Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Oct 15, 2026, 5:31 PM EDT

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Showing 505–528 of 1,605 matching trials from the live ClinicalTrials.gov search.
Conditions
Cardiac Amyloidosis
Interventions
I-124 evuzamitide
Drug
Lead sponsor
Sharmila Dorbala
Other
Eligibility
18 Years and older
Enrollment
204 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
20
States / cities
Scottsdale, Arizona • Duarte, California • San Francisco, California + 16 more
Conditions
Lysosomal Acid Lipase Deficiency, Wolman Disease
Interventions
Sebelipase alfa (SBC-102)
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
Up to 24 Months
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2018
U.S. locations
1
States / cities
Irvine, California
Conditions
Inherited Metabolic Disorders (IMD)
Interventions
Safety and efficacy assessments
Other
Lead sponsor
Magenta Therapeutics, Inc.
Industry
Eligibility
Up to 16 Years
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2020
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Niemann-Pick Type C Disease, GM1 Gangliosidosis, GM2 Gangliosidosis
Interventions
AZ-3102, Placebo
Drug
Lead sponsor
Azafaros B.V.
Industry
Eligibility
4 Years and older
Enrollment
147 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
5
States / cities
Oakland, California • Minneapolis, Minnesota • Rochester, Minnesota + 2 more
Conditions
Cholesterol Ester Storage Disease (CESD), Lysosomal Acid Lipase Deficiency, LAL-Deficiency
Interventions
sebelipase alfa
Drug
Lead sponsor
Alexion Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2017
U.S. locations
5
States / cities
Eureka, California • Sacramento, California • San Francisco, California + 2 more
Conditions
Fed
Interventions
Desloratadine
Drug
Lead sponsor
Dr. Reddy's Laboratories Limited
Industry
Eligibility
18 Years to 45 Years
Enrollment
28 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2005 – 2006
U.S. locations
1
States / cities
Fort Myers, Florida
Conditions
Wilson Disease
Interventions
Not listed
Lead sponsor
Ultragenyx Pharmaceutical Inc
Industry
Eligibility
12 Years and older
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2022
U.S. locations
5
States / cities
New Haven, Connecticut • Miami, Florida • Chicago, Illinois + 2 more
Conditions
Inborn Errors of Metabolism, Pregnancy, Acidemias
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
14 Years to 50 Years · Female only
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2018
U.S. locations
2
States / cities
Bethesda, Maryland
Conditions
Enchondromatosis
Interventions
Not listed
Lead sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)
NIH
Eligibility
2 Years to 100 Years
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2030
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Hematologic Malignancies, Nonmalignant Diseases, Immunodeficiencies, Hemoglobinopathies, Genetic Inborn Errors of Metabolism, Fanconi Anemia, Thalassemia, Sickle Cell Disease
Interventions
Fludarabine
Drug
Lead sponsor
University of California, San Francisco
Other
Eligibility
Up to 17 Years
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
1
States / cities
San Francisco, California
Conditions
Gout, Gouty Arthritis
Interventions
Pharmacist-Led
Other
Lead sponsor
VA Office of Research and Development
Federal
Eligibility
18 Years and older
Enrollment
310 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2031
U.S. locations
5
States / cities
Omaha, Nebraska • Portland, Oregon • Philadelphia, Pennsylvania + 2 more
Conditions
Transthyretin (TTR) Amyloid Cardiomyopathy
Interventions
AI Toolkit for ATTR-CM Diagnosis
Diagnostic Test
Lead sponsor
Yale University
Other
Eligibility
50 Years to 95 Years
Enrollment
1,500,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
12
States / cities
San Francisco, California • New Haven, Connecticut • Chicago, Illinois + 9 more
Completed Phase 4 Interventional Results available

Examining the Effect of Burosumab on Muscle Function

NCT04146935
Conditions
X-linked Hypophosphatemia
Interventions
Burosumab Injection [Crysvita]
Drug
Lead sponsor
Yale University
Other
Eligibility
18 Years to 65 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
New Haven, Connecticut
Conditions
Pseudohypoparathyroidism and Pseudopseudohypoparathyroidism, Pseudohypoparathyroidism Type Ia, Albright Hereditary Osteodystrophy
Interventions
Not listed
Lead sponsor
Vanderbilt University Medical Center
Other
Eligibility
6 Years to 50 Years
Enrollment
14 participants
Timeline
2019 – 2021
U.S. locations
1
States / cities
Nashville, Tennessee
Conditions
Generalized Lipodystrophy
Interventions
Metreleptin
Drug
Lead sponsor
Aegerion Pharmaceuticals, Inc.
Industry
Eligibility
1 Year and older
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
8
States / cities
Birmingham, Alabama • New Orleans, Louisiana • Ann Arbor, Michigan + 4 more
Recruiting No phase listed Observational

NYSCF Scientific Discovery Biobank

NCT06203106
Conditions
ALS, Amyotrophic Lateral Sclerosis, Alzheimer Disease, Alzheimer Disease, Early Onset, Alzheimer Disease, Late Onset, Batten Disease, Corticobasal Degeneration, Dementia, Frontotemporal Dementia, Huntington Disease, Lewy Body Disease, Multiple Sclerosis, Multiple System Atrophy, Parkinson Disease, Parkinson's Disease and Parkinsonism, Progressive Supranuclear Palsy, INAD, Diabetes, Diabetes Mellitus, Diabetes Mellitus, Type 2, Diabetes Mellitus, Type 1, Macular Degeneration, Ovarian Cancer, Cervical Cancer, Uterine Cancer, Vaginal Cancer, Vulvar Cancer, PTSD, Post Traumatic Stress Disorder
Interventions
Biological Sample Collection
Other
Lead sponsor
New York Stem Cell Foundation Research Institute
Other
Eligibility
30 Days and older
Enrollment
10,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2045
U.S. locations
1
States / cities
New York, New York
Completed Phase 2Phase 3 Interventional Results available

Stem Cell Transplant for Bone Marrow Failure Syndromes

NCT00176878
Conditions
Diamond-Blackfan Anemia, Kostmann's Neutropenia, Shwachman-Diamond Syndrome
Interventions
Stem cell transplant, Fludarabine monophosphate, Total lymphoid irradiation, Busulfan, anti-thymocyte globulin
Procedure · Drug · Biological
Lead sponsor
Masonic Cancer Center, University of Minnesota
Other
Eligibility
Up to 35 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2000 – 2009
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Congenital Adrenal Hyperplasia
Interventions
Tildacerfont/Placebo
Drug
Lead sponsor
Spruce Biosciences
Industry
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
18
States / cities
Birmingham, Alabama • Los Angeles, California • San Diego, California + 13 more
Completed No phase listed Observational Accepts healthy volunteers

Detecting Dopaminergic Deficits in Individuals At-risk for Parkinsonism

NCT01358474
Conditions
Parkinson Disease, Gaucher Disease, Idiopathic Rapid Eye Movement Sleep Disorder
Interventions
Not listed
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years to 85 Years
Enrollment
56 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2018
U.S. locations
1
States / cities
Minneapolis, Minnesota
Conditions
Phenylketonuria
Interventions
Kuvan
Drug
Lead sponsor
Washington University School of Medicine
Other
Eligibility
6 Years to 18 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2010
U.S. locations
4
States / cities
Chicago, Illinois • Columbia, Missouri • St Louis, Missouri + 1 more
Conditions
Cytomegalovirus Retinitis, HIV Infections
Interventions
Foscarnet sodium, Ganciclovir
Drug
Lead sponsor
National Institute of Allergy and Infectious Diseases (NIAID)
NIH
Eligibility
18 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
9
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 4 more
Terminated Not applicable Interventional Accepts healthy volunteers Results available

Bone Mineral Density in Adults With Hyperphenylalaninemia on Kuvan Therapy

NCT01541397
Conditions
Hyperphenylalaninemia, Phenylketonuria
Interventions
Sapropterin
Drug
Lead sponsor
The University of Texas Health Science Center, Houston
Other
Eligibility
18 Years to 50 Years
Enrollment
6 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2012
U.S. locations
1
States / cities
Houston, Texas
Conditions
Mild Cognitive Impairment
Interventions
Not listed
Lead sponsor
Pamlab, Inc.
Industry
Eligibility
55 Years to 89 Years
Enrollment
700 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2014
U.S. locations
1
States / cities
Springfield, Massachusetts
Conditions
GM2 Gangliosidoses, Tay-Sachs, Sandhoff Disease
Interventions
Zavesca (Miglustat)
Drug
Lead sponsor
Children's National Research Institute
Other
Eligibility
6 Months to 5 Years
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2007
U.S. locations
1
States / cities
Washington D.C., District of Columbia