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ClinicalTrials.gov public records Last synced Oct 17, 2026, 6:56 AM EDT

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Showing 1–24 of 270 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inherited Neuropathies Clear all
Recruiting No phase listed Observational

Studying the Presence of CFRD Complications With Thoughtful Recruitment (SPeCTRuM)

NCT06837181
View directory record Official record
Conditions
Cystic Fibrosis (CF), Cystic Fibrosis-related Diabetes, Diabetes, Retinopathy, Neuropathy, Nephropathy, Blood Pressure, Cardiovascular Risk, Microvascular
Interventions
observational study
Other
Lead sponsor
Jaeb Center for Health Research
Other
Eligibility
12 Years and older
Enrollment
200 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2028
U.S. locations
18
States / cities
Birmingham, Alabama • Los Angeles, California • Palo Alto, California + 15 more
Source: ClinicalTrials.gov public record
Updated Feb 10, 2026 · Synced Oct 17, 2026, 6:56 AM EDT
Completed Phase 1Phase 2 Interventional

Effects of Coenzyme Q10 on Charcot-Marie-Tooth Disease

NCT00541164
View directory record Official record
Conditions
Charcot Marie Tooth Disease
Interventions
Coenzyme Q10, Coenzyme Q10
Drug · Dietary Supplement
Lead sponsor
Memorial Medical Center
Other
Eligibility
18 Years to 75 Years
Enrollment
23 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2013
U.S. locations
1
States / cities
Johnstown, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 15, 2013 · Synced Oct 17, 2026, 6:56 AM EDT
Completed Phase 2 Interventional Results available

Mexiletine for Muscle Cramps in Charcot Marie Tooth Disease

NCT02561702
View directory record Official record
Conditions
Charcot Marie Tooth Disease
Interventions
Placebo, Mexiletine
Other · Drug
Lead sponsor
University of Rochester
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2015 – 2016
U.S. locations
1
States / cities
Rochester, New York
Source: ClinicalTrials.gov public record
Updated Mar 26, 2018 · Synced Oct 17, 2026, 6:56 AM EDT
Completed Phase 2Phase 3 Interventional Results available

Safety and Efficacy Study of Fx-1006A in Patients With Familial Amyloidosis

NCT00409175
View directory record Official record
Conditions
Familial Amyloid Polyneuropathy
Interventions
Fx-1006A, Placebo
Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 75 Years
Enrollment
128 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2007 – 2009
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Dec 17, 2012 · Synced Oct 17, 2026, 6:56 AM EDT
Terminated Phase 3 Interventional Results available

Long-Term Analgesic Efficacy And Safety Of Tanezumab Alone Or In Combination With Non-Steroidal Anti-Inflammatory Drugs (NSAIDs) Versus NSAIDs Alone In Patients With Osteoarthritis Of The Knee Or Hip

NCT00809354
View directory record Official record
Conditions
Osteoarthritis, Arthritis
Interventions
NSAID, tanezumab
Drug · Biological
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 99 Years
Enrollment
2,720 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
250
States / cities
Birmingham, Alabama • Huntsville, Alabama • Mobile, Alabama + 161 more
Source: ClinicalTrials.gov public record
Updated Jun 24, 2021 · Synced Oct 17, 2026, 6:56 AM EDT
Active, not recruiting Not applicable Interventional Accepts healthy volunteers

NuroSleeve Powered Brace & Stimulation System to Restore Arm Function

NCT04798378
View directory record Official record
Conditions
Neurologic Diseases, Hemiparesis, Quadriplegia, Muscular Dystrophies, Arthrogryposis, Spinal Cord Injuries, Charcot-Marie-Tooth, Stroke, Weakness of Extremities as Sequela of Stroke, Weakness Due to Upper Motor Neuron Dysfunction, Amyotrophic Lateral Sclerosis, Spinal Muscular Atrophy, Arm Paralysis, Cerebral Palsy
Interventions
Neurosleeve
Device
Lead sponsor
Thomas Jefferson University
Other
Eligibility
4 Years and older
Enrollment
20 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2020 – 2026
U.S. locations
2
States / cities
Wilmington, Delaware • Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Apr 9, 2025 · Synced Oct 17, 2026, 6:56 AM EDT
Completed Phase 2 Interventional Results available

Study Assessing the Efficacy and Safety of Alpelisib Plus Fulvestrant or Letrozole, Based on Prior Endocrine Therapy, in Patients With PIK3CA Mutant, HR+, HER2- Advanced Breast Cancer Who Have Progressed on or After Prior Treatments

NCT03056755
View directory record Official record
Conditions
Breast Cancer
Interventions
Alpelisib, Fulvestrant, Letrozole, Goserelin, Leuprolide
Drug
Lead sponsor
Novartis Pharmaceuticals
Industry
Eligibility
18 Years to 99 Years
Enrollment
383 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
25
States / cities
Gilbert, Arizona • Phoenix, Arizona • Anaheim, California + 20 more
Source: ClinicalTrials.gov public record
Updated Jan 13, 2026 · Synced Oct 17, 2026, 6:56 AM EDT
Recruiting Phase 4 Interventional

Cardiac Magnetic Resonance and Echocardiography Study With Acoramidis

NCT07695701
View directory record Official record
Conditions
Transthyretin Amyloidosis Cardiomyopathy (ATTR-CM), Amyloidosis, Amyloid Cardiomyopathy, Transthyretin Amyloidosis
Interventions
Acoramidis (AG10) oral tablet
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years to 80 Years
Enrollment
150 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2030
U.S. locations
1
States / cities
St Louis, Missouri
Source: ClinicalTrials.gov public record
Updated Jul 29, 2026 · Synced Oct 17, 2026, 6:56 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Types CMT1B, CMT2A, CMT4A, CMT4C, and Others

NCT01193075
View directory record Official record
Conditions
Charcot Marie Tooth Disease
Interventions
Not listed
Lead sponsor
Michael Shy
Other
Eligibility
Not listed
Enrollment
5,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2010 – 2030
U.S. locations
18
States / cities
Los Angeles, California • Palo Alto, California • Aurora, Colorado + 14 more
Source: ClinicalTrials.gov public record
Updated Oct 16, 2026 · Synced Oct 17, 2026, 6:56 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

A Study to Evaluate Administration of SBT101 Gene Therapy in Adult Patients With Adrenomyeloneuropathy (AMN)

NCT05394064
View directory record Official record
Conditions
AMN, AMN Gene Mutation, X-ALD
Interventions
SBT101 Treatment
Genetic
Lead sponsor
SwanBio Therapeutics, Inc.
Industry
Eligibility
18 Years to 65 Years · Male only
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2022 – 2025
U.S. locations
1
States / cities
Worcester, Massachusetts
Source: ClinicalTrials.gov public record
Updated Aug 26, 2026 · Synced Oct 17, 2026, 6:56 AM EDT
Completed Not applicable Interventional Results available

NMES Efficacy on Patients With NBPP

NCT01999465
View directory record Official record
Conditions
Neonatal Brachial Plexus Palsy
Interventions
Standard NMES device, Sham NMES device
Device
Lead sponsor
University of Michigan
Other
Eligibility
3 Months to 9 Months
Enrollment
20 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2018
U.S. locations
1
States / cities
Ann Arbor, Michigan
Source: ClinicalTrials.gov public record
Updated Dec 14, 2018 · Synced Oct 17, 2026, 6:56 AM EDT
Terminated Phase 2 Interventional Results available

Open-Label Study of AG10 in Patients With Cardiomyopathy

NCT03536767
View directory record Official record
Conditions
Amyloid Cardiomyopathy
Interventions
Acoramidis
Drug
Lead sponsor
Eidos Therapeutics, a BridgeBio company
Industry
Eligibility
18 Years and older
Enrollment
47 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2025
U.S. locations
9
States / cities
San Francisco, California • Stanford, California • New Haven, Connecticut + 6 more
Source: ClinicalTrials.gov public record
Updated Oct 14, 2026 · Synced Oct 17, 2026, 6:56 AM EDT
Completed No phase listed Observational Results available

Screening for Cardiac Amyloidosis With Nuclear Imaging for Minority Populations

NCT03812172
View directory record Official record
Conditions
Amyloid Cardiomyopathy, Transthyretin-Related
Interventions
99mTc-PYP or 99m Tc-HDP
Drug
Lead sponsor
Mathew S. Maurer, MD
Other
Eligibility
60 Years and older
Enrollment
646 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2024
U.S. locations
4
States / cities
New Haven, Connecticut • Boston, Massachusetts • New York, New York
Source: ClinicalTrials.gov public record
Updated Nov 19, 2025 · Synced Oct 17, 2026, 6:56 AM EDT
Completed No phase listed Observational

A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

NCT04201418
View directory record Official record
Conditions
Hereditary Transthyretin-mediated (ATTRv) Amyloidosis, Polyneuropathy
Interventions
Patisiran
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years and older
Enrollment
67 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
27
States / cities
Los Angeles, California • San Diego, California • Aurora, Colorado + 22 more
Source: ClinicalTrials.gov public record
Updated Jun 6, 2022 · Synced Oct 17, 2026, 6:56 AM EDT
Completed Phase 4 Interventional

D-Cycloserine for Serine Palmitoyltransferase Inhibition

NCT07542548
View directory record Official record
Conditions
Hereditary Spastic Paraplegia
Interventions
D-cycloserine
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
6 Years to 6 Years · Female only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 21, 2026 · Synced Oct 17, 2026, 6:56 AM EDT
Completed Phase 3 Interventional Results available

Tanezumab in Osteoarthritis of the Knee

NCT00733902
View directory record Official record
Conditions
Arthritis, Osteoarthritis
Interventions
tanezumab, Placebo
Biological
Lead sponsor
Pfizer
Industry
Eligibility
18 Years to 99 Years
Enrollment
697 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2008 – 2010
U.S. locations
82
States / cities
Phoenix, Arizona • Danbury, Connecticut • Stamford, Connecticut + 65 more
Source: ClinicalTrials.gov public record
Updated Mar 22, 2021 · Synced Oct 17, 2026, 6:56 AM EDT
Not yet recruiting Phase 1Phase 2 Interventional

Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)

NCT06948019
View directory record Official record
Conditions
HSP, Hereditary Spastic Paraplegia, Hereditary Spastic Paraparesis, Hereditary Spastic Paraplegia Type 50, Hereditary Spastic Paraplegia Type 47, Hereditary Spastic Paraplegia Type 51, Hereditary Spastic Paraplegia Type 52, SPG47, AP4B1, Neurogenetic Disorders, Neurodevelopmental Conditions, Movement Disorders, Gene Therapy
Interventions
BFB-101 (AAV9-CBh-AP4B1)
Biological
Lead sponsor
BlackfinBio Ltd
Industry
Eligibility
12 Months to 60 Months
Enrollment
5 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2032
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Apr 28, 2025 · Synced Oct 17, 2026, 6:56 AM EDT
Not listed No phase listed Observational

Amyloidosis Incidence in High-Risk Cardiac Device Patients

NCT06186167
View directory record Official record
Conditions
Amyloid, Cardiac Amyloidosis, Amyloidosis Cardiac, Systemic Amyloidosis, AL Amyloidosis, Infiltrative Cardiomyopathy, Amyloid, ATTR Amyloidosis Wild Type
Interventions
Chest Wall Fat Tissue Collection
Procedure
Lead sponsor
Midwest Heart & Vascular Specialists
Other
Eligibility
40 Years and older
Enrollment
100 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024
U.S. locations
1
States / cities
Overland Park, Kansas
Source: ClinicalTrials.gov public record
Updated Feb 20, 2024 · Synced Oct 17, 2026, 6:56 AM EDT
Enrolling by invitation Not applicable Interventional

AI Echocardiographic Screening of Cardiac Amyloidosis

NCT06664866
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
EchoNet-LVH Assessment
Diagnostic Test
Lead sponsor
Cedars-Sinai Medical Center
Other
Eligibility
22 Years and older
Enrollment
500 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
4
States / cities
Los Angeles, California • Palo Alto, California • Chicago, Illinois + 1 more
Source: ClinicalTrials.gov public record
Updated Jul 22, 2026 · Synced Oct 17, 2026, 6:56 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Development and Validation of a Disability Severity Index for CMT

NCT01455623
View directory record Official record
Conditions
Charcot Marie Tooth Disease
Interventions
Not listed
Lead sponsor
University of Iowa
Other
Eligibility
8 Years and older
Enrollment
291 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2014
U.S. locations
1
States / cities
Iowa City, Iowa
Source: ClinicalTrials.gov public record
Updated Mar 26, 2014 · Synced Oct 17, 2026, 6:56 AM EDT
Not listed Phase 3 Interventional

Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A

NCT03023540
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease, Type IA
Interventions
PXT3003
Drug
Lead sponsor
Pharnext S.C.A.
Other
Eligibility
17 Years to 67 Years
Enrollment
187 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2024
U.S. locations
9
States / cities
Los Angeles, California • Gainesville, Florida • Kansas City, Kansas + 6 more
Source: ClinicalTrials.gov public record
Updated Feb 20, 2024 · Synced Oct 17, 2026, 6:56 AM EDT
Completed No phase listed Observational

REALITY LHON Registry

NCT03295071
View directory record Official record
Conditions
Leber Hereditary Optic Neuropathy
Interventions
Patient-reported outcomes (PROs)
Other
Lead sponsor
GenSight Biologics
Industry
Eligibility
Not listed
Enrollment
44 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2018 – 2020
U.S. locations
5
States / cities
Pasadena, California • Atlanta, Georgia • Boston, Massachusetts + 2 more
Source: ClinicalTrials.gov public record
Updated Mar 2, 2026 · Synced Oct 17, 2026, 6:56 AM EDT
Recruiting No phase listed Observational Accepts healthy volunteers

Inherited Retinal Degenerative Disease Registry

NCT02435940
View directory record Official record
Conditions
Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome, Bassen-Kornzweig Syndrome, Batten Disease, Best Disease, Choroidal Dystrophy, Choroideremia, Cone Dystrophy, Cone-Rod Dystrophy, Congenital Stationary Night Blindness, Enhanced S-Cone Syndrome, Fundus Albipunctatus, Goldmann-Favre Syndrome, Gyrate Atrophy, Juvenile Macular Degeneration, Kearns-Sayre Syndrome, Leber Congenital Amaurosis, Refsum Syndrome, Retinitis Pigmentosa, Retinitis Punctata Albescens, Retinoschisis, Rod-Cone Dystrophy, Rod Dystrophy, Rod Monochromacy, Stargardt Disease, Usher Syndrome
Interventions
Not listed
Lead sponsor
Foundation Fighting Blindness
Other
Eligibility
Not listed
Enrollment
20,000 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2014 – 2037
U.S. locations
1
States / cities
Columbia, Maryland
Source: ClinicalTrials.gov public record
Updated May 19, 2026 · Synced Oct 17, 2026, 6:56 AM EDT
Recruiting Phase 2 Interventional

A Study of ROC-101 in Patients With Pulmonary Arterial Hypertension (PAH) and Pulmonary Hypertension Associated With Interstitial Lung Disease (ILD-PH) (ROCSTAR STUDY)

NCT07175038
View directory record Official record
Conditions
Pulmonary Arterial Hypertension (PAH), Pulmonary Hypertension, Interstitial Lung Disease
Interventions
ROC-101
Drug
Lead sponsor
AllRock Bio, Inc.
Industry
Eligibility
18 Years to 80 Years
Enrollment
40 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2029
U.S. locations
11
States / cities
Phoenix, Arizona • Rancho Cordova, California • Aurora, Colorado + 8 more
Source: ClinicalTrials.gov public record
Updated Oct 8, 2026 · Synced Oct 17, 2026, 6:56 AM EDT