- Conditions
- X-linked Hypophosphatemia
- Interventions
- burosumab, Placebo
- Biological · Other
- Lead sponsor
- Kyowa Kirin, Inc.
- Industry
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 134 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2015 – 2018
- U.S. locations
- 8
- States / cities
- Los Angeles, California • San Francisco, California • New Haven, Connecticut + 5 more
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- Conditions
- Spinal Muscular Atrophy, Fragile X Syndrome, Fragile X - Premutation, Duchenne Muscular Dystrophy, Hyperinsulinemic Hypoglycemia, Familial 1, Diabetes Mellitus, Adrenoleukodystrophy, Neonatal, Medium-chain Acyl-CoA Dehydrogenase Deficiency, Very Long Chain Acyl Coa Dehydrogenase Deficiency, Beta-ketothiolase Deficiency, Severe Combined Immunodeficiency Due to Adenosine Deaminase Deficiency, Primary Hyperoxaluria Type 1, Congenital Bile Acid Synthesis Defect Type 2, Pyridoxine-Dependent Epilepsy, Hereditary Fructose Intolerance, Hypophosphatasia, Hyperargininemia, Mucopolysaccharidosis Type 6, Argininosuccinic Aciduria, Citrullinemia, Type I, Wilson Disease, Maple Syrup Urine Disease, Type 1A, Maple Syrup Urine Disease, Type 1B, Biotinidase Deficiency, Neonatal Severe Primary Hyperparathyroidism, Intrinsic Factor Deficiency, Usher Syndrome Type 1D/F Digenic (Diagnosis), Cystic Fibrosis, Stickler Syndrome Type 2, Stickler Syndrome Type 1, Alport Syndrome, Autosomal Recessive, Alport Syndrome, X-Linked, Carbamoyl Phosphate Synthetase I Deficiency Disease, Carnitine Palmitoyl Transferase 1A Deficiency, Carnitine Palmitoyltransferase II Deficiency, Cystinosis, Chronic Granulomatous Disease, Cerebrotendinous Xanthomatoses, Maple Syrup Urine Disease, Type 2, Severe Combined Immunodeficiency Due to DCLRE1C Deficiency, Thyroid Dyshormonogenesis 6, Thyroid Dyshormonogenesis 5, Supravalvar Aortic Stenosis, Factor X Deficiency, Hemophilia A, Hemophilia B, Tyrosinemia, Type I, Fructose 1,6 Bisphosphatase Deficiency, Glycogen Storage Disease Type I, G6PD Deficiency, Glycogen Storage Disease II, Galactokinase Deficiency, Mucopolysaccharidosis Type IV A, Galactosemias, Guanidinoacetate Methyltransferase Deficiency, Agat Deficiency, Glutaryl-CoA Dehydrogenase Deficiency, Gtp Cyclohydrolase I Deficiency, Hyperinsulinism-Hyperammonemia Syndrome, Primary Hyperoxaluria Type 2, 3-Hydroxyacyl-CoA Dehydrogenase Deficiency, Long-chain 3-hydroxyacyl-CoA Dehydrogenase Deficiency, Mitochondrial Trifunctional Protein Deficiency, Sickle Cell Disease, Beta-Thalassemia, Holocarboxylase Synthetase Deficiency, 3-Hydroxy-3-Methylglutaric Aciduria, Primary Hyperoxaluria Type 3, Hermansky-Pudlak Syndrome 1, Hermansky-Pudlak Syndrome 4, Apparent Mineralocorticoid Excess, HSDB, CBAS1, Mucopolysaccharidosis Type 2, Mucopolysaccharidosis Type 1, Severe Combined Immunodeficiency, X Linked, Severe Combined Immunodeficiency Due to IL-7Ralpha Deficiency, Diabetes Mellitus, Permanent Neonatal, Isovaleric Acidemia, Severe Combined Immunodeficiency T-Cell Negative B-Cell Positive Due to Janus Kinase-3 Deficiency (Disorder), Jervell and Lange-Nielsen Syndrome 2, Hyperinsulinemic Hypoglycemia, Familial, 2, Diabetes Mellitus, Permanent Neonatal, With Neurologic Features, Jervell and Lange-Nielsen Syndrome 1, Lysosomal Acid Lipase Deficiency, CblF, 3-Methylcrotonyl CoA Carboxylase 1 Deficiency, 3-Methylcrotonyl CoA Carboxylase 2 Deficiency, Waardenburg Syndrome Type 2A, Methylmalonic Aciduria cblA Type, Methylmalonic Aciduria cblB Type, Methylmalonic Aciduria and Homocystinuria Type cblC, MAHCD, Methylmalonic Aciduria Due to Methylmalonyl-CoA Mutase Deficiency, Congenital Disorder of Glycosylation Type 1B, Mthfr Deficiency, Methylcobalamin Deficiency Type Cbl G (Disorder), Methylcobalamin Deficiency Type cblE, Usher Syndrome, Type 1B, N-acetylglutamate Synthase Deficiency, Ornithine Transcarbamylase Deficiency, Phenylketonurias, Waardenburg Syndrome Type 1, Congenital Hypothyroidism, Propionic Acidemia, Usher Syndrome, Type 1F, Pancreatic Agenesis 1, Hereditary Hypophosphatemic Rickets, Glycogen Storage Disease IXB, Glycogen Storage Disease IXC, MOWS, Epilepsy, Early-Onset, Vitamin B6-Dependent, Pyridoxal Phosphate-Responsive Seizures, Pituitary Hormone Deficiency, Combined, 1, Ptsd, Dihydropteridine Reductase Deficiency, Severe Combined Immunodeficiency Due to RAG1 Deficiency, Severe Combined Immunodeficiency Due to RAG2 Deficiency, Retinoblastoma, Multiple Endocrine Neoplasia Type 2B, Pseudohypoaldosteronism, Type I, Liddle Syndrome, Biotin-Responsive Basal Ganglia Disease, SCD, DIAR1, GSD1C, Acrodermatitis Enteropathica, Thyroid Dyshormonogenesis 1, Riboflavin Transporter Deficiency, Waardenburg Syndrome, Type 2E, SRD, Congenital Lipoid Adrenal Hyperplasia Due to STAR Deficiency, Barth Syndrome, Adrenocorticotropic Hormone Deficiency, Transcobalamin II Deficiency, Thyroid Dyshormonogenesis 3, Segawa Syndrome, Autosomal Recessive, Autosomal Recessive Nonsyndromic Hearing Loss, Thyroid Dyshormonogenesis 2A, Congenital Isolated Thyroid Stimulating Hormone Deficiency, Hypothyroidism Due to TSH Receptor Mutations, Usher Syndrome Type 1C, Usher Syndrome Type 1G (Diagnosis), Von Willebrand Disease, Type 3, Combined Immunodeficiency Due to ZAP70 Deficiency, Adenine Phosphoribosyltransferase Deficiency, Metachromatic Leukodystrophy, Canavan Disease, Menkes Disease, Carbonic Anhydrase VA Deficiency, Developmental and Epileptic Encephalopathy 2, 17 Alpha-Hydroxylase Deficiency, Smith-Lemli-Opitz Syndrome, Krabbe Disease, Glutathione Synthetase Deficiency, Mucopolysaccharidosis Type 7, Rett Syndrome, Molybdenum Cofactor Deficiency, Type A, Niemann-Pick Disease, Type C1, Niemann-Pick Disease Type C2, Ornithine Aminotransferase Deficiency, 3-Phosphoglycerate Dehydrogenase Deficiency, Leber Congenital Amaurosis 2, Dravet Syndrome, Mucopolysaccharidosis Type 3 A, Ornithine Translocase Deficiency, Carnitine-acylcarnitine Translocase Deficiency, Glucose Transporter Type 1 Deficiency Syndrome, Creatine Transporter Deficiency, Niemann-Pick Disease Type A, Pitt Hopkins Syndrome, Tuberous Sclerosis 1, Tuberous Sclerosis 2, Ataxia With Isolated Vitamin E Deficiency, Angelman Syndrome, Prader-Willi Syndrome, Homocystinuria, Permanent Neonatal Diabetes Mellitus, Transient Neonatal Diabetes Mellitus, Factor VII Deficiency, Glycogen Storage Disease Type IXA1, Glycogen Storage Disease, Type IXA2, Glycogen Storage Disease IC, Glycogen Storage Disease Type IB, Central Hypoventilation Syndrome With or Without Hirschsprung Disease
- Interventions
- Confirmatory Testing
- Diagnostic Test
- Lead sponsor
- RTI International
- Other
- Eligibility
- 1 Day to 31 Days
- Enrollment
- 30,000 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2018 – 2026
- U.S. locations
- 1
- States / cities
- Research Triangle Park, North Carolina
- Conditions
- Breast Cancer
- Interventions
- Not listed
- Lead sponsor
- New York Presbyterian Hospital
- Other
- Eligibility
- 18 Years and older · Female only
- Enrollment
- 2,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 1991
- U.S. locations
- 2
- States / cities
- New York, New York
- Conditions
- Dementia, Primary Palliative Care, Mild Cognitive Impairment
- Interventions
- nurse-led supportive care assessment
- Behavioral
- Lead sponsor
- Stanford University
- Other
- Eligibility
- 65 Years and older
- Enrollment
- 200 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2019 – 2026
- U.S. locations
- 1
- States / cities
- Palo Alto, California
- Conditions
- Bipolar Disorder (BD), Bipolar Disorder I or II, Bipolar Disorder Family Members, Screening Tool
- Interventions
- Co-design Workshop (Observational Procedure), Alpha Testing (Observational Procedure), Beta Testing (Observational Procedure), Youth Screening Pathway & Brief Follow-Up Interview (Observational Procedure)
- Other
- Lead sponsor
- University of Texas Southwestern Medical Center
- Other
- Eligibility
- 7 Years and older
- Enrollment
- 200 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2026 – 2027
- U.S. locations
- 1
- States / cities
- Dallas, Texas
- Conditions
- Amyloidosis
- Interventions
- Doxycycline 100 mg po bid x 12 months
- Drug
- Lead sponsor
- Boston University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2012 – 2015
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Atherosclerosis, Coronary Artery Disease
- Interventions
- lovastatin
- Drug
- Lead sponsor
- Merck Sharp & Dohme LLC
- Industry
- Eligibility
- 21 Years to 67 Years
- Enrollment
- 270 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1985 – 1992
- U.S. locations
- 1
- States / cities
- Los Angeles, California
- Conditions
- Alzheimer Disease
- Interventions
- Extra virgin olive oil
- Dietary Supplement
- Lead sponsor
- Augusta University
- Other
- Eligibility
- 30 Years to 55 Years
- Enrollment
- 40 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2023 – 2025
- U.S. locations
- 1
- States / cities
- Augusta, Georgia
- Conditions
- Type 1 Diabetes Mellitus
- Interventions
- Family Teamwork Intervention
- Behavioral
- Lead sponsor
- Joslin Diabetes Center
- Other
- Eligibility
- 8 Years to 17 Years
- Enrollment
- 105 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 1999 – 2002
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Substance Abuse, Post-partum Depression, Depression
- Interventions
- Family Spirit Intervention
- Procedure
- Lead sponsor
- Johns Hopkins Bloomberg School of Public Health
- Other
- Eligibility
- 12 Years to 19 Years · Female only
- Enrollment
- 322 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2006 – 2010
- U.S. locations
- 5
- States / cities
- Fort Defiance, Arizona • San Carlos, Arizona • Tuba City, Arizona + 2 more
- Conditions
- Diet, Healthy, Physical Activity, Obesity, Childhood
- Interventions
- Policy/program
- Behavioral
- Lead sponsor
- University of South Carolina
- Other
- Eligibility
- 2 Years to 4 Years
- Enrollment
- 300 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2017 – 2023
- U.S. locations
- 2
- States / cities
- Baltimore, Maryland • Columbia, South Carolina
- Conditions
- HIV Infections
- Interventions
- Family Centered Advanced Care Planning (FCACP) sessions, Health education control sessions
- Behavioral
- Lead sponsor
- Maureen Lyon
- Other
- Eligibility
- 12 Years and older
- Enrollment
- 82 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2005 – 2008
- U.S. locations
- 1
- States / cities
- Washington D.C., District of Columbia
- Conditions
- Familial Hypophosphatemic Rickets
- Interventions
- Cinacalcet
- Drug
- Lead sponsor
- Children's Mercy Hospital Kansas City
- Other
- Eligibility
- 5 Years to 21 Years
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2009 – 2012
- U.S. locations
- 1
- States / cities
- Kansas City, Missouri
- Conditions
- Arteriosclerosis, Hypertension, Heart Diseases
- Interventions
- Not listed
- Lead sponsor
- The University of Texas Health Science Center, Houston
- Other
- Eligibility
- Up to 64 Years
- Timeline
- Started 1995
- U.S. locations
- 3
- States / cities
- Rochester, Minnesota • Jackson, Mississippi • Rio Grande City, Texas
- Conditions
- Opioid-Related Disorders, Stimulant-Related Disorder, Child Neglect, Mental Health Impairment
- Interventions
- Prevention Adaptation of Families Actively Improving Relationships (PRE-FAIR), Standard Case Management and Referral
- Behavioral
- Lead sponsor
- Chestnut Health Systems
- Other
- Eligibility
- 16 Years to 30 Years
- Enrollment
- 241 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2021 – 2025
- U.S. locations
- 1
- States / cities
- Eugene, Oregon
- Conditions
- Autism
- Interventions
- Primary Care Stepping Stones Triple P (PC-SS Triple P), Treatment as Usual
- Behavioral · Other
- Lead sponsor
- St. Louis University
- Other
- Eligibility
- 18 Years and older
- Enrollment
- 26 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2014 – 2015
- U.S. locations
- 1
- States / cities
- St Louis, Missouri
- Conditions
- Heterozygous Familial Hypercholesterolemia
- Interventions
- Evolocumab, Placebo
- Drug
- Lead sponsor
- Amgen
- Industry
- Eligibility
- 10 Years to 17 Years
- Enrollment
- 158 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2016 – 2019
- U.S. locations
- 12
- States / cities
- Farmington, Connecticut • Wilmington, Delaware • Iowa City, Iowa + 9 more
- Conditions
- Congenital Adrenal Hyperplasia (CAH), Familial Male-Limited Precocious Puberty (FMPP)
- Interventions
- Not listed
- Lead sponsor
- Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
- NIH
- Eligibility
- 1 Day to 99 Years
- Enrollment
- 3,000 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- Started 2006
- U.S. locations
- 2
- States / cities
- Washington D.C., District of Columbia • Bethesda, Maryland
- Conditions
- Asthma
- Interventions
- MyAsthma Web Portal
- Other
- Lead sponsor
- Children's Hospital of Philadelphia
- Other
- Eligibility
- Not listed
- Enrollment
- 294 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2013 – 2015
- U.S. locations
- 2
- States / cities
- Elk Grove Village, Illinois • Philadelphia, Pennsylvania
- Conditions
- Diabetes
- Interventions
- Not listed
- Lead sponsor
- Baylor College of Medicine
- Other
- Eligibility
- 18 Years to 65 Years
- Enrollment
- 142 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2008 – 2009
- U.S. locations
- 1
- States / cities
- Houston, Texas
- Conditions
- Sleep Problems, Sleep Hygiene
- Interventions
- Good Nights Sleep Program
- Behavioral
- Lead sponsor
- Auburn University
- Other
- Eligibility
- 7 Years to 10 Years
- Enrollment
- 60 participants
- Healthy volunteers
- Accepts healthy volunteers
- Timeline
- 2024
- U.S. locations
- 1
- States / cities
- Auburn, Alabama
- Conditions
- Obsessive-Compulsive Disorder
- Interventions
- Cognitive Behavioral Therapy (CBT), Relaxation Training
- Behavioral
- Lead sponsor
- Rhode Island Hospital
- Other
- Eligibility
- 5 Years to 8 Years
- Enrollment
- 38 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2002 – 2006
- U.S. locations
- 1
- States / cities
- Providence, Rhode Island
- Conditions
- PAH
- Interventions
- 75mg CXA-10, 150mg CXA-10, Placebo
- Drug · Other
- Lead sponsor
- Complexa, Inc.
- Industry
- Eligibility
- 18 Years to 80 Years
- Enrollment
- 69 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2020
- U.S. locations
- 40
- States / cities
- Birmingham, Alabama • Phoenix, Arizona • Tucson, Arizona + 34 more
- Conditions
- Substance Use Disorders, Risk Behavior, Psychiatric Disorders, Family Dysfunction
- Interventions
- Family Therapy based on Structural Family Therapy, Group Therapy, Psychoeducational Sessions, Individual Adolescent Therapy Sessions
- Behavioral
- Lead sponsor
- University of Miami
- Other
- Eligibility
- 14 Years to 17 Years
- Enrollment
- 190 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2015
- U.S. locations
- 1
- States / cities
- Miami, Florida