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ClinicalTrials.gov public records Last synced Oct 17, 2026, 8:24 AM EDT

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Showing 25–48 of 270 matching trials from the live ClinicalTrials.gov search.
Filters: Condition: Inherited Neuropathies Clear all
Recruiting No phase listed Observational

Non-interventional Study of Patients With Transthyretin (ATTR) Amyloidosis

NCT06465810
View directory record Official record
Conditions
Transthyretin Amyloidosis, ATTR-CM, ATTRv-PN, ATTR, ATTR-Mixed, hATTR
Interventions
Treatment of transthyretin (ATTR) amyloidosis in observational study setting
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 130 Years
Enrollment
1,850 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2031
U.S. locations
33
States / cities
La Jolla, California • Los Angeles, California • San Francisco, California + 24 more
Source: ClinicalTrials.gov public record
Updated Sep 23, 2026 · Synced Oct 17, 2026, 8:24 AM EDT
Completed Phase 2 Interventional Results available

A Study of Tanezumab in Adults With Chronic Low Back Pain

NCT00876187
View directory record Official record
Conditions
Low Back Pain
Interventions
Tanezumab 20 mg IV, Placebo for naproxen, Tanezumab 10 mg IV, Tanezumab 5 mg IV, Placebo for tanezumab, Naproxen
Biological · Drug
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
1,359 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2009 – 2011
U.S. locations
135
States / cities
Anniston, Alabama • Birmingham, Alabama • Hueytown, Alabama + 101 more
Source: ClinicalTrials.gov public record
Updated Jul 7, 2021 · Synced Oct 17, 2026, 8:24 AM EDT
Terminated Phase 1Phase 2 Interventional Results available

Study of Miransertib (MK-7075) in Participants With PIK3CA-related Overgrowth Spectrum and Proteus Syndrome (MOSAIC) (MK-7075-002)

NCT03094832
View directory record Official record
Conditions
PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome
Interventions
Miransertib
Drug
Lead sponsor
ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)
Industry
Eligibility
2 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2017 – 2022
U.S. locations
6
States / cities
Atlanta, Georgia • Chicago, Illinois • Boston, Massachusetts + 3 more
Source: ClinicalTrials.gov public record
Updated Apr 13, 2023 · Synced Oct 17, 2026, 8:24 AM EDT
Completed Not applicable Interventional

Artificial Intelligence Enhanced ECG to Detect Cardiac Amyloidosis

NCT05557162
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
AI ECG Amyloid algorithm
Other
Lead sponsor
Mayo Clinic
Other
Eligibility
18 Years and older
Enrollment
20,143 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2023 – 2025
U.S. locations
4
States / cities
Scottsdale, Arizona • Jacksonville, Florida • Rochester, Minnesota + 1 more
Source: ClinicalTrials.gov public record
Updated Feb 17, 2026 · Synced Oct 17, 2026, 8:24 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Treatment of Charcot-Marie-Tooth Disease, Axonal, Type 2S (CMT2S) in an Individual Patient

NCT07223632
View directory record Official record
Conditions
Charcot Marie Tooth Disease (CMT), Neuromuscular Diseases (NMD)
Interventions
VCA-894A
Drug
Lead sponsor
Vanda Pharmaceuticals
Industry
Eligibility
Female only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Madison, Wisconsin
Source: ClinicalTrials.gov public record
Updated Aug 19, 2026 · Synced Oct 17, 2026, 8:24 AM EDT
Withdrawn Phase 1Phase 2 Interventional

Safety and Efficacy Study of Neratinib and Cetuximab to Treat Patients With Quadruple Wild-Type Metastatic Colorectal Cancer

NCT01960023
View directory record Official record
Conditions
Colorectal Cancer
Interventions
Cetuximab, Neratinib
Drug
Lead sponsor
NSABP Foundation Inc
Network
Eligibility
18 Years and older
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2016
U.S. locations
12
States / cities
Gainesville, Florida • Orlando, Florida • Boise, Idaho + 7 more
Source: ClinicalTrials.gov public record
Updated Oct 27, 2021 · Synced Oct 17, 2026, 8:24 AM EDT
Completed Phase 1Phase 2 Interventional

Combination Trial of Tipifarnib and Alpelisib in Adult Recurrent/ Metastatic Head and Neck Squamous Cell Carcinoma (R/M HNSCC)

NCT04997902
View directory record Official record
Conditions
HNSCC
Interventions
Tipifarnib, Alpelisib
Drug
Lead sponsor
Kura Oncology, Inc.
Industry
Eligibility
18 Years and older
Enrollment
45 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
11
States / cities
Duarte, California • Orlando, Florida • Baltimore, Maryland + 7 more
Source: ClinicalTrials.gov public record
Updated Sep 8, 2026 · Synced Oct 17, 2026, 8:24 AM EDT
Completed Phase 1 Interventional

Safety Study of a Single IVT Injection of QPI-1007 in Chronic Optic Nerve Atrophy and Recent Onset NAION Patients

NCT01064505
View directory record Official record
Conditions
Optic Atrophy, Non-arteritic Anterior Ischemic Optic Neuropathy
Interventions
QPI-1007 at various doses
Drug
Lead sponsor
Quark Pharmaceuticals
Industry
Eligibility
50 Years and older
Enrollment
48 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2013
U.S. locations
22
States / cities
Phoenix, Arizona • Beverly Hills, California • Los Angeles, California + 19 more
Source: ClinicalTrials.gov public record
Updated May 13, 2013 · Synced Oct 17, 2026, 8:24 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

A Study of NDI 1150-101 in Patients With Solid Tumors

NCT05128487
View directory record Official record
Conditions
Solid Tumor, Renal Cell Carcinoma (Kidney Cancer), Gastric and Gastroesophageal Junction (GEJ) Adenocarcinoma, Non-Small Cell Lung Cancer
Interventions
NDI-101150, Pembrolizumab
Drug
Lead sponsor
Nimbus Saturn, Inc.
Industry
Eligibility
18 Years and older
Enrollment
106 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2025
U.S. locations
19
States / cities
Scottsdale, Arizona • New Haven, Connecticut • Washington D.C., District of Columbia + 15 more
Source: ClinicalTrials.gov public record
Updated Nov 25, 2024 · Synced Oct 17, 2026, 8:24 AM EDT
Completed Phase 1 Interventional

Safety, Tolerability & Potential Anti-cancer Activity of Increasing Doses of AZD5363 in Different Treatment Schedules

NCT01226316
View directory record Official record
Conditions
Advanced Solid Malignancy, Safety and Tolerability, Pharmacokinetics, Pharmacodynamics, Tumour Response, Advanced or Metastatic Breast Cancer, Ovarian Cancer, Cervical Cancer, Endometrial Cancer, PIK3CA, AKT1, PTEN, ER Positive, HER2 Positive
Interventions
AZD5363
Drug
Lead sponsor
AstraZeneca
Industry
Eligibility
18 Years to 130 Years
Enrollment
285 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2010 – 2024
U.S. locations
12
States / cities
Los Angeles, California • Stanford, California • West Hollywood, California + 8 more
Source: ClinicalTrials.gov public record
Updated Jun 29, 2025 · Synced Oct 17, 2026, 8:24 AM EDT
Terminated Phase 1Phase 2 Interventional Accepts healthy volunteers Results available

Near-infrared Light-emitting Diode (NIR-LED) Therapy for Leber's Hereditary Optic Neuropathy (LHON)

NCT01389817
View directory record Official record
Conditions
Leber's Hereditary Optic Neuropathy (LHON)
Interventions
Near-infrared light-emitting diode (NIR-LED) therapy (Med Light 630 PRO (Medical Devices Inc.))
Device
Lead sponsor
Medical College of Wisconsin
Other
Eligibility
18 Years and older
Enrollment
4 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2011 – 2013
U.S. locations
3
States / cities
Milwaukee, Wisconsin
Source: ClinicalTrials.gov public record
Updated Sep 29, 2014 · Synced Oct 17, 2026, 8:24 AM EDT
Completed Phase 2 Interventional

An Open-Label Clinical Trial of Replagal Enzyme Therapy in Children Ages 7-17 Years With Fabry Disease

NCT00071877
View directory record Official record
Conditions
Fabry Disease
Interventions
Replagal
Drug
Lead sponsor
National Institute of Neurological Disorders and Stroke (NINDS)
NIH
Eligibility
Not listed
Enrollment
25 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2003 – 2005
U.S. locations
1
States / cities
Bethesda, Maryland
Source: ClinicalTrials.gov public record
Updated Mar 4, 2008 · Synced Oct 17, 2026, 8:24 AM EDT
Terminated Not applicable Interventional

Does Wearing Tetra-Grip Improve Arm Function in Children Diagnosed With Neonatal Brachial Plexus Palsy?

NCT03647761
View directory record Official record
Conditions
Neonatal Brachial Plexus Palsy
Interventions
Tetra-grip
Device
Lead sponsor
University of Texas Southwestern Medical Center
Other
Eligibility
3 Years to 7 Years
Enrollment
2 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2019 – 2022
U.S. locations
1
States / cities
Dallas, Texas
Source: ClinicalTrials.gov public record
Updated Jul 5, 2022 · Synced Oct 17, 2026, 8:24 AM EDT
Active, not recruiting Phase 2 Interventional

A Study of the Safety and Tolerability in Participants With PIK3CA-related Overgrowth Spectrum or Proteus Syndrome Who Are Being Treated With Miransertib (MK-7075) in Other Studies (MK-7075-006)

NCT04980872
View directory record Official record
Conditions
PIK3CA-Related Overgrowth Spectrum (PROS)/Proteus Syndrome (PS)
Interventions
Miransertib
Drug
Lead sponsor
Merck Sharp & Dohme LLC
Industry
Eligibility
2 Years to 120 Years
Enrollment
60 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2030
U.S. locations
5
States / cities
Atlanta, Georgia • Boston, Massachusetts • Cincinnati, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Feb 7, 2025 · Synced Oct 17, 2026, 8:24 AM EDT
Completed Phase 3 Interventional Results available

ENDEAVOUR: Phase 3 Multicenter Study of Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)

NCT02319005
View directory record Official record
Conditions
Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC), Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related, Familial Transthyretin Cardiac Amyloidosis
Interventions
Revusiran (ALN-TTRSC), Sterile Normal Saline (0.9% NaCl)
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years to 90 Years
Enrollment
206 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2017
U.S. locations
41
States / cities
Bakersfield, California • Beverly Hills, California • La Mesa, California + 30 more
Source: ClinicalTrials.gov public record
Updated Jul 18, 2018 · Synced Oct 17, 2026, 8:24 AM EDT
Completed No phase listed Observational Accepts healthy volunteers

Observational Study of Neuromuscular Function in CMT Type 1&2 and Healthy Controls

NCT04980807
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease
Interventions
Not listed
Lead sponsor
NMD Pharma A/S
Industry
Eligibility
18 Years and older
Enrollment
30 participants
Healthy volunteers
Accepts healthy volunteers
Timeline
2021 – 2022
U.S. locations
1
States / cities
Columbus, Ohio
Source: ClinicalTrials.gov public record
Updated Mar 7, 2022 · Synced Oct 17, 2026, 8:24 AM EDT
Recruiting Not applicable Interventional

CAPACITY (Cardiac Amyloidosis and Physical ACtivITY) Study

NCT06096675
View directory record Official record
Conditions
Cardiac Amyloidosis
Interventions
• Cardiac Rehabilitation
Behavioral
Lead sponsor
Wake Forest University Health Sciences
Other
Eligibility
18 Years and older
Enrollment
50 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Charlotte, North Carolina
Source: ClinicalTrials.gov public record
Updated Jul 21, 2026 · Synced Oct 17, 2026, 8:24 AM EDT
Completed Phase 2Phase 3 Interventional Results available

The Effect of Diflunisal on Familial Amyloidosis

NCT00294671
View directory record Official record
Conditions
Familial Amyloid Polyneuropathy, Familial Amyloidosis
Interventions
diflunisal, placebo
Drug · Other
Lead sponsor
Boston University
Other
Eligibility
18 Years to 75 Years
Enrollment
130 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2006 – 2012
U.S. locations
3
States / cities
Boston, Massachusetts • Rochester, Minnesota • New York, New York
Source: ClinicalTrials.gov public record
Updated Mar 17, 2017 · Synced Oct 17, 2026, 8:24 AM EDT
Completed Phase 3 Interventional Results available

Extension Study Assessing Long Term Safety and Efficacy of IONIS-TTR Rx in Familial Amyloid Polyneuropathy (FAP)

NCT02175004
View directory record Official record
Conditions
FAP, Familial Amyloid Polyneuropathy, TTR, Transthyretin, Amyloidosis
Interventions
Inotersen
Drug
Lead sponsor
Ionis Pharmaceuticals, Inc.
Industry
Eligibility
18 Years and older
Enrollment
135 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2021
U.S. locations
9
States / cities
Orange, California • Indianapolis, Indiana • Baltimore, Maryland + 5 more
Source: ClinicalTrials.gov public record
Updated Nov 18, 2023 · Synced Oct 17, 2026, 8:24 AM EDT
Not listed No phase listed Observational

Exercise Effect on Transthyretin Stability

NCT04986826
View directory record Official record
Conditions
Amyloidosis Transthyretin, Athlete Heart
Interventions
Not listed
Lead sponsor
Oregon Health and Science University
Other
Eligibility
18 Years and older
Enrollment
24 participants
Timeline
2021
U.S. locations
1
States / cities
Portland, Oregon
Source: ClinicalTrials.gov public record
Updated Aug 3, 2021 · Synced Oct 17, 2026, 8:24 AM EDT
Completed Phase 4 Interventional Results available

Study to Assess the Efficacy and Safety of Raxone in LHON Patients

NCT02774005
View directory record Official record
Conditions
Leber's Hereditary Optic Neuropathy (LHON)
Interventions
Idebenone
Drug
Lead sponsor
Santhera Pharmaceuticals
Industry
Eligibility
12 Years and older
Enrollment
199 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2016 – 2021
U.S. locations
9
States / cities
Phoenix, Arizona • Palo Alto, California • Stanford, California + 6 more
Source: ClinicalTrials.gov public record
Updated Apr 21, 2023 · Synced Oct 17, 2026, 8:24 AM EDT
Completed Phase 2 Interventional Results available

Open-Label, Dose-Escalating Study Assessing Safety, Tolerability, Efficacy, of RP103 in Mitochondrial Disease

NCT02023866
View directory record Official record
Conditions
Inherited Mitochondrial Disease, Including Leigh Syndrome
Interventions
Cysteamine Bitartrate
Drug
Lead sponsor
Amgen
Industry
Eligibility
6 Years to 17 Years
Enrollment
36 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2014 – 2016
U.S. locations
5
States / cities
San Diego, California • Stanford, California • Akron, Ohio + 2 more
Source: ClinicalTrials.gov public record
Updated Dec 27, 2024 · Synced Oct 17, 2026, 8:24 AM EDT
Terminated Phase 1 Interventional

PF-07284892 in Participants With Advanced Solid Tumors

NCT04800822
View directory record Official record
Conditions
Solid Tumor
Interventions
PF-07284892, lorlatinib, binimetinib, cetuximab, encorafenib
Drug · Biological
Lead sponsor
Pfizer
Industry
Eligibility
18 Years and older
Enrollment
53 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2021 – 2024
U.S. locations
11
States / cities
Phoenix, Arizona • Scottsdale, Arizona • Detroit, Michigan + 6 more
Source: ClinicalTrials.gov public record
Updated Jun 4, 2026 · Synced Oct 17, 2026, 8:24 AM EDT
Completed Phase 3 Interventional Results available

APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis

NCT01960348
View directory record Official record
Conditions
TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies, Amyloid Neuropathies, Amyloidosis, Hereditary, Transthyretin-Related
Interventions
patisiran (ALN-TTR02), Sterile Normal Saline (0.9% NaCl)
Drug
Lead sponsor
Alnylam Pharmaceuticals
Industry
Eligibility
18 Years to 85 Years
Enrollment
225 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2013 – 2017
U.S. locations
13
States / cities
La Mesa, California • Orange, California • Denver, Colorado + 9 more
Source: ClinicalTrials.gov public record
Updated Apr 22, 2024 · Synced Oct 17, 2026, 8:24 AM EDT