- 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 63
matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
- 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
- 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
- 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
- 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
- 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
- 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
- 22Q11 Deletion Syndrome
- Interventions
- Aware Program
- Behavioral
- Lead sponsor
- Innovation Research & Training
- Other
- Eligibility
- 12 Years to 19 Years
- Enrollment
- 110 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2024
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- 22q11.2 Deletion Syndrome, Velo-Cardio-Facial Syndrome
- Interventions
- Cognitive remediation program, Small group social skills training
- Behavioral
- Lead sponsor
- Duke University
- Other
- Eligibility
- 11 Years to 17 Years
- Enrollment
- 38 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2015
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Facial Injuries, Traumatic Wounds and Injuries, Craniofacial Injuries, Craniofacial Defects, Facial Transplantation, Facial Deformity
- Interventions
- Bone marrow cell-based therapy & 1-drug immunosuppression.
- Drug
- Lead sponsor
- Johns Hopkins University
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2031
- U.S. locations
- 1
- States / cities
- Baltimore, Maryland
- 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
- Marfan Syndrome, Turner Syndrome, Ehlers-Danlos Syndrome, Loeys-Dietz Syndrome, FBN1, TGFBR1, TGFBR2, ACTA2 or MYH11 Genetic Mutation, Bicuspid Aortic Valve Without Known Family History, Bicuspid Aortic Valve With Family History, Bicuspid Aortic Valve With Coarctation, Familial Thoracic Aortic Aneurysm and Dissections, Shprintzen-Goldberg Syndrome, Other Aneur/Diss of Thoracic Aorta Not Due to Trauma, <50yo, Other Congenital Heart Disease
- Interventions
- Not listed
- Lead sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
- NIH
- Eligibility
- Not listed
- Enrollment
- 3,706 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2007 – 2016
- U.S. locations
- 7
- States / cities
- Honolulu, Hawaii • Baltimore, Maryland • New York, New York + 3 more
- Conditions
- DiGeorge Syndrome, DiGeorge Anomaly, Complete DiGeorge Anomaly, Complete DiGeorge Syndrome
- Interventions
- Thymus Tissue for Transplantation
- Biological
- Lead sponsor
- Sumitomo Pharma Switzerland GmbH
- Industry
- Eligibility
- Not listed
- Enrollment
- 15 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2019
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- DiGeorge Anomaly, Complete DiGeorge Anomaly, Complete Atypical DiGeorge Anomaly, Complete DiGeorge Syndrome, Complete Atypical DiGeorge Syndrome
- Interventions
- Cultured Thymus Tissue for Implantation (CTTI), Cultured Thymus Tissue Implantation and Parental Parathyroid Transplantation, Blood Draw, Rabbit anti-thymocyte globulin, Cyclosporine, Tacrolimus, Methylprednisolone or Prednisolone, Daclizumab, Mycophenolate mofetil
- Biological · Other · Procedure + 1 more
- Lead sponsor
- Sumitomo Pharma Switzerland GmbH
- Industry
- Eligibility
- Not listed
- Enrollment
- 14 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2005 – 2017
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Down Syndrome, Edwards Syndrome, Patau Syndrome, Klinefelter Syndrome, Turner Syndrome, DiGeorge Syndrome, Chromosome Deletion, Aneuploidy
- Interventions
- Blood sampling for Laboratory Developed Test (LDT) analysis
- Other
- Lead sponsor
- Progenity, Inc.
- Industry
- Eligibility
- 18 Years to 54 Years · Female only
- Enrollment
- 760 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2018
- U.S. locations
- 8
- States / cities
- Scottsdale, Arizona • Eunice, Louisiana • Brooklyn, New York + 5 more
- Conditions
- DiGeorge Syndrome, Complete Typical DiGeorge Anomaly
- Interventions
- Cultured Thymus Tissue for Implantation (CTTI)
- Biological
- Lead sponsor
- Sumitomo Pharma Switzerland GmbH
- Industry
- Eligibility
- Not listed
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1991 – 2017
- U.S. locations
- 1
- States / cities
- Durham, North Carolina
- Conditions
- Hypoparathyroidism, DiGeorge Syndrome
- Interventions
- PTH 1-34
- Drug
- Lead sponsor
- National Institute of Dental and Craniofacial Research (NIDCR)
- NIH
- Eligibility
- 18 Years to 70 Years
- Enrollment
- 46 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2006 – 2017
- U.S. locations
- 1
- States / cities
- Bethesda, Maryland
- 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
- 22Q11.2 Deletion Syndrome
- Interventions
- ZYN002
- Drug
- Lead sponsor
- Harmony Biosciences Management, Inc.
- Industry
- Eligibility
- 4 Years to 17 Years
- Enrollment
- 20 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2020 – 2022
- U.S. locations
- 1
- States / cities
- Greenville, South Carolina
- Conditions
- Complete DiGeorge Anomaly, DiGeorge Syndrome, DiGeorge Anomaly, Complete DiGeorge Syndrome
- Interventions
- Cultured Thymus Tissue
- Biological
- Lead sponsor
- Sumitomo Pharma Switzerland GmbH
- Industry
- Eligibility
- Not listed
- Enrollment
- 29 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2023
- U.S. locations
- 1
- States / cities
- Durham, North Carolina