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ClinicalTrials.gov public records Last synced Jul 21, 2026, 10:25 PM EDT

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Showing 25–48 of 24 matching trials from the live ClinicalTrials.gov search.
Local D1 index available.
Conditions
Fabry Disease
Interventions
migalastat HCl 150 mg
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
12 Years to 17 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
5
States / cities
Tampa, Florida • Atlanta, Georgia • Minneapolis, Minnesota + 2 more
Conditions
Fabry Disease
Interventions
migalastat hydrochloride, agalsidase
Drug · Biological
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
16 Years to 74 Years
Enrollment
68 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
8
States / cities
Los Angeles, California • Aurora, Colorado • Decatur, Georgia + 5 more
Enrolling by invitation Not applicable Interventional Accepts healthy volunteers

Palliative Care Needs of Children With Rare Diseases and Their Families

NCT06938542
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
Congenital Heart Disease, Bicuspid Aortic Valve
Interventions
Not listed
Lead sponsor
Emory University
Other
Eligibility
10 Years to 18 Years
Enrollment
45 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2006 – 2012
U.S. locations
1
States / cities
Atlanta, Georgia
Recruiting No phase listed Observational

PDA for Kidneys Study

NCT06658496
Conditions
Patent Ductus Arteriosus, Congenital Heart Disease
Interventions
Near Infrared Spectroscopy
Device
Lead sponsor
University of Wisconsin, Madison
Other
Eligibility
1 Minute to 7 Days
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Madison, Wisconsin
Completed Not applicable Interventional Accepts healthy volunteers

Angiotensin II Antagonism of TGF-Beta 1

NCT00320970
Conditions
Diabetic Nephropathy, Hypertension
Interventions
Candesartan
Drug
Lead sponsor
Providence Health & Services
Other
Eligibility
18 Years and older
Enrollment
36 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2002 – 2004
U.S. locations
1
States / cities
Spokane, Washington
Conditions
Chronic Kidney Disease
Interventions
Vonapanitase, Placebo
Drug
Lead sponsor
Proteon Therapeutics
Industry
Eligibility
18 Years to 100 Years
Enrollment
696 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
36
States / cities
Birmingham, Alabama • Phoenix, Arizona • Tucson, Arizona + 30 more
Conditions
Down Syndrome, Bradycardia
Interventions
Monitoring patients undergoing surgery to look for association between ANS activity and bradycardia
Other
Lead sponsor
Children's Hospital Medical Center, Cincinnati
Other
Eligibility
1 Month to 8 Years
Enrollment
195 participants
Timeline
2022 – 2023
U.S. locations
1
States / cities
Cincinnati, Ohio
Conditions
Sensorineural Hearing Loss, Long QT Syndrome
Interventions
Not listed
Lead sponsor
Pediatrix
Other
Eligibility
1 Day to 90 Days
Enrollment
600 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2020
U.S. locations
12
States / cities
Anchorage, Alaska • Phoenix, Arizona • Waterford, Connecticut + 6 more
Conditions
Fabry Disease
Interventions
migalastat
Drug
Lead sponsor
Amicus Therapeutics
Industry
Eligibility
Female only
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Conditions
Coronary Disease
Interventions
Not listed
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
Up to 7 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
1
States / cities
New York, New York
Conditions
Congenital Disorders
Interventions
Not listed
Lead sponsor
Children's Healthcare of Atlanta
Other
Eligibility
18 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
1996 – 2008
U.S. locations
1
States / cities
Atlanta, Georgia
Conditions
Williams Syndrome, Lipedema
Interventions
Not listed
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
18 Years to 70 Years
Enrollment
144 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2018 – 2023
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Cancer, Cardiovascular Disease, Neurologic Dysfunction, Congenital Abnormalities, Hearing Loss
Interventions
Experimental
Behavioral
Lead sponsor
University of North Carolina, Chapel Hill
Other
Eligibility
Not listed
Enrollment
645 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Conditions
Aortic Stenosis, Hypoplastic Left Heart Syndrome
Interventions
Fetal Aortic Valvuloplasty
Procedure
Lead sponsor
University of California, San Francisco
Other
Eligibility
16 Years and older · Female only
Enrollment
9 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2012 – 2022
U.S. locations
1
States / cities
San Francisco, California
Conditions
Infantile Hemangiomas
Interventions
Timolol 0.5% Gel Forming Solution (GFS), Mupirocin 2% Ointment
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
1 Month to 8 Months
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2014
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Conditions
Heart Defects, Congenital
Interventions
Not listed
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
Up to 24 Months
Enrollment
78 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
Oak Lawn, Illinois
Completed Phase 1Phase 2 Interventional

Fibrosis and the Fontan

NCT04901975
Conditions
Single-ventricle
Interventions
Spironolactone
Drug
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
1 Year to 6 Years
Enrollment
98 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2026
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Conditions
Congenital Heart Disease
Interventions
Dexmedetomidine, Placebo
Drug
Lead sponsor
Aymen N Naguib
Other
Eligibility
1 Month to 12 Months
Enrollment
30 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2024
U.S. locations
1
States / cities
Columbus, Ohio
Conditions
Ventricular Septal Defect, Complete Atrioventricular Canal, Tetralogy of Fallot With Pulmonary Stenosis
Interventions
Fluorescite, Cellvizio 100 Series System with Confocal Miniprobes
Drug · Device
Lead sponsor
Aditya Kaza
Other
Eligibility
30 Days to 18 Years
Enrollment
70 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Danon Disease
Interventions
RP-A501
Biological
Lead sponsor
Rocket Pharmaceuticals Inc.
Industry
Eligibility
8 Years and older · Male only
Enrollment
7 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2025
U.S. locations
3
States / cities
La Jolla, California • Aurora, Colorado • Philadelphia, Pennsylvania
Completed Not applicable Interventional

Re-Energize Fontan

NCT04195451
Conditions
Single-ventricle
Interventions
Live-Video-Supervised Exercise Intervention, Usual Care then Live-Video Supervised Exercise Intervention
Other
Lead sponsor
Stanford University
Other
Eligibility
8 Years to 19 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2026
U.S. locations
1
States / cities
Palo Alto, California
Conditions
Atrial Septal Defect
Interventions
AMPLATZER Septal Occluder
Device
Lead sponsor
Abbott Medical Devices
Industry
Eligibility
Not listed
Enrollment
1,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2014
U.S. locations
51
States / cities
Birmingham, Alabama • Los Angeles, California • Madera, California + 44 more
Conditions
Congenital Heart Disease, Post Cardiac Surgery
Interventions
Daily Messages, Virtual Home Visits, Usual Care
Behavioral · Other
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
2 Weeks to 5 Months
Enrollment
218 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2017
U.S. locations
2
States / cities
Cincinnati, Ohio • Philadelphia, Pennsylvania