Clinical trials: Myasthenic Syndrome
Lambert Eaton Myasthenic Syndrome
Treatment of Lambert-Eaton Syndrome With 3,4 DAP
A new drug called 3,4-Diaminopyridine (3,4-DAP) is currently under investigation for treatment of the symptoms of Lambert-Eaton Myasthenic Syndrome (LEMS).
- Trial name
- NCT00704925
- Testing
- 3, 4 DAP
- Where
- United States
- Sponsor
- David P. Richman, MD
Lambert-Eaton Myasthenic Syndrome, Congenital Myasthenic Syndrome
3,4-Diaminopyridine Use in Lambert-Eaton Myasthenic Syndrome(LEMS) and Congenital Myasthenic Syndromes (CMS)
The purpose of this study is to determine the effectiveness and adverse effects of 3,4-diaminopyridine for the treatment of the Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenic Syndromes (CMS).
- Trial name
- LEMS (NCT00872950)
- Testing
- 3,4-DIAMINOPYRIDINE
- Where
- United States
- Sponsor
- Lahey Clinic
Muscle Weakness
Controlled Trial of 3,4-Diaminopyridine (3-4DAP) in Lambert-Eaton Myasthenic Syndrome (LEMS)
The main purpose for this study is to provide access to 3,4 DAP, a drug which has demonstrated to be effective in treating weakness associated with Lambert-Eaton Myasthenic Syndrome.
- Trial name
- 3-4DAP (NCT02090725)
- Testing
- 3-4 Diaminopyridine
- Where
- United States
- Volunteers
- 4 people
- Sponsor
- Jeffrey A. Cohen, MD
Myasthenic Syndromes, Congenital
A Prospective Natural History and Outcome Measure Validation Study of Congenital Myasthenic Syndromes
Background: Congenital myasthenic syndromes (CMSs) are a group of inherited disorders that affect how the nerves communicate with muscles. These can cause many problems that affect how people can move and use their bodies.
- Trial name
- NCT06630650
- Where
- United States
- Volunteers
- 75 people
- Sponsor
- National Institute of Neurological Disorders and Stroke (NINDS)
Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency, Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy)
Congenital Muscle Disease Study of Patient and Family Reported Medical Information
The Congenital Muscle Disease Patient and Proxy Reported Outcome Study (CMDPROS) is a longitudinal 10 year study to identify and trend care parameters, adverse events in the congenital muscle diseases using the Congenital Muscle…
- Conditions
- Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency, Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Fatty Liver and Infantile-onset Cataract Caused by TRAPPC11 Mutations), Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy With Hypoglycosylation of Dystroglycan)
- Trial name
- CMDPROS (NCT01403402)
- Where
- United States
- Volunteers
- 4,000 people
- Sponsor
- Cure CMD
Congenital Myasthenic Syndrome
A Natural History Study in Participants With Congenital Myasthenic Syndromes (CMS) Due to Mutations in DOK7, MUSK, AGRN, or LRP4
Participants will attend up to 4 study visits to collect clinical assessments. The assessments will evaluate participants' symptoms and quality of life to understand disease activity in patients with CMS due to mutations in DOK7…
- Trial name
- NCT06078553
- Where
- 33 sites in United States, Austria and 10 more
- Volunteers
- 100 people
- Sponsor
- argenx
Myasthenic Syndromes, Congenital
Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes
This randomized, double-blind, controlled, outpatient two-period, two-treatment crossover study is designed to evaluate the efficacy and safety of amifampridine phosphate in patients (ages 2 and above) diagnosed with certain…
- Trial name
- NCT02562066
- Testing
- amifampridine phosphate
- Where
- 4 sites in United States
- Volunteers
- 20 people
- Sponsor
- Catalyst Pharmaceuticals, Inc.
Myasthenia Gravis
Univent Tube for Thoracoscopic Thymectomy in Myasthenic Patients
This observational study included consecutive MG patients who underwent thoracoscopic thymectomy with Univent tube intubation under general anesthesia without NMBAs and combined with airway topical anesthesia between January 2016…
- Trial name
- NCT06101407
- Volunteers
- 83 people
- Sponsor
- Nguyen Dang Thu
Myasthenia Gravis
Prevalence and Impact of Fatigue in Myasthenic Patients Followed At Clermont-Ferrand University Hospital - "My" Asthenia
After identifying myasthenic patients meeting the inclusion criteria, telephone contact with patients to suggest that they participate in the study by responding to various questionnaires composed of validated scales used in…
- Trial name
- NCT06877403
- Where
- France
- Volunteers
- 100 people
- Sponsor
- University Hospital, Clermont-Ferrand
Congenital Myasthenic Syndrome
Patients With Congenital Myasthenic Syndrome Will be Treated With Umbilical or Placental Derived Mesenchymal Stem Cell Exosome Solution
Patients with Congenital Myasthenic Syndrome will be treated with Umbilical or Placental Derived Mesenchymal Stem Cell Exosome solution.
- Trial name
- NCT07226726
- Testing
- AlloEx exosomes
- Where
- 2 sites in United States, Greece
- Volunteers
- 20 people
- Sponsor
- The Foundation for Orthopaedics and Regenerative Medicine
Myasthenia Gravis, Thymoma
Incidence and Predictors of Early Clinical Relapse Within Six Months After Thymectomy in Patients With Myasthenia Gravis
Myasthenia gravis is an autoimmune disorder affecting neuromuscular transmission, characterized by fluctuating muscle weakness and fatigue.
- Trial name
- NCT07853378
- Volunteers
- 30 people
- Sponsor
- Assiut University
Congenital Myasthenic Syndrome
Treatment Use of 3,4 Diaminopyridine in Congenital Myasthenia
Congenital myasthenia is a potentially lethal disorder, which, even with careful management, significantly impedes participation in normal daily functions.
- Trial name
- NCT03062631
- Testing
- 3,4-Diaminopyridine
- Where
- United States
- Sponsor
- Ricardo Maselli
Lambert Eaton Myasthenic Syndrome (LEMS)
Treatment of Lambert-Eaton Myasthenic Syndrome (LEMS) With 3, 4 DAP
Lambert Eaton Myasthenic Syndrome (LEMS) is rare neurological disorder that results in muscle weakness and limited reflex activity.
- Trial name
- NCT01378546
- Testing
- 3,4-diaminopyridine
- Where
- United States
- Sponsor
- Louis H. Weimer, MD
Myasthenia Gravis, Myasthenic Syndrome
Patient Observation With Environmental and Wearable Sensors in Myasthenia Gravis
This observational study seeks to investigate the underlying processes of myasthenia gravis by employing multimodal monitoring techniques.
- Conditions
- Myasthenia Gravis, Myasthenic Syndrome, Lambert-Eaton Myasthenic Syndrome
- Trial name
- POWER-MG (NCT06441825)
- Testing
- Increased multimodal observation including digital monitoring
- Where
- Germany
- Volunteers
- 20 people
- Sponsor
- Heinrich-Heine University, Duesseldorf
Spinal Muscular Atrophy, Muscular Dystrophy
National Registry for Egyptian Pediatric Neuromuscular Diseases
Our aim is to establish multi-center national Egyptian database of information for inherited and acquired neuromuscular diseases in infants and children from 0 to 18 years of age.
- Conditions
- Spinal Muscular Atrophy, Muscular Dystrophy, Muscle Diseases, Myasthenic Syndromes
- Trial name
- NCT02124616
- Where
- Egypt
- Volunteers
- 200 people
- Sponsor
- Ain Shams University
Inclusion Body Myositis, Myotonic Dystrophy
Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disorders
Currently, there are no standardised fall risk scores or guidelines on when to use appropriate assistive gait devices (AGDs) for people with neuromuscular disorders (NMDs).
- Conditions
- Inclusion Body Myositis, Myotonic Dystrophy, Limb-girdle and Facioscapulohumeral Muscular Dystrophies, Pompe Disease
- Trial name
- FBIndex (NCT06605612)
- Testing
- FBIndex
- Where
- Germany
- Volunteers
- 108 people
- Sponsor
- LMU Klinikum
Motor Disorders
Patients With ALS and Other Motor Disorders Will be Treated With Umbilical or Placental Mesenchymal Cell Exosome Solution
Patients with ALS and other Motor Discorders will be treated with mesenchymal cell exosome solution.
- Trial name
- NCT07105371
- Testing
- Exosome Secretome
- Where
- 2 sites in United States, Greece
- Volunteers
- 70 people
- Sponsor
- The Foundation for Orthopaedics and Regenerative Medicine
Congenital Myasthenic Syndrome
Efficacy of Albuterol in the Treatment of Congenital Myasthenic Syndromes
The study tests the notion that patients suffering from certain types of congenital myasthenic syndromes are benefitted by the use of Albuterol at doses used in clinical practice.
- Trial name
- NCT01203592
- Testing
- Albuterol
- Where
- United States
- Volunteers
- 21 people
- Sponsor
- Mayo Clinic
Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis
Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease
This single-arm pilot study evaluates the effects of whole-body electrical muscle stimulation (WB-EMS) exercise on neuromuscular and physical function in adults with neuromuscular disease (NMD).
- Conditions
- Neuromuscular Diseases (NMD), Amyotrophic Lateral Sclerosis, Myasthenia Gravis, Lambert-eaton Myasthenic Syndrome
- Trial name
- NCT07478172
- Testing
- Whole-body Electrical Muscle Stimulation Exercise
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- University of Missouri-Columbia
Lambert-Eaton Myasthenic Syndrome, Eaton-Lambert Myasthenic Syndrome
Effectiveness of 3,4-Diaminopyridine in Lambert-Eaton Myasthenic Syndrome
Hypothesis: 3,4-Diaminopyridine base (3,4-DAP) improves Lambert-Eaton Myasthenic Syndrome (LEMS)-related weakness.
- Trial name
- DAPPER (NCT01511978)
- Testing
- Continuous 3,4-DAP
- Where
- 7 sites in United States
- Volunteers
- 32 people
- Sponsor
- Jacobus Pharmaceutical