Clinical trials: Charcot Disease
Amyotrophic Lateral Sclerosis
Validation of Czech Language Versions of Questionnaires for ALS Patients' Functional Status and Biomarker Long-term Follow-up
Questionnaires and scales used to assess the clinical status and quality of life of patients with amyotrophic lateral sclerosis (ALS) are an important tool to monitor the disease progression and current needs of patients.
- Trial name
- NCT06426030
- Where
- Czechia
- Volunteers
- 100 people
- Sponsor
- Masaryk University
MS (Multiple Sclerosis), Multiple Sclerosis (MS) - Relapsing-remitting
Comparison of Efficacy and Safety in Patients Switching From MabThera® to Rixathon® in Relapsing-Remitting Multiple Sclerosis
This observational study investigates if switching from MabThera® to Rixathon® is associated with changes in disease activity and/or safety in people with multiple sclerosis (MS).
- Conditions
- MS (Multiple Sclerosis), Multiple Sclerosis (MS) - Relapsing-remitting, Multiple Sclerosis (Relapsing Remitting)
- Trial name
- NCT07235644
- Where
- Sweden
- Volunteers
- 184 people
- Sponsor
- Uppsala University
Charcot-Marie-Tooth Disease
A Registered Cohort Study on Charcot-Marie-Tooth Disease
The aim of the study is to analyze the natural history data data from Charcot-Marie-Tooth disease and related disorders in China, to assess the clinical, genetic, epigenetic features of patients with Charcot-Marie-Tooth disease…
- Trial name
- NCT04010188
- Where
- China
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- Ning Wang, MD., PhD.
Charcot-Marie-Tooth Disease
Follow up and Observation of Charcot Marie Tooth Disease in Families
The study is aimed to test the hypothesis that there is anticipation in CMT
- Trial name
- NCT00149045
- Where
- Israel
- Sponsor
- Hadassah Medical Organization
Traumatic Brain Injury
The Biomarkers in the Hyperbaric Oxygen Brain Injury Treatment Trial (BioHOBIT)
There are no therapeutic agents that have been shown to improve outcomes from severe traumatic brain injury (TBI). Critical barriers to progress in developing treatments for severe TBI are the lack of: 1) monitoring biomarkers…
- Trial name
- NCT04855396
- Where
- 11 sites in United States, Canada
- Volunteers
- 150 people
- Sponsor
- University of Michigan
Amyotrophic Lateral Sclerosis
Italian Validation of the Beaumont Behavioural Inventory (BBI)
Up to 50% of patients affected with amyotrophic lateral sclerosis (ALS) can show behavioral dysfunctions within the spectrum of frontotemporal degenerations (FTD) - namely, apathy, disinhibition, loss of sympathy/empathy…
- Trial name
- BBI (NCT04440644)
- Testing
- BBI
- Where
- Italy
- Volunteers
- 190 people, healthy volunteers welcome
- Sponsor
- Istituti Clinici Scientifici Maugeri SpA
Charcot-Marie-Tooth Disease, Healthy
Charcot-Marie-Tooth Disease (CMT) Biological Sample Collection for IPSC Generation and Biobanking
The New York Stem Cell Foundation (NYSCF) Research Institute is performing this research to accelerate Charcot-Marie-Tooth disease research and drug development by using cells from the body (such as skin or blood cells) to make…
- Trial name
- NCT06203093
- Testing
- Biological Sample Collection
- Where
- United States
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- New York Stem Cell Foundation Research Institute
Amyotrophic Lateral Sclerosis
Individual Patient Expanded Access IND of Autologous HBadMSCs for the Treatment of Amyotrophic Lateral Sclerosis
The drug for this submission is Hope Biosciences' autologous, adipose-derived culture-expanded mesenchymal stem cells (HB-adMSCs) for the treatment of a single patient with Amyotrophic Lateral Sclerosis (ALS).
- Trial name
- NCT04514952
- Testing
- HB-adMSCs
- Where
- United States
- Sponsor
- Hope Biosciences Research Foundation
Motor Neuron Disease
EncoreAnywhere Use in Motor Neurone Disease
Motor neurone disease is a progressive incurable disease causing weakness and paralysis of muscles. Respiratory failure is the most common cause of death in motor neurone disease.
- Trial name
- NCT03154450
- Testing
- EncoreAnywhere with data available to review, EncoreAnywhere with no data available to review
- Where
- United Kingdom
- Volunteers
- 19 people
- Sponsor
- Sheffield Teaching Hospitals NHS Foundation Trust
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia
Open-label Extension (OLE) Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) and/or Frontotemporal Dementia (FTD)
This is an OLE study conducted to evaluate the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and clinical effects of WVE-004 in adult participants with ALS, FTD, or mixed ALS/FTD phenotype with a documented…
- Trial name
- NCT05683860
- Testing
- WVE-004 10 mg Q12W
- Where
- 3 sites in Netherlands, United Kingdom
- Volunteers
- 8 people
- Sponsor
- Wave Life Sciences USA, Inc.
Charcot-Marie-Tooth Disease, Hereditary Motor and Sensory Neuropathies
Ascorbic Acid Treatment in CMT1A Trial (AATIC)
Charcot-Marie-Tooth type IA (CMT1A) is the most prevalent hereditary peripheral neuropathy. Demyelination of peripheral nerves is the hallmark of CMT1A.
- Trial name
- AATIC (NCT00271635)
- Testing
- ascorbic acid
- Where
- Netherlands
- Volunteers
- 13 people
- Sponsor
- Academisch Medisch Centrum - Universiteit van Amsterdam (AMC-UvA)
Alpha-Thalassemia, Beta-Thalassemia
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases.
- Conditions
- Alpha-Thalassemia, Beta-Thalassemia, Amyloidosis, Amyotrophic Lateral Sclerosis
- Trial name
- NCT06539169
- Where
- United States
- Volunteers
- 1,000 people
- Sponsor
- xCures
Hypoxic-Ischemic Encephalopathy
A Study of a Blood Marker in Newborns With Brain Injury Caused by Lack of Oxygen at Birth
Perinatal asphyxia is a significant health problem with an incidence of 1 to 8 per 1,000 live births and can lead to serious morbidity and mortality during the neonatal period.
- Trial name
- NCT07292220
- Where
- Turkey (Türkiye)
- Volunteers
- 20 people, healthy volunteers welcome
- Sponsor
- Mustafa Gürkan
ALS
National Registry of Veterans With Amyotrophic Lateral Sclerosis and DNA Bank
This study will identify living veterans with amyotrophic lateral sclerosis (ALS) through a national registry that is being developed. Diagnosis of ALS will be verified by study neurologists via medical record review.
- Trial name
- ALS Registry (NCT00076154)
- Where
- United States
- Volunteers
- 2,121 people
- Sponsor
- US Department of Veterans Affairs
Amyotrophic Lateral Sclerosis
Home Care Needs and Current Nursing Status of Patients With Amyotrophic Lateral Sclerosis
The purpose of this study is to comprehensively investigate and analyze the home care needs and status quo of ALS patients in China, and to clarify their home care needs and status quo, so as to provide reference for the…
- Trial name
- NCT04953481
- Testing
- survey
- Where
- China
- Volunteers
- 252 people
- Sponsor
- Peking University Third Hospital
Charcot-Marie-Tooth Disease
Irisin Levels in Patients With Charcot-Marie-Tooth (CMT) Disease
Irisin is an exercise-mimetic myokine secreted by skeletal muscle. Compelling evidence in animal models and humans showed that Irisin prevents onset of musculoskeletal atrophy and its low serum levels are predictive of…
- Trial name
- IRICDE (NCT04786522)
- Testing
- Bioimpedenziometry, Handgrip strenght evaluation, Evaluation on blood specimens
- Where
- Italy
- Volunteers
- 20 people
- Sponsor
- University of Bari
Duchenne Muscular Dystrophy, Amyotrophic Lateral Sclerosis
Evaluation of a Mechanical Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders
The hypothesis is that a mechanical insufflation-exsufflation (MI-E) is associated with a decrease in the number of intubations and more rapid clinical improvement in children and adults with neuromuscular disease who are…
- Conditions
- Duchenne Muscular Dystrophy, Amyotrophic Lateral Sclerosis, Neuromuscular Diseases
- Trial name
- Nemucough (NCT00839033)
- Testing
- mechanical insufflation - exsufflation, Standard respiratory physiotherapy
- Where
- France
- Volunteers
- 14 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Amyotrophic Lateral Sclerosis
Appetite and Related Factors in Patients With ALS
Chinese cnaq scale was used to evaluate the appetite changes of Chinese ALS patients; Objective to investigate the related factors of appetite changes in ALS patients; Objective to investigate the effect of anorexia on the…
- Trial name
- NCT04953624
- Testing
- Appetite rating scale CNAQ
- Where
- China
- Volunteers
- 200 people
- Sponsor
- Peking University Third Hospital
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
Asthma
Charcot Leyden Crystals in Pathology
Charcot Leyden crystals (CLC) are sometime reported in pathological reports of airway or sinonasal samples but their clinical significance remain elusive.
- Trial name
- CLCpath (NCT03999242)
- Where
- France
- Volunteers
- 200 people
- Sponsor
- University Hospital, Montpellier