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ClinicalTrials.gov public records Last synced Oct 17, 2026, 2:27 PM EDT

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Showing 169–192 of 270 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inherited Neuropathies Clear all
Completed Phase 3 Interventional

Efficacy of [18F]Florbetaben PET for Diagnosis of Cardiac AL Amyloidosis

NCT05184088
View directory record Official record
Conditions
Cardiac Amyloidosis, AL Amyloidosis, ATTR Amyloidosis
Interventions
[18F]florbetaben
Drug
Lead sponsor
Lantheus Germany GmbH
Industry
Eligibility
18 Years and older
Enrollment
244 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2026
U.S. locations
3
States / cities
Kansas City, Kansas • Philadelphia, Pennsylvania • Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Aug 3, 2026 · Synced Oct 17, 2026, 2:27 PM EDT
Terminated Early Phase 1 Interventional

Molecular Imaging of the Underlying Mechanism of Cardiotoxicity in Patients With Light Chain Amyloidosis Using PET/CT

NCT04105634
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
FNOS, Florbetaben
Drug
Lead sponsor
University of Pennsylvania
Other
Eligibility
18 Years and older
Enrollment
3 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Nov 12, 2024 · Synced Oct 17, 2026, 2:27 PM EDT
Terminated No phase listed Observational

Giant Axonal Neuropathy Natural History Study

NCT01503125
View directory record Official record
Conditions
Giant Axonal Neuropathy
Interventions
Not listed
Lead sponsor
Columbia University
Other
Eligibility
Not listed
Enrollment
13 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2015
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jan 27, 2017 · Synced Oct 17, 2026, 2:27 PM EDT
Recruiting Not applicable Interventional

Elevated Rate Pacing of Cardiac Amyloidosis

NCT07583823
View directory record Official record
Conditions
Cardiac Amyloidosis, Pacemaker
Interventions
Pacemaker Rate
Other
Lead sponsor
University of Minnesota
Other
Eligibility
18 Years and older
Enrollment
10 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2026
U.S. locations
1
States / cities
Minneapolis, Minnesota
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 17, 2026, 2:27 PM EDT
Completed Phase 1Phase 2 Interventional

Phase 1 and 2 Study of PX-866 and Cetuximab

NCT01252628
View directory record Official record
Conditions
Incurable Metastatic Colorectal Carcinoma, Incurable Progressive, Recurrent or Metastatic Squamous Cell Carcinoma of the Head and Neck
Interventions
PX-866 (SCCHN), Cetuximab (SCCHN), PX-866 (CRC), Cetuximab (CRC)
Drug
Lead sponsor
Cascadian Therapeutics Inc.
Industry
Eligibility
18 Years and older
Enrollment
178 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2014
U.S. locations
31
States / cities
Birmingham, Alabama • Escondido, California • Monterey, California + 24 more
Source: ClinicalTrials.gov public record
Updated May 16, 2018 · Synced Oct 17, 2026, 2:27 PM EDT
Completed Phase 3 Interventional Results available

Efficacy and Safety of RAD001 in Patients Aged 18 and Over With Angiomyolipoma Associated With Either Tuberous Sclerosis Complex (TSC) or Sporadic Lymphangioleiomyomatosis (LAM)

NCT00790400
View directory record Official record
Conditions
Tuberous Sclerosis Complex (TSC), Lymphangioleiomyomatosis (LAM)
Interventions
Everolimus (RAD001), Everolimus Placebo
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
118 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2015
U.S. locations
6
States / cities
Birmingham, Alabama • Phoenix, Arizona • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Feb 17, 2017 · Synced Oct 17, 2026, 2:27 PM EDT
Recruiting Not applicable Interventional Accepts healthy volunteers

Electrical Impedance Myography: Natural History Studies inNeuromuscular Disorders and Healthy Volunteers

NCT01900132
View directory record Official record
Conditions
Neuromuscular Disease, Motor Neuron Disease, Inherited Neuromuscular Conditions, Inherited Neuropathies
Interventions
EIM testing, Nerve & muscle ultrasonography, Nerve conduction studies, Electromyography (EMG)
Device
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
2 Years to 110 Years
Enrollment
275 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2013 – 2027
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 17, 2026, 2:27 PM EDT
Terminated Phase 2 Interventional Results available

Javelin BRCA/ATM: Avelumab Plus Talazoparib in Patients With BRCA or ATM Mutant Solid Tumors

NCT03565991
View directory record Official record
Conditions
Locally Advanced or Metastatic Solid Tumors, Genes, BRCA 1
Interventions
Avelumab, Talazoparib
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
202 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2023
U.S. locations
72
States / cities
Palo Alto, California • Stanford, California • Alpharetta, Georgia + 35 more
Source: ClinicalTrials.gov public record
Updated Sep 25, 2023 · Synced Oct 17, 2026, 2:27 PM EDT
Terminated Phase 2 Interventional

Phase 2 Study to Determine if RTB101 Reduces the Severity of COVID-19 in Older Adults Residing in Nursing Homes

NCT04409327
View directory record Official record
Conditions
COVID19
Interventions
RTB101, Placebo
Drug
Lead sponsor
Restorbio Inc.
Industry
Eligibility
65 Years and older
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2021
U.S. locations
1
States / cities
Middletown, Rhode Island
Source: ClinicalTrials.gov public record
Updated Feb 10, 2021 · Synced Oct 17, 2026, 2:27 PM EDT
Completed No phase listed Observational

Disability Severity Scale (DSI) and Hereditary Motor and Sensory Neuropathy Overall Disability Scale (HMSN-R-ODS)

NCT02194010
View directory record Official record
Conditions
Charcot Marie Tooth Disease, Inherited Peripheral Neuropathy
Interventions
Not listed
Lead sponsor
University of Iowa
Other
Eligibility
10 Years and older
Enrollment
563 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2019
U.S. locations
2
States / cities
Iowa City, Iowa • Rochester, New York
Source: ClinicalTrials.gov public record
Updated Feb 7, 2019 · Synced Oct 17, 2026, 2:27 PM EDT
Recruiting No phase listed Observational

Determining the Association of TTR Stabilizing Therapy With Circulating TTR Amyloid Aggregates Over Time in Patients With ATTR-CM: Longitudinal Biomarker Study

NCT07196839
View directory record Official record
Conditions
Transthyretin (TTR) Amyloid Cardiomyopathy
Interventions
Not listed
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
30 Years to 80 Years
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jan 7, 2026 · Synced Oct 17, 2026, 2:27 PM EDT
Recruiting Phase 1Phase 2 Interventional

Phase 1/2 Study of ETX-636 in Participants With Advanced Solid Tumors

NCT06993844
View directory record Official record
Conditions
Advanced Solid Tumors, Advanced Breast Cancer
Interventions
ETX-636 dose escalation, ETX-636 dose escalation in combination with fulvestrant, ETX-636 dose expansion in combination with fulvestrant
Drug
Lead sponsor
Ensem Therapeutics
Industry
Eligibility
18 Years and older
Enrollment
233 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
10
States / cities
Newport Beach, California • San Francisco, California • New Haven, Connecticut + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 17, 2026, 2:27 PM EDT
Completed No phase listed Observational Accepts healthy volunteers

Early Detection of Neuropathy in ATTRv

NCT05311488
View directory record Official record
Conditions
Hereditary Amyloidosis, Transthyretin-Related
Interventions
neurofilament light chain, In-vivo Meissner Corpuscle imaging, Nerve conduction study
Diagnostic Test
Lead sponsor
University of Pennsylvania
Other
Eligibility
30 Years to 90 Years
Enrollment
47 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2022 – 2026
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Mar 12, 2026 · Synced Oct 17, 2026, 2:27 PM EDT
Recruiting Phase 2 Interventional

Carboplatin or Olaparib for BRcA Deficient Prostate Cancer

NCT04038502
View directory record Official record
Conditions
Metastatic Castrate Resistant Prostate Cancer, BARD1, BRCA1, BRCA2, BRIP1, CHEK1, FANCL, PALB2, RAD51B, RAD51C, RAD51D, or RAD54L Mutations
Interventions
Carboplatin, Olaparib
Drug
Lead sponsor
VA Office of Research and Development
Federal
Eligibility
18 Years and older · Male only
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2027
U.S. locations
18
States / cities
West Los Angeles, California • Aurora, Colorado • Washington D.C., District of Columbia + 15 more
Source: ClinicalTrials.gov public record
Updated Sep 17, 2026 · Synced Oct 17, 2026, 2:27 PM EDT
Recruiting No phase listed Observational

Artificial Intelligence Guided Echocardiographic Screening of Rare Diseases (EchoNet-Screening)

NCT05139797
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
EchoNet-LVH screening for cardiac amyloidosis
Other
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
18 Years and older
Enrollment
300 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2027
U.S. locations
1
States / cities
Los Angeles, California
Source: ClinicalTrials.gov public record
Updated Jul 22, 2026 · Synced Oct 17, 2026, 2:27 PM EDT
Recruiting No phase listed Observational

ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)

NCT04561518
View directory record Official record
Conditions
Transthyretin-Mediated Amyloidosis, ATTR Amyloidosis
Interventions
Not listed
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
Not listed
Enrollment
1,500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2020 – 2033
U.S. locations
13
States / cities
La Jolla, California • Los Angeles, California • Jacksonville, Florida + 10 more
Source: ClinicalTrials.gov public record
Updated Oct 13, 2026 · Synced Oct 17, 2026, 2:27 PM EDT
No Longer Available No phase listed Expanded access

Compassionate Use of Triheptanoin (C7) for Inherited Disorders of Energy Metabolism

NCT01461304
View directory record Official record
Conditions
Very Long-chain acylCoA Dehydrogenase (VLCAD) Deficiency, Carnitine Palmitoyltransferase Deficiencies (CPT1, CPT2), Mitochondrial Trifunctional Protein Deficiency, Long-chain Hydroxyacyl-CoA Dehydrogenase Deficiency, Glycogen Storage Disorders, Pyruvate Carboxylase Deficiency Disease, ACYL-CoA DEHYDROGENASE FAMILY, MEMBER 9, DEFICIENCY of, Barth Syndrome
Interventions
triheptanoin
Drug
Lead sponsor
Jerry Vockley, MD, PhD
Other
Eligibility
1 Month and older
Healthy volunteers
Healthy volunteers not accepted
U.S. locations
1
States / cities
Pittsburgh, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Dec 10, 2021 · Synced Oct 17, 2026, 2:27 PM EDT
Completed Phase 2 Interventional Results available

A Study of INCB050465 in Participants With Autoimmune Hemolytic Anemia

NCT03538041
View directory record Official record
Conditions
Autoimmune Hemolytic Anemia
Interventions
Parsaclisib
Drug
Lead sponsor
Incyte Corporation
Industry
Eligibility
18 Years and older
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2024
U.S. locations
6
States / cities
Washington D.C., District of Columbia • Minneapolis, Minnesota • St Louis, Missouri + 3 more
Source: ClinicalTrials.gov public record
Updated Jul 11, 2025 · Synced Oct 17, 2026, 2:27 PM EDT
Completed Phase 1 Interventional Results available

Safety Study of an Adeno-associated Virus Vector for Gene Therapy of Leber's Hereditary Optic Neuropathy

NCT02161380
View directory record Official record
Conditions
Leber's Hereditary Optic Neuropathy
Interventions
injection of scAAV2-P1ND4v2 1.18x10e9 vg (Low),, injection of scAAV2-P1ND4v2 5.81 X10e9 vg (Med), injection of scAAV2-P1ND4v2 2.4 X10e10vg (High), injection of scAAV2-P1ND4v2 1.0 X10e11vg (Higher)
Drug
Lead sponsor
Byron Lam
Other
Eligibility
15 Years and older
Enrollment
28 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2025
U.S. locations
1
States / cities
Miami, Florida
Source: ClinicalTrials.gov public record
Updated Jun 5, 2025 · Synced Oct 17, 2026, 2:27 PM EDT
Completed Phase 1 Interventional Results available

Dose Escalation Study of TAK-117 (MLN1117) in Subjects With Advanced Cancer

NCT01449370
View directory record Official record
Conditions
Metastatic Solid Tumors
Interventions
TAK-117
Drug
Lead sponsor
Millennium Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
125 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2011 – 2016
U.S. locations
3
States / cities
Boston, Massachusetts • Detroit, Michigan • Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Mar 9, 2017 · Synced Oct 17, 2026, 2:27 PM EDT
Completed No phase listed Observational

Identification of Tongue Involvement in Late-Onset Pompe Disease

NCT02765828
View directory record Official record
Conditions
Myopathy, Neuropathy, Glycogen Storage Disease Type II (Late-onset Pompe Disease)
Interventions
Observational study
Other
Lead sponsor
Duke University
Other
Eligibility
12 Years and older
Enrollment
73 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2019
U.S. locations
1
States / cities
Durham, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 6, 2021 · Synced Oct 17, 2026, 2:27 PM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Gene Therapy Clinical Trial for the Treatment of Leber's Hereditary Optic Neuropathy Associated With ND4 Mutations

NCT05293626
View directory record Official record
Conditions
Leber Hereditary Optic Neuropathy (LHON)
Interventions
NR082 Injection, Injection needle
Drug · Device
Lead sponsor
Neurophth Therapeutics Inc
Other
Eligibility
18 Years to 75 Years
Enrollment
12 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2029
U.S. locations
3
States / cities
Palo Alto, California • Aurora, Colorado • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Sep 4, 2024 · Synced Oct 17, 2026, 2:27 PM EDT
Recruiting No phase listed Observational

Hereditary Spastic Paraplegia Genomic Sequencing Initiative (HSPseq)

NCT05354622
View directory record Official record
Conditions
Hereditary Spastic Paraplegia, Neurodegenerative Diseases, Pediatric Disorder, Spasticity, Muscle, Motor Neuron Disease, Movement Disorders
Interventions
Not listed
Lead sponsor
Boston Children's Hospital
Other
Eligibility
1 Month to 30 Years
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 18, 2026 · Synced Oct 17, 2026, 2:27 PM EDT
Completed Phase 2 Interventional

Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease

NCT06482437
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease
Interventions
NMD670, Placebo
Drug
Lead sponsor
NMD Pharma A/S
Industry
Eligibility
18 Years to 70 Years
Enrollment
81 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
8
States / cities
Kansas City, Kansas • Boston, Massachusetts • Columbia, Missouri + 5 more
Source: ClinicalTrials.gov public record
Updated Nov 12, 2025 · Synced Oct 17, 2026, 2:27 PM EDT