- Conditions
- Chronic Pain, Marfan Syndrome, Vascular Ehlers-Danlos Syndrome, Loeys-Dietz Syndrome
- Interventions
- Empowered Relief
- Behavioral
- Lead sponsor
- Stanford University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 92 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2024
- U.S. locations
- 1
- States / cities
- Palo Alto, California
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Showing 1–24
of 178
matching trials from the live ClinicalTrials.gov search.
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- 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
- Conditions
- Velopharyngeal Insufficiency, Velopharyngeal Incompetence Due to Cleft Palate, Inadequate Velopharyngeal Closure, Palatopharyngeal Incompetence, Hypernasality
- Interventions
- Expiratory Muscle Strength Training, Maintenance Training
- Device
- Lead sponsor
- Noel Jabbour
- Other
- Eligibility
- 5 Years to 17 Years
- Enrollment
- 30 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2025 – 2027
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Craniosynostoses
- Interventions
- MRI,DTI, functional MRI
- Diagnostic Test
- Lead sponsor
- University of Arkansas
- Other
- Eligibility
- Up to 4 Years
- Enrollment
- 9 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2024
- U.S. locations
- 1
- States / cities
- Little Rock, Arkansas
- Conditions
- Brain Malformation, Neuronal Migration Disorder, Cognition Disorder, Epilepsy
- Interventions
- Not listed
- Lead sponsor
- Harvard University Faculty of Medicine
- Other
- Eligibility
- Not listed
- Enrollment
- 3,500 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1996 – 2030
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- 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
- Craniofacial Abnormalities
- Interventions
- SMART-3RP
- Behavioral
- Lead sponsor
- Massachusetts General Hospital
- Other
- Eligibility
- 14 Years and older
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2015 – 2016
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- UC (Urothelial Cancer)
- Interventions
- pemigatinib
- Drug
- Lead sponsor
- Incyte Corporation
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 263 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2022
- U.S. locations
- 34
- States / cities
- Tucson, Arizona • San Diego, California • San Francisco, California + 31 more
- Conditions
- Plagiocephaly
- Interventions
- Premie Pouch
- Device
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- Up to 40 Days
- Enrollment
- 11 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2016 – 2019
- U.S. locations
- 3
- States / cities
- Boston, Massachusetts • Brighton, Massachusetts • Weymouth, Massachusetts
- Conditions
- Epilepsy; Seizure, Neuromuscular Diseases, Brain Malformation, Intellectual Disability, Autism Spectrum Disorder, Hypotonia, Inborn Errors of Metabolism, Movement Disorders, Genetic Disease, Development Delay, Chromosome Abnormality, Hearing Loss, Dysmorphic Features, Skeletal Dysplasia, Congenital Abnormality, Microcephaly, Macrocephaly
- Interventions
- Pre-visit prep, usual care + exome seq
- Behavioral · Diagnostic Test
- Lead sponsor
- University of North Carolina, Chapel Hill
- Other
- Eligibility
- 0 Years and older
- Enrollment
- 548 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2024
- U.S. locations
- 3
- States / cities
- Asheville, North Carolina • Chapel Hill, North Carolina • Greenville, North Carolina
- 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
- DiGeorge Syndrome, Hypoparathyroidism, Complete DiGeorge Syndrome
- Interventions
- Thymus/Parathyroid Transplantation
- Biological
- Lead sponsor
- Sumitomo Pharma Switzerland GmbH
- Industry
- Eligibility
- Up to 24 Months
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2019
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- 22q11.2 Deletion Syndrome
- Interventions
- NFC-1
- Drug
- Lead sponsor
- Aevi Genomic Medicine, LLC, a Cerecor company
- Industry
- Eligibility
- 12 Years to 17 Years
- Enrollment
- 2 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2017
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania
- Conditions
- Gastric Adenocarcinoma
- Interventions
- Derazantinib, Derazantinib-paclitaxel-ramucirumab combination
- Drug
- Lead sponsor
- Basilea Pharmaceutica
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 47 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2022
- U.S. locations
- 2
- States / cities
- Orlando, Florida • New York, New York
- Conditions
- Urothelial Cancer, Receptors, Fibroblast Growth Factor
- Interventions
- Archival Tumor Tissue Sample
- Other
- Lead sponsor
- Janssen Research & Development, LLC
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 3,679 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2022
- U.S. locations
- 23
- States / cities
- Tucson, Arizona • Denver, Colorado • Newark, Delaware + 20 more
- Conditions
- Epilepsy, Focal Cortical Dysplasia, Tuberous Sclerosis, Hemimegalencephaly, Polymicrogyria, Rasmussen Encephalitis, Sturge-Weber Syndrome, Gliosis, Stroke, Tumor, Brain
- Interventions
- Intra-operative brain tonometry
- Device
- Lead sponsor
- University of California, Los Angeles
- Other
- Eligibility
- Not listed
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2023
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Subglottic Stenosis (SGS), Pierre Robin Sequence (PRS), Micrognathia, Normal Controls From CT Scans of the Upper Airway
- Interventions
- Not listed
- Lead sponsor
- University of North Carolina, Chapel Hill
- Other
- Eligibility
- Up to 17 Years
- Enrollment
- 39 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2015
- U.S. locations
- 1
- States / cities
- Chapel Hill, North Carolina
- Conditions
- Common Variable Immunodeficiency (CVID), Primary Immune Regulatory Disorder, Immune Dysregulation, DiGeorge Syndrome, STAT 1 Gain of Function, STAT 3 Gain of Function, Hypomorphic RAG1 Deficiency, CD40 Ligand Deficiency, Mendelian Susceptibility to Mycobacterial Disease, GATA2 Associated Immunodeficiency, CD40 Deficiency, Hypomorphic RAG2 Deficiency, Immune Dysregulation Polyendocrinopathy Enteropathy X-Linked Syndrome, Omenn Syndrome, Chronic Granulomatous Disease
- Interventions
- Hematopoietic stem cell transplant (HSCT)
- Biological
- Lead sponsor
- Paul Szabolcs
- Other
- Eligibility
- 5 Years to 40 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2026 – 2031
- U.S. locations
- 1
- States / cities
- Pittsburgh, Pennsylvania
- Conditions
- Nonsynostotic Plagiocephaly
- Interventions
- Non-interventional
- Other
- Lead sponsor
- Boston Children's Hospital
- Other
- Eligibility
- Up to 8 Months
- Enrollment
- 10 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2013 – 2020
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- 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
- Metastatic Urothelial Carcinoma, Unresectable Urothelial Carcinoma
- Interventions
- Pemigatinib, Pembrolizumab, Gemcitabine, Carboplatin
- Drug
- Lead sponsor
- Incyte Corporation
- Industry
- Eligibility
- 18 Years and older
- Enrollment
- 7 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2021
- U.S. locations
- 13
- States / cities
- Greenbrae, California • Newark, Delaware • Marietta, Georgia + 10 more
- Conditions
- Cleft Lip, Cleft Palate, Cleft Lip and Palate
- Interventions
- NA - survey instrument
- Other
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- 0 Years to 18 Years
- Enrollment
- 629 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2017 – 2020
- U.S. locations
- 1
- States / cities
- Philadelphia, Pennsylvania