Clinical trials: Motor Neuropathy
Charcot-Marie-Tooth Disease, Type IA
Assessing Long Term Safety and Tolerability of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A
All randomised patients with Charcot-Marie-Tooth Type 1A (CMT1A) who completed the primary study CLN-PXT3003-02, i.e. treatment with PXT3003 or placebo, are eligible to continue in the extension study CLN-PXT3003-03.
- Trial name
- PLEO-CMT-FU (NCT03023540)
- Testing
- PXT3003
- Where
- 23 sites in United States, Belgium and 5 more
- Volunteers
- 187 people
- Sponsor
- Pharnext S.C.A.
Spinal Muscular Atrophy, Charcot-Marie-Tooth Disease
Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases
The goal of this study is to establish a genetic registry of patients with early-onset motor neuron and neuromuscular diseases. The investigators will collect samples from patients with a motor neuron or a neuromuscular disorder…
- Conditions
- Spinal Muscular Atrophy, Charcot-Marie-Tooth Disease, Muscular Dystrophy, Spinal Muscular Atrophy With Respiratory Distress 1
- Trial name
- NCT02532244
- Testing
- sample collection
- Where
- 3 sites in United States
- Volunteers
- 230 people, healthy volunteers welcome
- Sponsor
- Nemours Children's Clinic
Giant Axonal Neuropathy (GAN)
Intraneural Administration of scAAV9/JeT-GAN Into the Vagus Nerve for Patients With Giant Axonal Neuropathy (GAN)
Giant axonal neuropathy (GAN) is a rare pediatric disorder caused by autosomal recessive mutations in the GAN gene. GAN is a multisystem, neurodegenerative disorder affecting the peripheral nervous system (PNS), central nervous…
- Trial name
- NCT07543991
- Testing
- scAAV9/JeT-GAN
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- University of Texas Southwestern Medical Center
Obstetric Brachial Plexus Injury, Birth Related Brachial Plexus Injury
Investigating the Effectiveness of Graded Motor Imagery Training in Children With Obstetric Brachial Plexus Injury
The aim of this study is to investigate the effect of graded motor imagery training on upper extremity motor function, proprioception, functional mobility, balance and quality of life in patients with obstetric brachial plexus…
- Conditions
- Obstetric Brachial Plexus Injury, Birth Related Brachial Plexus Injury, Neonatal Brachial Plexus Palsy
- Trial name
- NCT06966544
- Testing
- Conventional Physiotherapy, Graded Motor Imagery
- Where
- Turkey (Türkiye)
- Volunteers
- 42 people
- Sponsor
- Izmir Bakircay University
Charcot-Marie-Tooth Disease
Charcot-Marie-Tooth Disease (CMT) Infant Scale (INC-6611)
The purpose of this study is to develop and validate a clinical outcome measure to evaluate disability and disease progression of children 3 years of age and younger (infants and toddlers) with various types of…
- Trial name
- NCT02979145
- Testing
- No intervention involved
- Where
- 4 sites in United States, Australia and 1 more
- Volunteers
- 200 people, healthy volunteers welcome
- Sponsor
- Sydney Children's Hospitals Network
Amyotrophic Lateral Sclerosis, Frontotemporal Dementia
Phenotype, Genotype & Biomarkers in ALS and Related Disorders
The goals of this study are: (1) to better understand the relationship between the phenotype and genotype of amyotrophic lateral sclerosis (ALS) and related diseases, including primary lateral sclerosis (PLS), hereditary spastic…
- Conditions
- Amyotrophic Lateral Sclerosis, Frontotemporal Dementia, Primary Lateral Sclerosis, Hereditary Spastic Paraplegia
- Trial name
- NCT02327845
- Where
- 15 sites in United States, Germany and 1 more
- Volunteers
- 708 people, healthy volunteers welcome
- Sponsor
- University of Miami
Hereditary Spastic Paraparesis
Testing Miglustat Administration in Subjects With Spastic Paraplegia 11
Hereditary spastic paraparesis type 11 (SPG11) is caused by mutations in the SPG11 gene that produces spatacsin, a protein involved in lysosomal function.
- Trial name
- TreatSPG11 (NCT04768166)
- Testing
- Miglustat 100 MG
- Where
- Italy
- Volunteers
- 10 people
- Sponsor
- IRCCS Fondazione Stella Maris
CMT, CMT (Charcot Marie Tooth Disease)
A Multi-omic Approach to the Identification of Novel Biomarkers in Early Charcot-Marie-Tooth 1A Disease (CMT1A)
The most common inherited neuropathy is Charcot-Marie-Tooth disease type 1A (CMT1A), caused by a duplication of the gene expressing PMP22.
- Conditions
- CMT, CMT (Charcot Marie Tooth Disease), CMT - Charcot-Marie-Tooth Disease, CMT1A
- Trial name
- CMT-MODs (NCT07476365)
- Where
- Germany
- Volunteers
- 70 people, healthy volunteers welcome
- Sponsor
- University Medical Center Goettingen
Hereditary Spastic Paraplegia, Spastic Paraplegia Type 11
Trehalose Administration in Subjects With Spastic Paraplegia 11 (3AL-SPG11)
Hereditary spastic paraparesis type 11 (SPG11) is caused by mutations in the SPG11 gene that produces spatacsin, a protein involved in lysosomal function.
- Trial name
- 3AL-SPG11 (NCT04912609)
- Where
- Italy
- Volunteers
- 13 people
- Sponsor
- IRCCS Fondazione Stella Maris
Gulf War Illness, Persian Gulf War Syndrome
Study of Gulf War Illness (GWI) by Comparing GWI and Healthy Veterans
Determine if: genetic differences of CNDP1 gene the previously defined GWI/Chronic Fatigue Syndrome (GWI/CFS) cerebrospinal fluid proteome contents psychometric other variables can differentiate between veterans of the 1990-1991…
- Trial name
- NCT00810225
- Where
- United States
- Volunteers
- 516 people, healthy volunteers welcome
- Sponsor
- Georgetown University
Peroneal Muscular Atrophy
Genetics of Charcot-Marie-Tooth Dystrophy and Related Diseases
This is a cross-sectional study to clarify the gene lineage distribution of CMT genes in CMT patients in my country, draw a frequency map of CMT gene distribution, and assist in determining the genetic diagnosis strategy of CMT…
- Trial name
- NCT04967716
- Testing
- basic information, venous blood test
- Where
- China
- Volunteers
- 200 people
- Sponsor
- Peking University Third Hospital
Hereditary Spastic Paraplegia
A Prospective Cohort Study of ITB Treatment for HSP
The investigators conduct a prospective cohort study to explore the treatment effectiveness of continuous infusion of intrathecal baclofen (ITB) for hereditary spastic paraplegia (HSP) in China, delve into the optimal timing for…
- Trial name
- ITB-HSP (NCT06844734)
- Where
- China
- Volunteers
- 50 people
- Sponsor
- Shanghai 6th People's Hospital
HSP, Hereditary Spastic Paraplegia
Safety and Efficacy of AAV9/AP4B1 (BFB-101) For Patients With AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47)
Safety and Efficacy of AAV9/AP4B1 For Patients with AP4B1-related Hereditary Spastic Paraplegia Type 47 (SPG47): A Phase 1/2 Single-Center, Open-Label Study of Stereotactic Intra-cisterna Magna Administration.
- Conditions
- HSP, Hereditary Spastic Paraplegia, Hereditary Spastic Paraparesis, Hereditary Spastic Paraplegia Type 50
- Trial name
- NCT06948019
- Testing
- BFB-101 (AAV9-CBh-AP4B1)
- Where
- United States
- Volunteers
- 5 people
- Sponsor
- BlackfinBio Ltd
Chemotherapy-induced Peripheral Neuropathy, Pediatric Cancer
Preventing Sensory and Motor Dysfunctions in Children Receiving Neurotoxic Chemotherapy
The investigators would like to conduct a prospective, multicenter, two-armed trial (RCT with follow-up). Patients will be recruited from 7 centers (CH/D).
- Trial name
- PrepAIR (NCT05606588)
- Testing
- Playful sensorimotor training
- Where
- 2 sites in Switzerland
- Volunteers
- 131 people
- Sponsor
- University of Basel
Charcot-Marie-Tooth Disease, Type IA, Charcot-Marie-Tooth Disease Type 2A
Muscle MRI in Charcot Mary Tooth Disease: a Prospective Cohort Study
This cohort study (participants with CMT and control participants) has two parts (Part 1: CMT1A cohort; Part2: CMT1B, CMT2A and CMTX1 cohort) and is proposed to take place over 3 years across three sites.
- Conditions
- Charcot-Marie-Tooth Disease, Type IA, Charcot-Marie-Tooth Disease Type 2A, Charcot-Marie-Tooth Disease, Type IB, Charcot-Marie-Tooth Disease, Type X
- Trial name
- NCT03550300
- Where
- United Kingdom
- Volunteers
- 130 people
- Sponsor
- University College, London
Hereditary Spastic Paraplegia
Robot-assisted Walking Treatment in Hereditary Spastic Paraplegia (HSP)
Patients will undergo a combined rehabilitation treatment consisting of 15 sessions on Lokomat and 15 sessions of physical therapy over three weeks for a total of 30 sessions. Two sessions/day for 5 days a week.
- Trial name
- NCT06728787
- Testing
- Robot-Assisted Gait Training
- Where
- Italy
- Volunteers
- 50 people
- Sponsor
- IRCCS Eugenio Medea