Clinical trials: Polyneuropathies
Myasthenia Gravis, CIDP - Chronic Inflammatory Demyelinating Polyneuropathy
A Worldwide Pregnancy Safety Study to Assess Maternal, Fetal, and Infant Outcomes Following Exposure to Efgartigimod During Pregnancy and/or Breastfeeding.
This is a multi-country, prospective safety study of pregnant women exposed to efgartigimod or efgartigimod PH20 SC any time within 25 days prior to conception or any time during pregnancy.
- Trial name
- NCT06299748
- Testing
- Efgartigimod
- Where
- 4 sites in United States, Germany and 2 more
- Volunteers
- 279 people
- Sponsor
- argenx
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
Familial Dysautonomia
Dexmedetomidine Sublingual Film for the Ambulatory Treatment of Hyperadrenergic Autonomic Crisis in Patients With Familial Dysautonomia
The purpose of this placebo controlled interventional study is to collect preliminary data on administering dexmedetomidine in patients with Familial Dysautonomia (FD) during a rapid cessation of autonomic crisis.
- Trial name
- NCT06148311
- Testing
- Dexmedetomidine
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- NYU Langone Health
Cerebral Palsy, Acquired Brain Injury
Assessment of the Psychophysical State During Rehabilitation Treatment With Lokomat
The use of robotic technologies in rehabilitation is an increasingly widespread practice in the health sector: the Lokomat is a medical device intended for walking rehabilitation, consisting of an exoskeleton, a treadmill and a…
- Conditions
- Cerebral Palsy, Acquired Brain Injury, Hereditary Spastic Paraplegia
- Trial name
- NCT05767268
- Testing
- Lokomat
- Where
- Italy
- Volunteers
- 48 people
- Sponsor
- IRCCS Eugenio Medea
Charcot-Marie-Tooth Disease
Phase III Trial Assessing the Efficacy and Safety of PXT3003 in CMT1A Patients
The study will consist of 2 periods: Double-blind Treatment and Open-Label Extension(OLE) Period. -Double-blind Treatment Period - This will be randomized, double-blind, placebo-controlled part of the study which will be…
- Trial name
- PREMIER (NCT04762758)
- Testing
- (RS)-baclofen, naltrexone hydrochloride and D-sorbitol
- Where
- 50 sites in United States, Belgium and 7 more
- Volunteers
- 350 people
- Sponsor
- Pharnext S.C.A.
Chronic Inflammatory Demyelinating Polyradiculoneuropathy (CIDP)
The Evaluation of Efficacy and Safety of Rituximab in Refractory CIDP Patients With IgG4 Autoantibodies
To evaluate the efficacy and safety of rituximab (genetical recombination) intravenously administered to CIDP patients with positive or negative IgG4 autoantibody.
- Trial name
- RECIPE (NCT03864185)
- Testing
- Rituximab (genetical recombination)
- Where
- 4 sites in Japan
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Nagoya University
Guillain Barré Syndrome, Guillain-Barré Syndrome (GBS)
Living With Guillain-Barré Syndrome as Children.
The goal of this qualitative observational study is to explore the lived experience and psychological aftermath of children who were affected by Guillain-Barré Syndrome (GBS) two to five years earlier.
- Trial name
- GUIREQUAL (NCT06940908)
- Where
- 2 sites in France
- Volunteers
- 25 people
- Sponsor
- University Hospital, Toulouse
Antibody-mediated Rejection, NMO Spectrum Disorder
Biorepository and Registry for Plasma Exchange Patients
Patients who have immune mediated diseases commonly undergo plasma exchange (PLEX) procedures to remove pathological substances, typically believed to be antibodies.
- Conditions
- Antibody-mediated Rejection, NMO Spectrum Disorder, TTP, CIDP
- Trial name
- NCT05004493
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Charles M Knudson
Body Mass Index Quantitative Trait Locus 7 Disorder, Tangier Disease
Effect of High Protein Diet on Cardiovascular Diseases Risk Factors Among Overweight and Obese Children
The investigators aimed to determine the effects of HP diet on CVD risk factors among overweight and obese children.
- Trial name
- NCT01886482
- Testing
- nutrition intervention
- Where
- Iran
- Volunteers
- 50 people, healthy volunteers welcome
- Sponsor
- Isfahan University of Medical Sciences
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
Diabetic Polyneuropathy, Diabetes Mellitus, Type 2
Alpha Lipoic Acid and Diabetes Mellitus: Potential Effects on Peripheral Neuropathy
Patients with type 2 DM who are following will be enrolled into the study. Two visits were scheduled for data collection, physical examination and laboratory testing of the patients: the first prior to initiation of alpha lipoic…
- Trial name
- NCT04322240
- Testing
- Alpha lipoic acid
- Where
- Egypt
- Volunteers
- 90 people
- Sponsor
- Benha University
Giant Axonal Neuropathy
Giant Axonal Neuropathy Natural History Study
Giant Axonal Neuropathy (GAN) is a devastating and rare childhood disease. Children with GAN develop increasing muscle weakness, impaired sensation, and at times mental retardation.
- Trial name
- NCT01503125
- Where
- United States
- Volunteers
- 13 people
- Sponsor
- Columbia University
Port Wine Stain
The Role of Angiogenesis-related Pathways in the Development of Port Wine Stains
1. Port wine stain (PWS) is a congenital, progressive vascular malformation of human skin involving the superficial vascular plexus that occurs in estimated 3-5 children per 1,000 live births.
- Trial name
- NCT03948997
- Testing
- Pulsed dye laser (PDL)
- Volunteers
- 80 people
- Sponsor
- xjpfW
Charcot Marie Tooth Disease
Development of Charcot Marie Tooth Disease (CMT) Pediatric Scale for Children With CMT
The primary goal of this project is to develop and test a Charcot Marie Tooth disease (CMT) Pediatric Scale for use in evaluation in natural history CMT study.
- Trial name
- INC-6603 (NCT01203085)
- Where
- 14 sites in United States, Australia and 3 more
- Volunteers
- 1,201 people
- Sponsor
- University of Iowa
Eye Diseases Hereditary, Retinal Disease
Inherited Retinal Degenerative Disease Registry
The My Retina Tracker® Registry is sponsored by the Foundation Fighting Blindness and is for people affected by one of the rare inherited retinal degenerative diseases studied by the Foundation.
- Conditions
- Eye Diseases Hereditary, Retinal Disease, Achromatopsia, Bardet-Biedl Syndrome
- Trial name
- MRTR (NCT02435940)
- Where
- United States
- Volunteers
- 20,000 people, healthy volunteers welcome
- Sponsor
- Foundation Fighting Blindness
Epigastric Pain, Retrosternal Pain
Transient Receptor Potential Vanilloid One (TRPV1) Receptor Expression in Children With Retrosternal Pain
We would like to evaluate the changes in nerve innervation and TRPV1 receptor expression along with microscopic changes associated with heart burn and abdominal pain
- Conditions
- Epigastric Pain, Retrosternal Pain, Esophagitis, Gastrointestinal Symptoms
- Trial name
- TRPV1 (NCT00677378)
- Where
- United States
- Volunteers
- 77 people
- Sponsor
- Medical College of Wisconsin
Hereditary Spastic Paraplegia
STOP-HSP.Net: a Registry for Hereditary Spastic Paraplegia as an Integration Tool for Future Therapeutic Strategies
Our goal is to create a solid and harmonious disease registry of patient affected by hereditary spastic paraplegia (HSP) that facilitates the collection and management of patients' data over time encouraging the research and the…
- Trial name
- NCT06572046
- Where
- Italy
- Volunteers
- 500 people
- Sponsor
- IRCCS Fondazione Stella Maris
Dysautonomia, Familial, Baroreflex Failure Syndrome
Carbidopa for the Treatment of Excessive Blood Pressure Variability
The overall study objectives are to determine whether carbidopa (Lodosyn®) is safe and well tolerated and to assess whether it can inhibit catecholamine-induced paroxysmal hypertension and normalize or reduce the exaggerated…
- Trial name
- CarbiFD (NCT02553265)
- Testing
- Carbidopa Low-Dose, Carbidopa High-Dose
- Where
- 2 sites in United States
- Volunteers
- 22 people
- Sponsor
- NYU Langone Health
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
Hereditary Spastic Paraplegia
Calcium Folinate Treatment of Spastic Paraplegia 56
SPG56 is one of the complicated and early-onset HSP subtypes caused by genetic mutations in CYP2U1. So far, there is no standardized and specific clinical therapy for SPG56.
- Trial name
- CFT-SPG56 (NCT06478238)
- Testing
- calcium folinate
- Where
- China
- Volunteers
- 10 people
- Sponsor
- Shanghai 6th People's Hospital