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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: 23
States / cities: Palo Alto, California • Sacramento, California • Aurora, Colorado + 19 more

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: Heart Valve Prosthesis
Interventions: INSPIRIS Resilia (model 11500A); Device
Lead sponsor: Baylor College of Medicine; Other
Eligibility: 0 Years to 64 Years
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2023 – 2025
U.S. locations: 1
States / cities: San Antonio, Texas

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: 21
States / cities: Los Angeles, California • Orange, California • Aurora, Colorado + 18 more

Conditions: Congenital Heart Disease
Interventions: Not listed
Lead sponsor: Medical College of Wisconsin; Other
Eligibility: 6 Years to 20 Years
Enrollment: 51 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2007 – 2012
U.S. locations: 1
States / cities: Milwaukee, Wisconsin

Conditions: Neurodevelopmental Disorders, Neurodevelopmental Disorders (NDD), Neurodevelopmental Disorders and Developmental Abnormalities, Developmental Delay (Disorder), Cerebral Palsy (CP), Cerebral Palsy Hemiparetic Cerebral Palsy Spasticity Gait Disorders, Neurologic Postural Balance Impairment, Cerebral Palsy Infantile, Cerebral Palsy Spastic Hemiplegic, Cerebral Palsy, Dyskinetic, Autism Spectrum Disorder, Autism Spectrum Disorder (ASD, Hypoxic Ischemic Encephalopathy, Hypoxic Ischemic Encephalopathy (HIE), Traumatic Brain Injury (TBI), Sensory Processing Disorder, Chromosomal Abnormalities, Genetic Disorders, Down Syndrome (Trisomy 21), Fragile X Syndrome (FXS), RETT Syndrome With Proven MECP2 Mutation, Williams Syndrome, 22q11.2 Deletion Syndrome, Sensorimotor Integration
Interventions: Not listed
Lead sponsor: Healing Hope International; Other
Eligibility: 4 Years to 12 Years
Enrollment: 100 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2036
U.S. locations: 1
States / cities: The Woodlands, Texas

Conditions: Alagille Syndrome, Cholestasis, Biliary Atresia
Interventions: magnesium gluconate, magnesium sulfate; Drug
Lead sponsor: National Center for Research Resources (NCRR); NIH
Eligibility: 3 Years to 18 Years
Enrollment: 25 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: Started 2000
U.S. locations: 1
States / cities: Cincinnati, Ohio

Conditions: Cardiac Anomalies
Interventions: AI Pocus_Cardiac; Diagnostic Test
Lead sponsor: Nicklaus Children's Hospital f/k/a Miami Children's Hospital; Other
Eligibility: 1 Month to 21 Years
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2026 – 2028
U.S. locations: 1
States / cities: Miami, Florida

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

Conditions: Congenital Heart Disease
Interventions: Strong Hearts Exercise Program; Behavioral
Lead sponsor: Duke University; Other
Eligibility: 10 Years to 50 Years
Enrollment: 9 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2024
U.S. locations: 1
States / cities: Durham, North Carolina

Conditions: Heart Failure, Cardiogenic Shock, Congenital Heart Disease, Cardiomyopathies
Interventions: Cardiohelp device (VA-ECMO), Heparin, Bivalirudin; Device · Drug
Lead sponsor: Stanford University; Other
Eligibility: 0 Years to 16 Years
Enrollment: 50 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2025 – 2029
U.S. locations: 5
States / cities: Palo Alto, California • New York, New York • Durham, North Carolina + 2 more

Conditions: Heart Defects, Congenital, Heart Septal Defects, Ventricular, Heart Failure, Congestive
Interventions: Enalapril, Placebo; Drug · Other
Lead sponsor: University of Utah; Other
Eligibility: Up to 18 Years
Enrollment: 5 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2006
U.S. locations: 6
States / cities: Boston, Massachusetts • New York, New York • Durham, North Carolina + 3 more

Conditions: Pulmonary Hypertension, Heart Failure, Single Ventricle
Interventions: Actigraph to check activity levels at home; Device
Lead sponsor: Children's Mercy Hospital Kansas City; Other
Eligibility: 8 Years to 18 Years
Enrollment: 56 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2016 – 2022
U.S. locations: 1
States / cities: Kansas City, Missouri

Conditions: Tetralogy of Fallot (TOF), Ventricular Septal Defects (VSD), Atrioventricular Septal Defects (AVSD)
Interventions: Fentanyl (High Dose), Fentanyl (Low Dose), Fentanyl (Low Dose) + Dexmedetomidine; Drug
Lead sponsor: Nationwide Children's Hospital; Other
Eligibility: 1 Month to 3 Years
Enrollment: 52 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2013
U.S. locations: 1
States / cities: Columbus, Ohio

Conditions: Marfan Syndrome
Interventions: Exercise; Behavioral
Lead sponsor: Stanford University; Other
Eligibility: 10 Years to 19 Years
Enrollment: 24 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2017 – 2019
U.S. locations: 1
States / cities: Palo Alto, California

Conditions: Congenital Heart Disease
Interventions: Angiotensin Converting Enzyme Inhibitor, No ACE Inhibitor; Drug · Other
Lead sponsor: Vanderbilt University Medical Center; Other
Eligibility: Up to 17 Years
Enrollment: 21 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2008 – 2010
U.S. locations: 1
States / cities: Nashville, Tennessee

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: Heart Disease, Congenital, Single Ventricle, Hypoplastic Left Heart Syndrome
Interventions: Not listed
Lead sponsor: Emory University; Other
Eligibility: Not listed
Enrollment: 30 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005
U.S. locations: 1
States / cities: Atlanta, Georgia

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: 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: Congenital Heart Defects
Interventions: Not listed
Lead sponsor: Emory University; Other
Eligibility: Up to 21 Years
Enrollment: 200 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2005 – 2006
U.S. locations: 1
States / cities: Atlanta, Georgia

Conditions: Intellectual Disability, Neurodevelopmental Disorders, Autism Spectrum Disorder, Down Syndrome, Fragile X Syndrome, Cri-du-Chat Syndrome, De Lange Syndrome, Mental Retardation, X-Linked, Prader-Willi Syndrome, Rubinstein-Taybi Syndrome, Trisomy 13 Syndrome, WAGR Syndrome, Williams Syndrome
Interventions: PACE Program; Behavioral
Lead sponsor: University of North Carolina, Chapel Hill; Other
Eligibility: 18 Years and older
Enrollment: 376 participants
Healthy volunteers: Accepts healthy volunteers
Timeline: 2025 – 2028
U.S. locations: 2
States / cities: Fayetteville, Arkansas • Chapel Hill, North Carolina

Conditions: Hypoplastic Left Heart Syndrome (HLHS)
Interventions: Collaborative Learning Network; Other
Lead sponsor: Children's Hospital Medical Center, Cincinnati; Other
Eligibility: Up to 15 Months
Enrollment: 1,000 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2016 – 2028
U.S. locations: 61
States / cities: Birmingham, Alabama • Phoenix, Arizona • Little Rock, Arkansas + 53 more

Conditions: Congenital Heart Defects, Blood Platelet Disorders
Interventions: Clopidogrel (SR25990); Drug
Lead sponsor: Sanofi; Industry
Eligibility: Up to 24 Months
Enrollment: 92 participants
Healthy volunteers: Healthy volunteers not accepted
Timeline: 2004 – 2006
U.S. locations: 1
States / cities: Bridgewater, New Jersey

Search by objective public record fields.

Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders

Palliative Care Needs of Children With Rare Diseases and Their Families

Outcomes of Pulmonary Valve Replacement With the INSPIRIS (Model 11500A) Valve

A Study of Vosoritide in Children With Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment

Multi-site Near Infrared Spectroscopy (NIRS) Monitoring of Children During Exercise

Intensive Multimodal Neurorehabilitation Targeting Neuroplasticity in Pediatric Neurodevelopmental and Chromosomal Disorders

Study of Magnesium Sulfate in Children With Reduced Bone Density Secondary to Chronic Cholestatic Liver Disease

Prospective Evaluation of Cardiac Point-of-Care Ultrasound (POCUS) Performed by PEM Fellows and Artificial Intelligence on Children With Pre-existing Cardiac Conditions.

A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9

Strong Hearts: A Remote, App-Enabled, Exercise Program for Patients With Congenital Heart Disease (Strong Hearts App)

Multicenter Trial of ECMO in Children With Severe Cardiac Failure Using the Cardiohelp System

Angiotensin Converting Enzyme Inhibition in Children With Mitral Regurgitation

Frailty in Children With Cardiac Disease

Measures to Lower the Stress Response in Pediatric Cardiac Surgery

Children and Adolescents With Marfan Syndrome: 10,000 Healthy Steps and Beyond

Angiotensin Converting Enzyme Inhibitors During Cardiopulmonary Bypass in Infants and Children

A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to Grow

Single Ventricle Outcome

Open-Label Study of ZYN002 Administered as a Transdermal Gel to Children and Adolescents With 22q11.2 Deletion Syndrome (INSPIRE)

Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial

Using Tissue Doppler/Synchronization to Determine Heart Function in Children With Congenital Heart Disease

Physical Activity and Community EmPOWERment Project

National Collaborative to Improve Care of Children With Complex Congenital Heart Disease

Platelet Aggregation Inhibition in Children on Clopidogrel (PICOLO)