Clinical trials: Neuropathy, open to volunteers
Familial Dysautonomia (Riley-Day Syndrome), Hereditary Sensory and Autonomic Neuropathies
The Natural History of Familial Dysautonomia
The study will collect clinical information from patients with FD and allow them to give blood to help develop biological markers of the disease to aid diagnosis and treatment.
- Conditions
- Familial Dysautonomia (Riley-Day Syndrome), Hereditary Sensory and Autonomic Neuropathies, Hereditary Sensory and Autonomic Neuropathy 3
- Trial name
- NCT03920774
- Where
- 2 sites in United States, Israel
- Volunteers
- 400 people
- Sponsor
- NYU Langone Health
Otoferlin Gene-mediated Hearing Loss
Long Term Follow-up Study of AAVAnc80-hOTOF Gene Therapy
This multi-center, observational, post-intervention long term follow-up (LTFU) study will monitor for safety and efficacy of AAVAnc80-hOTOF in individuals with sensorineural hearing loss due to otoferlin gene mutations who have…
- Trial name
- NCT06696456
- Where
- 5 sites in United States, Spain and 1 more
- Volunteers
- 30 people
- Sponsor
- Eli Lilly and Company
Painful Diabetic Peripheral Neuropathy, Diabetes Complication
Safety and Effect of Peripheral Nerve Decompression Combined With Peripheral Nerve Stimulation for Painful Diabetic Peripheral Neuropathy
Diabetic peripheral neuropathy (DPN) is one of the most common chronic complications of diabetes mellitus and a major cause of lower extremity pain, numbness, sensory loss, balance disturbance, diabetic foot ulcer and amputation.
- Trial name
- NCT07911410
- Where
- 4 sites in China
- Volunteers
- 100 people
- Sponsor
- Qilu Hospital of Shandong University
Rare Disorders, Undiagnosed Disorders
Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford
The Coordination of Rare Diseases at Sanford (CoRDS) is an international patient registry that serves as a centralized resource for rare disease research.
- Conditions
- Rare Disorders, Undiagnosed Disorders, Disorders of Unknown Prevalence, Cornelia De Lange Syndrome
- Trial name
- CoRDS (NCT01793168)
- Where
- United States
- Volunteers
- 20,000 people
- Sponsor
- Sanford Health
Charcot-Marie-Tooth Disease, Type Ia (Disorder), HMSN
Genetics of Charcot Marie Tooth (CMT) - Modifiers of CMT1A, New Causes of CMT2
This project includes two projects. One is looking for new genes that cause Charcot Marie Tooth disease (CMT). The other is looking for genes that do not cause CMT, but may modify the symptoms a person has.
- Trial name
- INC-6602 (NCT01193088)
- Where
- 24 sites in United States, Australia and 4 more
- Volunteers
- 1,050 people, healthy volunteers welcome
- Sponsor
- University of Iowa
Charcot Marie Tooth Disease
Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Types CMT1B, CMT2A, CMT4A, CMT4C, and Others
This is an observational longitudinal study to determine the natural history and genotype-phenotype correlations of disease causing mutations in Charcot Marie Tooth disease (CMT) type 1B (CMT1B), 2A (CMT2A), 4A (CMT4A), and 4C…
- Trial name
- INC-6601 (NCT01193075)
- Where
- 24 sites in United States, Australia and 4 more
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- Michael Shy
Critical Illness, Neuropathy
Establishing Normative Data for Pupillometer Assessments in Neuro-Intensive Care
The purpose of this study is to collect a large amount of data that may assist in addressing the gap in knowledge of understanding pupillometer readings using a pupillometer device.
- Trial name
- ENDPANIC (NCT02804438)
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- University of Texas Southwestern Medical Center
Amyloidogenic Transthyretin (ATTR) Amyloidosis, Cardiac Amyloidosis
PET Imaging Study Using Evuzamitide to Detect Cardiac Amyloidosis in Patients With Inconclusive Nuclear Scans and Elevated TAD1 Levels
The goal of this study is to learn whether PET-CT imaging using evuzamitide can help diagnose transthyretin cardiac amyloidosis (ATTR-CA) in patients whose standard nuclear imaging results are unclear but who have elevated TAD1…
- Trial name
- TRACE (NCT07538518)
- Testing
- Evuzamitide
- Where
- 3 sites in United States
- Volunteers
- 25 people
- Sponsor
- Lorena Saelices
Peripheral Neuropathy, Trigeminal Neuralgia
Safety of Cultured Allogeneic Adult Umbilical Cordor Placental Derived Stem Cells Exosomes for Trigeminal Neuralgia
This trial will study the safety and efficacy of instillation of cultured allogeneic adult umbilical cord or placental derived mesenchymal stem cell exosomes for the treatment of Trigeminal Neuralgia.
- Trial name
- NCT05152368
- Testing
- cultured allogeneic adult umbilical cord or placental derived mesenchymal stem cell exosomes
- Where
- 2 sites in United States, Greece
- Volunteers
- 20 people
- Sponsor
- The Foundation for Orthopaedics and Regenerative Medicine
Vascular Anomalies, Vascular Anomaly
24VA022; VATCH Alpelisib for TIE2/PIK3CA Pathway VAs
The study will enroll participants 2 months of age up to 30 years of age. The purpose of this study is to assess the effectiveness and safety of Alpelisib (the "Study Drug") in patients with PIK3CA/TIE-2/TEK pathway driven…
- Conditions
- Vascular Anomalies, Vascular Anomaly, TIE2/PIK3CA Pathway Driven Vascular Anomalies
- Trial name
- VATCH (NCT07543822)
- Testing
- alpelisib (BYL719)
- Where
- United States
- Volunteers
- 61 people
- Sponsor
- Children's Hospital of Philadelphia
Neuromuscular Disease, Motor Neuron Disease
Electrical Impedance Myography: Natural History Studies inNeuromuscular Disorders and Healthy Volunteers
Background: Electrical impedance myography (EIM) is a new technique being studied to see if it is helpful in evaluating muscle disorders and nerve disorders.
- Conditions
- Neuromuscular Disease, Motor Neuron Disease, Inherited Neuromuscular Conditions, Inherited Neuropathies
- Trial name
- NCT01900132
- Testing
- EIM testing, Nerve & muscle ultrasonography, Nerve conduction studies
- Where
- United States
- Volunteers
- 275 people, healthy volunteers welcome
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Eye Diseases, Retinitis Pigmentosa
Safety of Cultured Allogeneic Adult Umbilical Cord or Placental Derived Mesenchymal Stem Cell Exosomes for NAION
This trial will study the safety and efficacy of intranasal delivery of cultured allogeneic adult umbilical cord or placental derived mesenchymal stem cell exosomes for the treatment of non-arteritic ischemic optic neuropathy
- Conditions
- Eye Diseases, Retinitis Pigmentosa, Diabetic Retinopathy, Macular Degeneration
- Trial name
- NCT05147701
- Testing
- cultured allogeneic adult umbilical cord or placental derived mesenchymal stem cell exosomes
- Where
- 2 sites in United States, Greece
- Volunteers
- 20 people
- Sponsor
- The Foundation for Orthopaedics and Regenerative Medicine
Transthyretin-Mediated Amyloidosis, ATTR Amyloidosis
ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)
The purpose of this study is to: Describe epidemiological and clinical characteristics, natural history and real-world clinical management of ATTR amyloidosis patients Characterize the safety and effectiveness of patisiran and…
- Trial name
- ConTTRibute (NCT04561518)
- Where
- 40 sites in United States, Brazil and 10 more
- Volunteers
- 1,500 people
- Sponsor
- Alnylam Pharmaceuticals
Hereditary Transthyretin-mediated (hATTR) Amyloidosis, Polyneuropathy
Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program
The purpose of this study is to collect and evaluate pregnancy outcomes, pregnancy complications, and fetal/neonatal/infant outcomes in women exposed to patisiran-LNP.
- Trial name
- NCT05040373
- Where
- 7 sites in United States, France and 5 more
- Volunteers
- 10 people
- Sponsor
- Alnylam Pharmaceuticals
Sensorineural Hearing Loss, Bilateral
Gene Therapy Trial for Otoferlin Gene-mediated Hearing Loss
Part A of this trial evaluated the safety and tolerability of a single surgical administration procedure in one or both ear(s) with one of two dose levels of AAVAnc80-hOTOF (LY888891) and evaluated the Akouos Delivery Device…
- Trial name
- NCT05821959
- Testing
- AAVAnc80-hOTOF via Akouos Delivery Device, AAVAnc80-hOTOF via Akouos Delivery Device and Precision Delivery Mechanism
- Where
- 9 sites in United States, Canada and 2 more
- Volunteers
- 48 people
- Sponsor
- Eli Lilly and Company
Optic Neuritis, Optic Neuropathy
Prospective Evaluation of the 2022 Optic Neuritis Criteria When Optic Neuritis Is Suspected
Optic neuritis is an important cause of acute or subacute visual loss. In clinical practice, optic neuritis must often be distinguished from other optic neuropathies, retinal diseases, anterior-segment or ocular media disorders…
- Conditions
- Optic Neuritis, Optic Neuropathy, Visual Loss
- Trial name
- GX-ICON (NCT07623252)
- Where
- 9 sites in China
- Volunteers
- 500 people
- Sponsor
- First Affiliated Hospital of Guangxi Medical University
Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth
Natural History Study for Charcot Marie Tooth Disease
The goal of this Natural History Study for Charcot-Marie-Tooth is to acquire, record, and analyze patient-reported data and associated genetic reports, Electronic Health Records (EHRs) and clinical notes to identify the burden…
- Conditions
- Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth, Charcot-Marie-Tooth Disease, Type IA, Charcot-Marie-Tooth Disease Type 2A
- Trial name
- NCT05902351
- Where
- United States
- Volunteers
- 10,000 people
- Sponsor
- Hereditary Neuropathy Foundation
AL Amyloidosis (AL), Cardiac Amyloidosis
Multimodal Radiomics Model (18F-FAPI PET/CT + CMR) for AL Cardiac Amyloidosis Prognosis
1. Goal of the Study: The goal of this prospective observational study is to develop and validate a novel, non-invasive method for predicting the prognosis of patients with light-chain cardiac amyloidosis (AL-CA).
- Trial name
- AL-CA (NCT07103863)
- Where
- China
- Volunteers
- 49 people
- Sponsor
- Beijing Anzhen Hospital
Post-COVID-19 Condition, Ulcerative Colitis
Emerging Indications for Hyperbaric Oxygen Treatment
This study will evaluate the effectiveness of hyperbaric oxygen therapy (HBOT) on treating emerging indications (i.e., conditions that have shown to potentially benefit from HBOT) using the Multicenter Registry for Hyperbaric…
- Conditions
- Post-COVID-19 Condition, Ulcerative Colitis, Crohn Disease, Calciphylaxis
- Trial name
- NCT07240649
- Testing
- Hyperbaric oxygen treatment (HBOT), 100% Oxygen
- Volunteers
- 100 people
- Sponsor
- Jay C. Buckey Jr.
Deep Gluteal Syndrome
Neural Gliders With and Without ESWT for Deep Gluteal Syndrome
This study is a randomized controlled trial comparing Neural Gliders with and without Extracorporeal Shock Wave Therapy (ESWT) in patients with Deep Gluteal Syndrome.
- Trial name
- Neural Gliders (NCT07772674)
- Testing
- Group A= Neural Gliders with ESWT, Group B= Neural Gliders without ESWT
- Where
- Pakistan
- Volunteers
- 46 people
- Sponsor
- University of Management and Technology Sialkot Pakistan