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ClinicalTrials.gov public records Last synced Oct 17, 2026, 1:35 PM EDT

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Showing 145–168 of 270 matching trials from the live ClinicalTrials.gov search.
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Recruiting Phase 1Phase 2 Interventional

Evaluating Efficacy of Tivozanib (AV-951) in Biliary Tract Cancers

NCT04645160
View directory record Official record
Conditions
Cholangiocarcinoma, Bile Duct Neoplasm, Biliary Tract Malignancy
Interventions
Tivozanib
Drug
Lead sponsor
National Cancer Institute (NCI)
NIH
Eligibility
18 Years and older
Enrollment
31 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2029
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 17, 2026, 1:35 PM EDT
Completed Phase 3 Interventional Results available

Efficacy Study of GS010 for Treatment of Vision Loss From 7 Months to 1 Year From Onset in LHON Due to the ND4 Mutation (REVERSE)

NCT02652780
View directory record Official record
Conditions
Optic, Atrophy, Hereditary, Leber
Interventions
GS010, Sham Intravitreal Injection
Biological · Device
Lead sponsor
GenSight Biologics
Industry
Eligibility
15 Years and older
Enrollment
37 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2018
U.S. locations
3
States / cities
Los Angeles, California • Atlanta, Georgia • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Oct 17, 2026, 1:35 PM EDT
Withdrawn Phase 1Phase 2 Interventional

Phase I/IIa Trial of scAAV1.tMCK.NTF3 for Treatment of CMT1A

NCT03520751
View directory record Official record
Conditions
Charcot-Marie-Tooth Neuropathy Type 1A
Interventions
scAAV1.tMCK.NTF3
Drug
Lead sponsor
Nationwide Children's Hospital
Other
Eligibility
18 Years to 35 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2027 – 2030
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Oct 17, 2026, 1:35 PM EDT
Recruiting Phase 2 Interventional

A Study of Reduced-dose Radiation in People With Metastatic Tumors With a Genetic Change

NCT05010031
View directory record Official record
Conditions
Metastatic Cancer
Interventions
Palliative radiotherapy
Radiation
Lead sponsor
Memorial Sloan Kettering Cancer Center
Other
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
7
States / cities
Basking Ridge, New Jersey • Middletown, New Jersey • Montvale, New Jersey + 4 more
Source: ClinicalTrials.gov public record
Updated May 11, 2026 · Synced Oct 17, 2026, 1:35 PM EDT
Active, not recruiting Not applicable Interventional

Clinical Trial to Assess the Safety of a Novel Scaffold Biomaterial

NCT03584022
View directory record Official record
Conditions
Peripheral Neuropathy, Chronic Inflammatory Demyelinating Polyradiculoneuropathy, Vasculitic Neuropathy (Disorder), Amyloidosis, Hereditary Neuropathy, Sarcoid Neuropathy
Interventions
Biopsy + Nerve Repair, Biopsy Only
Device · Procedure
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years to 75 Years
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2027
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated May 1, 2026 · Synced Oct 17, 2026, 1:35 PM EDT
Active, not recruiting Phase 2 Interventional

A Phase 2 Study of RTB101 as COVID-19 Post-Exposure Prophylaxis in Older Adults

NCT04584710
View directory record Official record
Conditions
Covid19
Interventions
RTB101, Placebo
Drug
Lead sponsor
Restorbio Inc.
Industry
Eligibility
65 Years and older
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
2
States / cities
Farmington, Connecticut • Roslindale, Massachusetts
Source: ClinicalTrials.gov public record
Updated Feb 9, 2021 · Synced Oct 17, 2026, 1:35 PM EDT
Completed No phase listed Observational

Carpal Tunnel Syndrome and Amyloid Cardiomyopathy

NCT02792790
View directory record Official record
Conditions
Amyloidosis, Cardiomyopathy, Transthyretin Amyloidosis, Senile Systemic Amyloidosis, Carpal Tunnel Syndrome
Interventions
Not listed
Lead sponsor
The Cleveland Clinic
Other
Eligibility
50 Years and older
Enrollment
96 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
1
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 22, 2021 · Synced Oct 17, 2026, 1:35 PM EDT
Completed Phase 2 Interventional Results available

A Phase II Study of Single Agent MEK162 in Patients With Advanced Melanoma

NCT01320085
View directory record Official record
Conditions
BRAF or NRAS Mutant Metastatic Melanoma
Interventions
MEK162
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
183 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2023
U.S. locations
10
States / cities
Fayetteville, Arkansas • Tampa, Florida • Portland, Oregon + 3 more
Source: ClinicalTrials.gov public record
Updated Jan 24, 2024 · Synced Oct 17, 2026, 1:35 PM EDT
Recruiting No phase listed Observational

The Myelin Disorders Biorepository Project

NCT03047369
View directory record Official record
Conditions
Leukodystrophy, White Matter Disease, Leukoencephalopathies, 4H Syndrome, Adrenoleukodystrophy, AMN, ALD, ALD Gene Mutation, ALD (Adrenoleukodystrophy), X-linked Adrenoleukodystrophy, X-ALD, Adrenomyeloneuropathy, Aicardi Goutieres Syndrome, AGS, Alexander Disease, Alexanders Leukodystrophy, AxD, ADLD, Canavan Disease, CTX, Cerebrotendinous Xanthomatoses, Krabbe Disease, GALC Deficiency, Globoid Leukodystrophy, TUBB4A-Related Leukodystrophy, H-ABC - Hypomyelination, Atrophy of Basal Ganglia and Cerebellum, HBSL, HBSL - Hypomyelination, Brain Stem, Spinal Cord, Leg Spasticity, LBSL, Leukoencephalopathy With Brain Stem and Spinal Cord Involvement and High Lactate Syndrome (Disorder), Leukoencephalopathy With Brainstem and Spinal Cord Involvement and Lactate Elevation, ALSP, CSF1R Gene Mutation, HCC - Hypomyelination and Congenital Cataract, MLC1, Megalencephalic Leukoencephalopathy With Subcortical Cysts, MLD, Metachromatic Leukodystrophy, PMD, Pelizaeus-Merzbacher Disease, PLP1 Null Syndrome, PLP1 Gene Duplication | Blood or Tissue | Mutations, Pelizaeus Merzbacher Like Disease, Peroxisomal Biogenesis Disorder, Zellweger Syndrome, Refsum Disease, Salla Disease, Sialic Storage Disease, Sjögren, Sjogren-Larsson Syndrome, Van Der Knapp Disease, Vanishing White Matter Disease, Charcot-Marie-Tooth, CMT, Mct8 (Slc16A2)-Specific Thyroid Hormone Cell Transporter Deficiency, Allan-Herndon-Dudley Syndrome, Cadasil, Cockayne Syndrome, Multiple Sulfatase Deficiency, Gangliosidoses, GM2 Gangliosidosis, BPAN, Labrune Syndrome, LCC, Mucopolysaccharidoses, TBCK-Related Intellectual Disability Syndrome
Interventions
Not listed
Lead sponsor
Children's Hospital of Philadelphia
Other
Eligibility
Not listed
Enrollment
12,000 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2030
U.S. locations
23
States / cities
Los Angeles, California • Orange, California • Palo Alto, California + 18 more
Source: ClinicalTrials.gov public record
Updated Oct 23, 2025 · Synced Oct 17, 2026, 1:35 PM EDT
Completed Phase 1Phase 2 Interventional Results available

CB-839 + Capecitabine in Solid Tumors and Fluoropyrimidine Resistant PIK3CA Mutant Colorectal Cancer

NCT02861300
View directory record Official record
Conditions
Colorectal Cancer, Colon Cancer, Rectal Cancer, Solid Tumor
Interventions
CB-839, Capecitabine
Drug
Lead sponsor
David Bajor, MD
Other
Eligibility
19 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2023
U.S. locations
2
States / cities
Cleveland, Ohio
Source: ClinicalTrials.gov public record
Updated Dec 19, 2024 · Synced Oct 17, 2026, 1:35 PM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia

NCT04712812
View directory record Official record
Conditions
Hereditary Spastic Paraplegia, SPG47, SPG50, SPG51, SPG52, AP4-related Hereditary Spastic Paraplegia, Early Onset Hereditary Spastic Paraplegia, SPG4, SPG3A, SPG15, SPG11
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
Up to 30 Years
Enrollment
700 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2030
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 18, 2026 · Synced Oct 17, 2026, 1:35 PM EDT
Completed Phase 1 Interventional

Phase 1 Trial of Idebenone to Treat Patients With Friedreich's Ataxia

NCT00078481
View directory record Official record
Conditions
Friedreich Ataxia
Interventions
Idebenone
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2004 – 2006
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 17, 2026, 1:35 PM EDT
Completed Phase 2 Interventional

The Nutritional Supplement Phosphatidylserine in Patients With Familial Dysautonomia

NCT02276716
View directory record Official record
Conditions
Familial Dysautonomia
Interventions
Phosphatidylserine
Dietary Supplement
Lead sponsor
NYU Langone Health
Other
Eligibility
12 Years and older
Enrollment
26 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Feb 21, 2021 · Synced Oct 17, 2026, 1:35 PM EDT
Recruiting Phase 3 Interventional

Acoramidis Transthyretin Amyloidosis Prevention Trial in the Young (ACT-EARLY) Study in Asymptomatic Carriers of a Pathogenic TTR Variant

NCT06563895
View directory record Official record
Conditions
Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis, Cardiomyopathies, Heart Diseases, Polyneuropathies
Interventions
Acoramidis, Placebo oral tablet
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 75 Years
Enrollment
587 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
40
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 31 more
Source: ClinicalTrials.gov public record
Updated Aug 4, 2026 · Synced Oct 17, 2026, 1:35 PM EDT
Completed Not applicable Interventional

Proprioception and Sensorimotor Control in Hereditary Sensory and Autonomic Neuropathy

NCT02876939
View directory record Official record
Conditions
Atunomic Neuropathy
Interventions
kinesiology tape around the hip, knee and ankle joints, No kinesiology tape around the hip, knee and ankle joints
Other
Lead sponsor
NYU Langone Health
Other
Eligibility
16 Years to 40 Years
Enrollment
16 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 22, 2020 · Synced Oct 17, 2026, 1:35 PM EDT
Recruiting No phase listed Observational

Natural History Study of Patients with HPDL Mutations

NCT05848271
View directory record Official record
Conditions
Mitochondrial Encephalomyopathies, Hereditary Spastic Paraplegia, Spastic Paraplegia, White Matter Disease, Neonatal Encephalopathy, Mutation, Genetic Disease
Interventions
Patient Registry, Dry blood spots sampling
Other
Lead sponsor
University of California, San Diego
Other
Eligibility
Not listed
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2027
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Mar 30, 2025 · Synced Oct 17, 2026, 1:35 PM EDT
Completed Phase 1 Interventional

Study to Test How Well Patients With Advanced Solid Tumors Respond to Treatment With the Elimusertib in Combination With Pembrolizumab, to Find the Optimal Dose for Patients, How the Drug is Tolerated and the Way the Body Absorbs, Distributes and Discharges the Drug

NCT04095273
View directory record Official record
Conditions
Advanced Solid Tumors
Interventions
Elimusertib (BAY1895344), Pembrolizumab (Keytruda®)
Drug
Lead sponsor
Bayer
Industry
Eligibility
18 Years and older
Enrollment
56 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2023
U.S. locations
8
States / cities
Palo Alto, California • New Haven, Connecticut • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Apr 2, 2024 · Synced Oct 17, 2026, 1:35 PM EDT
Completed Phase 3 Interventional Results available

Setmelanotide (RM-493), Melanocortin-4 Receptor (MC4R) Agonist, in Bardet-Biedl Syndrome (BBS) and Alström Syndrome (AS) Participants With Moderate to Severe Obesity

NCT03746522
View directory record Official record
Conditions
Bardet Biedl Syndrome (BBS), Alström Syndrome (AS)
Interventions
Setmelanotide, Placebo
Drug
Lead sponsor
Rhythm Pharmaceuticals, Inc.
Industry
Eligibility
6 Years and older
Enrollment
52 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2021
U.S. locations
6
States / cities
San Diego, California • Springfield, Massachusetts • New York, New York + 3 more
Source: ClinicalTrials.gov public record
Updated Dec 1, 2023 · Synced Oct 17, 2026, 1:35 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Intracardiac Flow Assessment in Cardiac Amyloidosis

NCT05379101
View directory record Official record
Conditions
Amyloidosis Cardiac, Healthy Adults
Interventions
Cardiac magnetic resonance image (CMR), Transthoracic Echocardiogram (TTE), Six-minute Walk Test
Diagnostic Test
Lead sponsor
Mayo Clinic
Other
Eligibility
40 Years and older
Enrollment
81 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2026
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jun 29, 2026 · Synced Oct 17, 2026, 1:35 PM EDT
Active, not recruiting No phase listed Observational Accepts healthy volunteers

Phenotype, Genotype & Biomarkers in ALS and Related Disorders

NCT02327845
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, Primary Lateral Sclerosis, Hereditary Spastic Paraplegia, Progressive Muscular Atrophy, Multisystem Proteinopathy
Interventions
Not listed
Lead sponsor
University of Miami
Other
Eligibility
Not listed
Enrollment
708 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2015 – 2031
U.S. locations
13
States / cities
Palo Alto, California • San Diego, California • San Francisco, California + 10 more
Source: ClinicalTrials.gov public record
Updated Jun 15, 2026 · Synced Oct 17, 2026, 1:35 PM EDT
Active, not recruiting Phase 3 Interventional

Open-Label Safety Study of Acoramidis (AG10) in Symptomatic ATTR Participants

NCT04988386
View directory record Official record
Conditions
Amyloid Cardiomyopathy, Transthyretin-Related
Interventions
Acoramidis (AG10)
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 90 Years
Enrollment
389 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2028
U.S. locations
26
States / cities
Los Angeles, California • Aurora, Colorado • Chicago, Illinois + 21 more
Source: ClinicalTrials.gov public record
Updated Oct 14, 2025 · Synced Oct 17, 2026, 1:35 PM EDT
Completed Phase 2 Interventional

Tuvusertib Combined With Niraparib or Lartesertib in Participants With Epithelial Ovarian Cancer (DDRiver EOC 302)

NCT06433219
View directory record Official record
Conditions
Ovarian Cancer
Interventions
Tuvusertib (M1774), Niraparib, Lartesertib (M4076)
Drug
Lead sponsor
EMD Serono Research & Development Institute, Inc.
Industry
Eligibility
18 Years and older · Female only
Enrollment
63 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
9
States / cities
Little Rock, Arkansas • San Francisco, California • Columbus, Georgia + 4 more
Source: ClinicalTrials.gov public record
Updated Sep 14, 2026 · Synced Oct 17, 2026, 1:35 PM EDT
Not listed No phase listed Observational Accepts healthy volunteers

Charcot-Marie-Tooth Disease (CMT) Infant Scale (INC-6611)

NCT02979145
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease
Interventions
No intervention involved
Other
Lead sponsor
Sydney Children's Hospitals Network
Other
Eligibility
Up to 4 Years
Enrollment
200 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
Started 2016
U.S. locations
2
States / cities
Iowa City, Iowa • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 1, 2016 · Synced Oct 17, 2026, 1:35 PM EDT
Withdrawn Phase 2 Interventional

The Safety, Tolerability and Efficacy of Dronabinol, for the Treatment of Nausea and Vomiting in Familial Dysautonomia

NCT02608931
View directory record Official record
Conditions
Nausea, Vomiting, Familial Dysautonomia
Interventions
Dronabinol, Placebo
Drug · Other
Lead sponsor
NYU Langone Health
Other
Eligibility
18 Years to 60 Years
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2019
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jun 13, 2019 · Synced Oct 17, 2026, 1:35 PM EDT