + 18 more
- 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
- 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
- 17
- States / cities
- Palo Alto, California • Sacramento, California • Aurora, Colorado + 13 more
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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
- 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