Independent directory Public ClinicalTrials.gov records United States
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ClinicalTrials.gov public records Last synced Oct 17, 2026, 9:12 AM EDT

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Showing 49–72 of 270 matching trials from the live ClinicalTrials.gov search.
Conditions
FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis
Interventions
Inotersen, Placebo
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years to 82 Years
Enrollment
173 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
9
States / cities
Orange, California • Indianapolis, Indiana • Baltimore, Maryland + 5 more
Conditions
Familial Dysautonomia
Interventions
CBT sessions, Talking Sessions
Behavioral
Lead sponsor
NYU Langone Health
Other
Eligibility
18 Years to 80 Years
Healthy volunteers
Accepts healthy volunteers
Timeline
2019 – 2021
U.S. locations
1
States / cities
New York, New York
Conditions
Kidney Cancer, Leukemia, Lymphoma, Neurotoxicity, Peripheral Neuropathy, Sarcoma
Interventions
Glutamine, Placebo
Drug · Other
Lead sponsor
Columbia University
Other
Eligibility
5 Years to 21 Years
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2012
U.S. locations
1
States / cities
New York, New York
Conditions
Breast Cancer, Solid Tumors, Adult
Interventions
STX-478, Fulvestrant, Ribociclib, Palbociclib, Letrozole, Anastrozole, Exemestane, Tamoxifen, Abemaciclib, Imlunestrant, Metformin
Drug
Lead sponsor
Eli Lilly and Company
Industry
Eligibility
18 Years and older
Enrollment
880 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2030
U.S. locations
28
States / cities
Los Angeles, California • San Francisco, California • Aurora, Colorado + 22 more
Conditions
Cardiac Amyloidosis, Heart Diseases, TTR Cardiac Amyloidosis
Interventions
Beta blocker
Drug
Lead sponsor
Weill Medical College of Cornell University
Other
Eligibility
65 Years and older
Enrollment
14 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
New York, New York
Conditions
AMPD3, OMIM*102772, AMP Deaminase Deficiency, AK1, OMIM *103000, Adenylate Kinase Deficiency, AMPD1, OMIM *102770, Myopathy Due to Myoadenylate Deaminase Deficiency, TPMT, OMIM *187680, Thoipurines, Poor Metabolism of, IMPDH1, OMIM *146690, Retinitis Pigmentosa Type 10, Leber Congenital Amauriosis Type 11, APRT, OMIM *102600, Adenine Phosphoribosyltransferase Deficiency, HPRT1, OMIM *308000 Lesch-Nyhan Disease, XDH, OMIM *607633, Xanthinuria Type 1, SLC2A9, OMIM *606142 Hypouricemia, SLC22A12, OMIM *607096 Hypouricemia, PRPS1 Def, OMIM *311850, Arts Syndrome; Charcot-Marie-Tooth Disease, PRPS1 SA, OMIM *311850 Gout, PRPS-related Phosphoribosylpyrophosphate Synthetase Superactivity, AMPD2, OMIM *102771, Spastic Paraplegia 63; Pontocerebellar Hypoplasia, ITPA, OMIM *147520, Inosine Triphosphatase Deficiency; Developmental and Epileptic Encephalopathy 35, ADSL, OMIM *608222, Adenylosuccinate Lyase Deficiency, PNP, OMIM *164050, Nucleoside Phosphorylase Deficiency, ADA2, OMIM *607575,Sneddon Syndrome; VAIHS, CAD, *1140120, Developmental and Epileptic Encephalopathy, UPB1, OMIM *606673, Beta-ureidopropionase Deficiency, DPYS, OMIM *613326, Dihydropyrimidinase Deficiency, DPYD, OMIM *274270, Dihydropyrimidine Dehydrogenase Deficiency, DHODH, OMIM *126064, Miller Syndrome (Postaxial Acrofacial Dysostosis), UMPS, OMIM *613891, Orotic Aciduria, NT5C3A<TAB>, OMIM *606224, Anemia, Hemolytic, Due to UMPH1 Deficiency, UNG, OMIM *191525, Hyper-IgM Syndrome 5, AICDA, OMIM *605257, Immunodeficiency With Hyper-IgM, Type 2; HIGM2, Purine-Pyrimidine Metabolism, Metabolic Disease
Interventions
Not listed
Lead sponsor
National Human Genome Research Institute (NHGRI)
NIH
Eligibility
1 Month to 100 Years
Enrollment
999 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2099
U.S. locations
1
States / cities
Bethesda, Maryland
Conditions
Rheumatoid Arthritis, Diabetic Neuropathy, Fibromyalgia, Irritable Bowel Syndrome
Interventions
DiNaMo Study App, Placebo App
Device
Lead sponsor
Click Therapeutics, Inc.
Industry
Eligibility
18 Years to 65 Years
Enrollment
124 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2023
U.S. locations
1
States / cities
New York, New York
Conditions
Myelofibrosis
Interventions
Ruxolitinib, 9-ING-41
Drug
Lead sponsor
Actuate Therapeutics Inc.
Industry
Eligibility
18 Years and older
Enrollment
17 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2024
U.S. locations
9
States / cities
Los Angeles, California • Augusta, Georgia • Rochester, Minnesota + 5 more
Conditions
Charcot Marie Tooth Disease (CMT)
Interventions
Ankle-foot Orthoses
Device
Lead sponsor
Bopha Chrea
Other
Eligibility
25 Years to 55 Years
Enrollment
66 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
North Liberty, Iowa
Conditions
Rare Unresectable or Metastatic BRAF V600E Mutation-positive Solid Tumors
Interventions
Non-investigational
Other
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
1 Year to 100 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2028
U.S. locations
8
States / cities
Torrance, California • Denver, Colorado • Washington D.C., District of Columbia + 5 more
Conditions
Cardiomyopathy, Ischemic Heart Disease, Cardiac Amyloidosis
Interventions
Transthoracic Echocardiography (TTE)
Diagnostic Test
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years and older
Enrollment
10,040,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Rochester, Minnesota
Conditions
Cardiac Amyloidosis
Interventions
[64Cu]FBP8 PET/MR
Drug
Lead sponsor
Brigham and Women's Hospital
Other
Eligibility
18 Years and older
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Conditions
Charcot-Marie-Tooth Disease Type 1A
Interventions
PXT3003 dose 1, PXT3003 dose 2, placebo
Drug
Lead sponsor
Pharnext S.C.A.
Other
Eligibility
16 Years to 65 Years
Enrollment
323 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2018
U.S. locations
11
States / cities
Los Angeles, California • New Britain, Connecticut • Gainesville, Florida + 8 more
Conditions
Specific Advanced Solid Tumors
Interventions
ACR-2316
Drug
Lead sponsor
Acrivon Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
15
States / cities
Phoenix, Arizona • Beverly Hills, California • Newport Beach, California + 12 more
Conditions
Dysautonomia, Familial, Baroreflex Failure Syndrome
Interventions
Carbidopa Low-Dose, Placebo, Carbidopa High-Dose
Drug · Other
Lead sponsor
NYU Langone Health
Other
Eligibility
10 Years to 60 Years
Enrollment
22 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
2
States / cities
New York, New York
Conditions
Leber Hereditary Optic Neuropathy (LHON)
Interventions
NFS-02 Injection
Drug
Lead sponsor
Neurophth Therapeutics Inc
Other
Eligibility
18 Years to 75 Years
Enrollment
11 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2024
U.S. locations
1
States / cities
Aurora, Colorado
Conditions
Charcot-Marie-Tooth Disease, Peripheral Neuropathy, Chronic Inflammatory Demyelinating Polyneuropathy
Interventions
NT3 blood draw
Diagnostic Test
Lead sponsor
Zarife Sahenk
Other
Eligibility
7 Years and older
Enrollment
8 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020
U.S. locations
1
States / cities
Columbus, Ohio
Conditions
Transthyretin (TTR) Amyloid Cardiomyopathy
Interventions
Tafamidis, Placebo
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 90 Years
Enrollment
441 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
50
States / cities
Birmingham, Alabama • Phoenix, Arizona • Scottsdale, Arizona + 24 more
Conditions
Charcot Marie Tooth Disease (CMT), Hereditary Sensory and Motor Neuropathy, Nerve Compression Syndromes, Tooth Diseases, Congenital Abnormalities, Genetic Diseases, Inborn, Heredodegenerative Disorders, Nervous System
Interventions
Not listed
Lead sponsor
University of South Florida
Other
Eligibility
18 Years and older
Enrollment
411 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2012 – 2013
U.S. locations
1
States / cities
Tampa, Florida
Conditions
Transthyretin (TTR) Amyloid Cardiomyopathy
Interventions
Tafamidis
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
1,733 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
41
States / cities
Birmingham, Alabama • Beverly Hills, California • La Jolla, California + 20 more
Conditions
Breast Cancer
Interventions
Not listed
Lead sponsor
Ohio State University Comprehensive Cancer Center
Other
Eligibility
18 Years and older
Enrollment
62 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
1
States / cities
Columbus, Ohio
Conditions
Epigastric Pain, Retrosternal Pain, Esophagitis, Gastrointestinal Symptoms
Interventions
Not listed
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
5 Years to 17 Years
Enrollment
77 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2015
U.S. locations
1
States / cities
Milwaukee, Wisconsin
Conditions
Carpal Tunnel Syndrome, Charcot-Marie-Tooth Disease, Inherited Peripheral Neuropathy
Interventions
Web-based survey
Other
Lead sponsor
University of South Florida
Other
Eligibility
18 Years and older
Enrollment
333 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016
U.S. locations
1
States / cities
Tampa, Florida
Conditions
Amyloidosis, Hereditary, Amyloidosis Cardiac, Amyloidosis, Familial, Transthyretin-Related (ATTR) Familial Amyloid Cardiomyopathy, Transthyretin Gene Mutation
Interventions
Not listed
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
30 Years to 80 Years
Enrollment
500 participants
Timeline
2022 – 2027
U.S. locations
3
States / cities
New York, New York • Cleveland, Ohio • Dallas, Texas