Clinical trials: Charcot Marie Tooth Disease
Charcot-Marie-Tooth Disease Type 1A
Evaluate the Safety and Efficacy of EN001 in Patients With Charcot-Marie-Tooth Disease Type 1A(CMT1A) (Phase 1b: Open-label, Dose-escalation, Single-center; Phase 2a: Randomized, Double-blind, Placebo-controlled, Multicenter)
A Phase 1b/2a Clinical Trial to Evaluate the Safety and Efficacy of EN001 in Patients with Charcot-Marie-Tooth Disease type 1A(CMT1A) (Phase 1b: Open-label, Dose-escalation, Single-center; Phase 2a: Randomized, Double-blind…
- Trial name
- NCT06328712
- Testing
- EN001
- Where
- 3 sites in South Korea
- Volunteers
- 27 people
- Sponsor
- ENCell
CMT
Analysis of Pain and Quality of Life in Patients With Charcot-Marie-Tooth Neuropathy (CMT)
The study aims to qualitatively and quantitively evaluate pain in patients suffering from CMT with 3 standardized questionnaires (SF-36, NPSI-G, PAIN DETECT) as well as its impact on the quality of life.
- Trial name
- NCT03966287
- Where
- Germany
- Volunteers
- 200 people
- Sponsor
- University Medical Center Goettingen
Down Syndrome
Non-Invasive Brain Stimulation to Improve Language in Down Syndrome.
Down syndrome (DS) is associated with cognitive deficits, caused by alterations in neuroplasticity and synaptic transmission. Non-invasive brain stimulation techniques, such as transcranial direct current stimulation (tDCS), can…
- Trial name
- NCT07044804
- Testing
- Active Group (Active Anodal tDCS to the left IFG or Cathodal tDCS to the left IFG)
- Where
- Italy
- Volunteers
- 36 people
- Sponsor
- Floriana Costanzo
Mood Disorders
Neurofilament Light Chain Correlation With Severity of Symptoms and Cognitive Decline in Mood Disorders
Explore correlation of neurofilament light chain serum level and severity of symptoms and cognitive impairment in mood disorders Explore How novel brain markers as neurofilament light chain can be useful in detection and…
- Trial name
- NCT06877442
- Testing
- serum level of neurofilament light chain
- Volunteers
- 90 people
- Sponsor
- Assiut University
Hypercapnia
Effects of Permissive Hypercapnia on Concentration of Postoperative Plasma Neurofilament Light
The aim of this study is to conduct a prospective, single-center randomized controlled study to investigate the changes of plasma NFL concentration in patients undergoing laparoscopic surgery with mild hypercapnia, and further…
- Trial name
- NCT05793437
- Testing
- permissive hypercapnia, normocapnia
- Where
- China
- Volunteers
- 84 people
- Sponsor
- Affiliated Hospital of Jiaxing University
Charcot-Marie-Tooth Disease, Type IA
Determine the Safety and Dose of EN001 in Patients With Charcot-Marie-Tooth Disease (CMT) Type 1A
Open-label, Dose-escalation, Phase 1 Clinical Trial to Determine the Safety and Dose of EN001 in Patients with Charcot-Marie-Tooth disease (CMT) type 1A
- Trial name
- NCT05333406
- Testing
- EN001
- Where
- South Korea
- Volunteers
- 9 people
- Sponsor
- ENCell
Charcot-Marie-Tooth Disease
A Study to Assess the Safety and Effectiveness of FLX-787 in Subjects With Charcot-Marie-Tooth Disease Experiencing Muscle Cramps.
The COMMIT Study will assess the safety and effectiveness of FLX-787 in men and women with Charcot-Marie-Tooth disease (CMT) experiencing muscle cramps.
- Trial name
- COMMIT (NCT03254199)
- Testing
- FLX-787-ODT (orally disintegrating tablet)
- Where
- 20 sites in United States
- Volunteers
- 27 people
- Sponsor
- Flex Pharma, Inc.
Frontotemporal Dementia (FTD), GRN Related Frontotemporal Dementia
Predict to Prevent PGRN Disease
Progranulin (GRN or PGRN) mutations are among the most common genetic causes of frontotemporal lobar degeneration (FTLD). With the advent of gene-specific therapeutic interventions, an accurate knowledge of the presymptomatic…
- Trial name
- PREVENT-PGRN (NCT06711510)
- Testing
- Clinical, behavioral and cognitive evaluation, blood sampling, brain MRI
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
Brain Trauma
Plasma Neurofilament Light and Its Relationship With Omega-3 Status and Soccer Heading in Women Soccer Players
The purpose of this study is to (1) assess how a competitive season of purposeful gameplay soccer heading in collegiate women soccer players is related to a blood biomarker for sports-related brain injury, plasma neurofilament…
- Trial name
- NCT05457023
- Testing
- Plasma Neurofilament Light (NF-L), Omega-3 Index, HeadCount Questionnaire
- Where
- 2 sites in United States
- Volunteers
- 27 people, healthy volunteers welcome
- Sponsor
- University of Kansas Medical Center
Charcot-Marie-Tooth Disease Type 1A
Identification of Novel Biomarkers in Early Charcot-Marie-Tooth 1A Disease
This is a 2-year follow-up study of a cohort of 35 CMT1A patients and 20 healthy volunteers. The main objective is identifying prognostic markers for CMT1A using multi-omics analysis.
- Trial name
- CMT-MODS (NCT07049588)
- Testing
- Quantitative neuromuscular MRI, Skin biopsy, Clinical scores
- Where
- France
- Volunteers
- 55 people, healthy volunteers welcome
- Sponsor
- Assistance Publique Hopitaux De Marseille
Carpal Tunnel Syndrome, Entrapment Neuropathy
Efficacy of Keyhole Approach to Carpal Tunnel Syndrome Under Ambulatory Strategy
Prospective controlled clinical trial in which a minimally invasive microsurgical approach was used following the keyhole principle in 55 patients and 65 hands under local anesthesia and ambulatory strategy.
- Trial name
- NCT03062722
- Testing
- keyhole approach
- Volunteers
- 55 people
- Sponsor
- Ivan Segura Duran
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)
Charcot Marie Tooth Disease (CMT), Pes Cavovarus
Walking Function Outcomes Following Surgical Correction With Rehabilitation Versus Physical Therapy Alone in Charcot-Marie-Tooth Disease: A Bidirectional Cohort Study
The goal of this study is to compare changes in walking ability in people with Charcot-Marie-Tooth disease (CMT) who receive two different treatment approaches for foot deformities that affect walking.
- Conditions
- Charcot Marie Tooth Disease (CMT), Pes Cavovarus, Surgery, Exercise Training
- Trial name
- CMT-WALK (NCT07432035)
- Testing
- Functional Surgery plus Rehabilitation, Physical Exercise
- Volunteers
- 200 people
- Sponsor
- Peking University Third Hospital
Alzheimer Disease, Cognitive Dysfunction, Cognitive Disorder
DIV-AD BARCELONA: Alzheimer's Blood-Based Biomakers for a Diverse Community
The DIV-AD study aims to find out whether levels of Alzheimer's disease markers in blood differ among the main ethnical groups living in central Barcelona.
- Conditions
- Alzheimer Disease, Cognitive Dysfunction, Cognitive Disorder, Dementia, Neurodegenerative Disease
- Trial name
- DIV-AD (NCT07521722)
- Where
- Spain
- Volunteers
- 250 people, healthy volunteers welcome
- Sponsor
- Barcelonabeta Brain Research Center, Pasqual Maragall Foundation
Cognitive Impairment, Frail Older Adults
Cognitive Fraility and Biomarkers in Older Patients
this study aimed to investigate the association between cognitive fraility and selected plasma and cerebrospinal fluid biomarkers related to neurodegenetaion and neuroinflammation in older adult patients undergoing elective…
- Conditions
- Cognitive Impairment, Frail Older Adults, Orthopaedic Surgery
- Trial name
- NCT07184073
- Testing
- assessment Mini mental state examination, assessment FRAIL scale, collecting cerebrospinal fluid during regional anesthesia
- Where
- Turkey (Türkiye)
- Volunteers
- 129 people
- Sponsor
- Baskent University
CMT1A
Ulipristal Acetate In Disease Charcot-Marie-Tooth Type of 1A
The disease Charcot-Marie-Tooth disease type 1A (CMT1A) is the most common inherited peripheral neuropathy, for which no treatment has proved its effectiveness.
- Trial name
- UPACOMT (NCT02600286)
- Testing
- EllaOne
- Where
- 5 sites in France
- Volunteers
- 23 people
- Sponsor
- University Hospital, Strasbourg, France
Charcot-Marie-Tooth Disease
Hearing and Balance Disorders in Peripheral Neuropathy
The long term goal of this proposal is to precisely characterize the auditory and vestibular abilities of children with Charcot-Marie-Tooth (CMT) and how these abilities evolve during the progression of the disorder.
- Trial name
- NCT05827419
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Nemours Children's Clinic
Low Back Pain, Nerve Entrapment Syndrome
Ultrasound Localization and Guided Injection for Superior Cluneal Nerve Entrapment
Low back pain (LBP) is a common complaint in the clinical setting. Among all the differential diagnosis for LBP, superior cluneal nerve (SCN) entrapment is the commonly omitted one.
- Trial name
- NCT04478344
- Testing
- Ultrasound guided hydrodissection of superior cluneal nerve
- Where
- Taiwan
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- National Taiwan University Hospital
CMT
The Safety and Tolerability of Engensis (VM202) in Patients With Charcot-Marie-Tooth Disease Subtype 1A (CMT1A)
To assess the safety and tolerability of the investigational product Engensis (VM202) injected in the weakened lower limb muscles of CMT1A patients
- Trial name
- NCT05361031
- Testing
- Engensis (VM202)
- Where
- South Korea
- Volunteers
- 12 people
- Sponsor
- Helixmith Co., Ltd.