Clinical trials: Polyneuropathies
Chronic Inflammatory Demyelinating Polyneuropathy
Non-invasive Vagus Nerve Stimulation (nVNS) in Pediatric Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Participants will be requested to deliver non-invasive vagus nerve stimulation (nVNS) two times per day, at least five days per week.
- Trial name
- NCT03772717
- Testing
- Non-invasive vagus nerve stimulation (nVNS), Standard of care treatment
- Where
- United States
- Volunteers
- 2 people
- Sponsor
- Emory University
Guillain-Barre Syndrome
Early and Late Prognosis in Patients With Guillain-Barre Syndrome
The goal of this observational study is to explore significant indicators to predict the early prognosis and late prognosis in patients with Guillain-Barre syndrome.
- Trial name
- NCT06041451
- Testing
- Intravenous Immunoglobulins, Human, Methylprednisolone 500 MG Injection
- Where
- China
- Volunteers
- 450 people
- Sponsor
- First Affiliated Hospital of Wenzhou Medical University
Mitochondrial Encephalomyopathies, Hereditary Spastic Paraplegia
Natural History Study of Patients with HPDL Mutations
This study uses medical records that allow retrospective data extraction of clinical manifestation to assess the natural history of HPDL mutations
- Conditions
- Mitochondrial Encephalomyopathies, Hereditary Spastic Paraplegia, Spastic Paraplegia, White Matter Disease
- Trial name
- NCT05848271
- Testing
- Patient Registry, Dry blood spots sampling
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of California, San Diego
Blue Rubber Bleb Nevus Syndrome, Venous Malformation
Efficacy of Rapamycin (Sirolimus) in the Treatment of BRBNS, Hereditary or Sporadic Venous Malformation
A prospective, nonrandomized, open-label, single-arm clinical trial to study efficacy of rapamycin (sirolimus) in the treatment of Blue Rubber Bleb Nevus Syndrome, hereditary or sporadic venous malformation
- Trial name
- NCT03767660
- Testing
- Rapamycin
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Peking Union Medical College Hospital
Pediatric Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Two Dose Levels of Privigen in Pediatric CIDP
A randomized, open-label, prospective, multicenter study designed to investigate 2 dose levels in pediatric subjects 2 to ≤ 17 years of age with confirmed or possible CIDP, either previously exposed to IVIG treatment or unexposed…
- Trial name
- NCT03684018
- Testing
- IgPro10
- Where
- 9 sites in United States
- Volunteers
- 30 people
- Sponsor
- CSL Behring
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
Idiopathic Toe Walking, Cerebral Palsy
The Effect of Targeting the Plantaris Muscle-tendon in Surgical Correction of Ankle Equinus in Children
Tight ankle muscles can produce ankle equinus (limited ability to pull the foot upward) and occur often in children, significantly impacting their ability to walk.
- Conditions
- Idiopathic Toe Walking, Cerebral Palsy, Hereditary Spastic Paraparesis, Traumatic Brain Injury
- Trial name
- Plantaris Sx (NCT07090057)
- Testing
- Tendoachilles (TA) lengthening or gastrocnemius (GN) recession, then Plantaris tenotomy, Plantaris tenotomy, then Tendoachilles (TA) lengthening or gastrocnemius (GN) recession
- Where
- Canada
- Volunteers
- 42 people
- Sponsor
- University of Alberta
Motor Function, Gait
Spinal Cord Stimulation Therapy for Hereditary Spastic Paraplegias Patients
It's a single-center, prospective, open label clinical study with a 12 months follow-up period, to investigate the therapeutic effect and safety of spinal cord stimulation (SCS) on motor function and gait in patients with pure…
- Trial name
- NCT05196178
- Testing
- Spinal cord stimulation
- Where
- China
- Volunteers
- 12 people
- Sponsor
- Xuanwu Hospital, Beijing
Pediatric Cancer, Pediatric Brain Tumor
Playful Sensorimotor Training in Pediatric Brain Tumor Patients
Chemotherapy-induced peripheral neuropathy (CIPN) is a highly prevalent and clinically relevant side-effect of cancer treatment.
- Conditions
- Pediatric Cancer, Pediatric Brain Tumor, Chemotherapy-induced Peripheral Neuropathy, Sensorimotor Polyneuropathy
- Trial name
- RESET (NCT03334162)
- Testing
- Sensorimotor training
- Where
- Switzerland
- Volunteers
- 20 people
- Sponsor
- University of Basel
Motor Neuron Diseases, Spinocerebellar Ataxias
Rare Disease Registry Database for Movement Disorders in Southern Anhui Province
According to the World Health Organization (WHO) definition, a rare disease is a disease affecting fewer than 6.5 people per 10,000; in China, it generally refers to diseases with a prevalence of less than 1/10,000 (i.e., with a…
- Conditions
- Motor Neuron Diseases, Spinocerebellar Ataxias, Hereditary Muscular Dystrophy, Hereditary Spastic Paraplegia
- Trial name
- W-MoSCA (NCT07838883)
- Where
- China
- Volunteers
- 180 people
- Sponsor
- First Affiliated Hospital of Wannan Medical College
Encephalitis, Myelitis
Neurologic Manifestations of the Arbovirus Infection in Colombia
This is a multi-center case-control study that aims to define the association between the exposure to an arbovirus infection and the development of a neurological syndrome in patients from Colombia.
- Conditions
- Encephalitis, Myelitis, Guillain-Barre Syndrome, Cranial Nerve Palsies
- Trial name
- NCT03206541
- Where
- 8 sites in Colombia
- Volunteers
- 570 people, healthy volunteers welcome
- Sponsor
- Johns Hopkins University
Proteus Syndrome, PIK3CA-Related Overgrowth Spectrum (PROS)
Expanded Access to Provide ARQ 092 for the Treatment of Overgrowth Diseases and/or Vascular Anomalies
ARQ 092 is being investigated for patients with overgrowth diseases and/or vascular anomalies with genetic alterations of the PI3K/AKT pathway and may be available for patients who are ineligible for an ongoing ARQ 092 clinical…
- Conditions
- Proteus Syndrome, PIK3CA-Related Overgrowth Spectrum (PROS), Growth Disorders
- Trial name
- NCT03317366
- Testing
- ARQ 092
- Sponsor
- ArQule, Inc., a subsidiary of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc. (Rahway, NJ USA)
Cauda Equina Syndrome, Functional Neurological Disorder
BLB Study Back or Leg Pain and Bladder Symptoms Study
The research focuses on Cauda Equina Syndrome (CES) which describes damage to the nerves in the lower back which supply bladder, bowels, sexual function and the legs usually from slipped discs in the spine.
- Trial name
- BLB (NCT03325374)
- Testing
- Questionnaires and examination
- Where
- United Kingdom
- Volunteers
- 100 people
- Sponsor
- University of Edinburgh
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
Vascular Malformations
Treatment of Congenital Vascular Malformations Using Sirolimus: Improving Quality of Life
Congenital vascular anomalies are uncommon and belong to the group of rare diseases.These vascular malformations can cause serious complications including obstruction of vital organs and their function, recurrent infection and…
- Trial name
- Sirolimus (NCT03987152)
- Testing
- Sirolimus
- Where
- Netherlands
- Volunteers
- 75 people
- Sponsor
- Radboud University Medical Center
Familial Dysautonomia
The Safety and Tolerability of Kinetin, in Patients With Familial Dysautonomia
This is a study of kinetin, a nutritional supplement that corrects the mRNA splicing defect in patients with familial dysautonomia (FD, also known as Riley Day syndrome or hereditary sensory and autonomic neuropathy type III).
- Trial name
- NCT02274051
- Testing
- Kinetin
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- NYU Langone Health
Hereditary Spastic Paraplegia, Neurodegenerative Diseases
Hereditary Spastic Paraplegia Genomic Sequencing Initiative (HSPseq)
The purpose of the HSP Sequencing Initiative is to better understand the role of genetics in hereditary spastic paraplegia (HSP) and related disorders.
- Conditions
- Hereditary Spastic Paraplegia, Neurodegenerative Diseases, Pediatric Disorder, Spasticity, Muscle
- Trial name
- NCT05354622
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Boston Children's Hospital
Hypoxic-Ischemic Encephalopathy
Study of Cerebrolysin for Treatment of Infants With History of Neonatal Hypoxic Ischemic Encephalopathy
The purpose of this study is to determine whether nerve growth factor (cerebrolysin®) therapy will improve the psychomotor outcome in infants with moderate and severe hypoxic ischemic encephalopathy after hospital discharge.
- Trial name
- CerebroHIE (NCT01059461)
- Testing
- Cerebrolysin®
- Where
- 2 sites in Egypt
- Volunteers
- 40 people
- Sponsor
- Sahar M.A. Hassanein, MD
Hereditary Spastic Paraplegia
12-Week Strength and Functional Exercise Program for Hereditary Spastic Paraplegia Trial (HSPMOVE)
This study aims to evaluate whether a 12-week program of strength and functional exercises can improve movement and daily function in people with Hereditary Spastic Paraplegia (HSP).
- Trial name
- HSPMOVE (NCT07561359)
- Testing
- Individualized Functional and Strength Exercises
- Where
- Brazil
- Volunteers
- 20 people
- Sponsor
- Hospital de Clinicas de Porto Alegre
Megalencephaly-capillary Malformation Polymicrogyria Syndrome (MCAP)
Assessment of the Efficacy and Safety of Alpelisib (BYL719) in Pediatric and Adult Patients With Megalencephaly-CApillary Malformation Polymicrogyria Syndrome (MCAP)
This study is a two periods multi-center Phase II trial, with a 6 months double-blind, placebo-controlled period followed by open label period, to assess the efficacy and safety of alpelisib (BYL719) in pediatric and adult…
- Trial name
- SESAM (NCT05577754)
- Testing
- Alpelisib (BYL719), Optional lumbar puncture + blood sample
- Where
- 13 sites in France
- Volunteers
- 20 people
- Sponsor
- Centre Hospitalier Universitaire Dijon